Study record managers: refer to the Data Element Definitions if submitting registration or results information.
研究记录管理器：如果提交注册或结果信息，请参阅数据元素定义。
A type of eligibility criteria that indicates whether people who do not have the condition/disease being studied can participate in that clinical study.
An arm type in which a group of participants receives an intervention/treatment considered to be effective (or active) by health care providers.
An unfavorable change in the health of a participant, including abnormal laboratory findings, that happens during a clinical study or within a certain amount of time after the study has ended. This change may or may not be caused by the intervention/treatment being studied.
A type of eligibility criteria that indicates the age a person must be to participate in a clinical study. This may be indicated by a specific age or the following age groups:
The age groups are:
Child (birth-17)
Adult (18-64)
Older Adult (65+)
A measure of all deaths, due to any cause, that occur during a clinical study.
A method used to assign participants to an arm of a clinical study. The types of allocation are randomized allocation and nonrandomized.
A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
A general description of the clinical trial arm. It identifies the role of the intervention that participants receive. Types of arms include experimental arm, active comparator arm, placebo comparator arm, sham comparator arm, and no intervention arm.
Data collected at the beginning of a clinical study for all participants and for each arm or comparison group. These data include demographics, such as age, sex/gender, race and ethnicity, and study-specific measures (for example, systolic blood pressure, prior antidepressant treatment).
Indicates that the study sponsor or investigator recalled a submission of study results before quality control (QC) review took place. If the submission was canceled on or after May 8, 2018, the date is shown. After submission of study results, a study record cannot be modified until QC review is completed, unless the submission is canceled.
Information required by the Food and Drug Administration Amendments Act of 2007. In general, this is a description of any agreement between the sponsor of a clinical study and the principal investigator (PI) that does not allow the PI to discuss the results of the study or publish the study results in a scientific or academic journal after the study is completed.
A sponsor or investigator may submit a certification to delay submission of results information if they are applying for FDA approval of a new drug or device, or new use of an already approved drug or device. A sponsor or investigator who submits a certification can delay results submission up to 2 years after the certification/extension first submitted date, unless certain events occur sooner. See Delay Results Type in the Results Data Element definitions for more information about this certification.
The date on which information about a certification to delay submission of results or an extension request was first available on ClinicalTrials.gov. ClinicalTrials.gov does not indicate whether the submission was a certification or extension request. There is typically a delay between the date the study sponsor or investigator submitted the certification or extension request and the first posted date.
The date on which the study sponsor or investigator first submitted a certification or an extension request to delay submission of results. A sponsor or investigator who submits a certification can delay results submission up to 2 years after this date, unless certain events occur sooner. There is typically a delay between the date the certification or extension request was submitted and the date the information is first available on ClinicalTrials.gov (certification/extension first posted).
The date on which the study sponsor or investigator first submitted a certification or an extension request that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a certification or extension request one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria. Meeting QC criteria for an extension request does not mean that the National Institutes of Health (NIH) has determined that the request demonstrates good cause. The process for review and granting of extension requests by the NIH is being developed.
In the search feature, the City field is used to find clinical studies with locations in a specific city. The Distance field is used to find studies with locations within the specified distance from a city in number of miles. For example, if you choose Illinois as the state, identifying "Chicago" as the city and "100 miles" as the distance will find all studies listing a location within 100 miles of Chicago.
A research study involving human volunteers (also called participants) that is intended to add to medical knowledge. There are two types of clinical studies: interventional studies (also called clinical trials) and observational studies.
Another name for an interventional study.
The unique identification code given to each clinical study upon registration at ClinicalTrials.gov. The format is "NCT" followed by an 8-digit number (for example, NCT00000419).
An organization other than the sponsor that provides support for a clinical study. This support may include activities related to funding, design, implementation, data analysis, or reporting.
The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.
The name and contact information for the person who can answer enrollment questions for a clinical study. Each location where the study is being conducted may also have a specific contact, who may be better able to answer those questions.
In the search feature, the Country field is used to find clinical studies with locations in a specific country. For example, if you choose the United States, you can then narrow your search by selecting a state and identifying a city and distance.
A type of intervention model describing a clinical trial in which groups of participants receive two or more interventions in a specific order. For example, two-by-two cross-over assignment involves two groups of participants. One group receives drug A during the initial phase of the trial, followed by drug B during a later phase. The other group receives drug B during the initial phase, followed by drug A. So during the trial, participants "cross over" to the other drug. All participants receive drug A and drug B at some point during the trial but in a different order, depending on the group to which they are assigned.
A group of independent scientists who monitor the safety and scientific integrity of a clinical trial. The DMC can recommend to the sponsor that the trial be stopped if it is not effective, is harming participants, or is unlikely to serve its scientific purpose. Members are chosen based on the scientific skills and knowledge needed to monitor the particular trial. Also called a data safety and monitoring board, or DSMB.
A phase of research used to describe exploratory trials conducted before traditional phase 1 trials to investigate how or whether a drug affects the body. They involve very limited human exposure to the drug and have no therapeutic or diagnostic goals (for example, screening studies, microdose studies).
The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.
The number of participants in a clinical study. The "estimated" enrollment is the target number of participants that the researchers need for the study.
A type of eligibility criteria. These are reasons that a person is not allowed to participate in a clinical study.
A way for patients with serious diseases or conditions who cannot participate in a clinical trial to gain access to a medical product that has not been approved by the U.S. Food and Drug Administration (FDA). Also called compassionate use. There are different expanded access types.
Available:Expanded access is currently available for this investigational treatment, and patients who are not participants in the clinical study may be able to gain access to the drug, biologic, or medical device being studied.
No longer available:Expanded access was available for this intervention previously but is not currently available and will not be available in the future.
Temporarily not available:Expanded access is not currently available for this intervention but is expected to be available in the future.
Approved for marketing: The intervention has been approved by the U.S. Food and Drug Administration for use by the public.
Describes the category of expanded access under U.S. Food and Drug Administration (FDA) regulations. There are three types of expanded access:
Individual Patients: Allows a single patient, with a serious disease or condition who cannot participate in a clinical trial, access to a drug or biological product that has not been approved by the FDA. This category also includes access in an emergency situation.
Intermediate-size Population: Allows more than one patient (but generally fewer patients than through a Treatment IND/Protocol) access to a drug or biological product that has not been approved by the FDA. This type of expanded access is used when multiple patients with the same disease or condition seek access to a specific drug or biological product that has not been approved by the FDA.
Treatment IND/Protocol: Allows a large, widespread population access to a drug or biological product that has not been approved by the FDA. This type of expanded access can only be provided if the product is already being developed for marketing for the same use as the expanded access use.
An arm type in which a group of participants receives the intervention/treatment that is the focus of the clinical trial.
In certain circumstances, a sponsor or investigator may request an extension to delay the standard results submission deadline (generally one year after the primary completion date). The request for an extension must demonstrate good cause (for example, the need to preserve the scientific integrity of an ongoing masked trial). All requests must be reviewed and granted by the National Institutes of Health. This process for review and granting of extension requests is being developed. See Delay Results Type in the Results Data Element definitions for more information.
A type of intervention model describing a clinical trial in which groups of participants receive one of several combinations of interventions. For example, two-by-two factorial assignment involves four groups of participants. Each group receives one of the following pairs of interventions: (1) drug A and drug B, (2) drug A and a placebo, (3) a placebo and drug B, or (4) a placebo and a placebo. So during the trial, all possible combinations of the two drugs (A and B) and the placebos are given to different groups of participants.
A FDAAA 801 Violation is shown on a study record when the U.S. Food and Drug Administration (FDA) has issued a Notice of Noncompliance to the responsible party of an applicable clinical trial. A Notice of Noncompliance indicates that the FDA has determined the responsible party was not in compliance with the registration or results reporting requirements for the clinical trial under the Food and Drug Administration Amendments Act of 2007, Section 801 (FDAAA 801).
The National Library of Medicine (NLM) is required by FDAAA 801 to add information to a study record about any FDAAA 801 Violation. This information is provided by the FDA. There are three categories of information that may be included:
Violation: Shown when the FDA issues a Notice of Noncompliance and posts the Notice of Noncompliance on its designated webpage. There are three types of violations:
Failure to submit required clinical trial information
Submission of false or misleading clinical trial information
Failure to submit primary and secondary outcomes
Correction: Shown when the FDA confirms that the responsible party has updated the study record to correct the violation and posts the correction notice on its designated webpage. Because of the time for FDA review and processing, there may be a delay between the date when the study record was updated and the addition of correction information to the FDAAA 801 Violation information.
Penalty: Shown when the FDA imposes a penalty for the violation and posts the penalty notice on its designated webpage.
The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
U.S. Public Law 110-85, which was enacted on September 27, 2007. Section 801 of FDAAA amends Section 402 of the U.S. Public Health Service Act to expand ClinicalTrials.gov and create a clinical study results database. For more information on FDAAA 801, see the History, Policies, and Laws page on this site.
Describes the organization that provides funding or support for a clinical study. This support may include activities related to funding, design, implementation, data analysis, or reporting. Organizations listed as sponsors and collaborators for a study are considered the funders of the study. ClinicalTrials.gov refers to four types of funders:
U.S. National Institutes of Health
Other U.S. Federal agencies (for example, Food and Drug Administration, Centers for Disease Control and Prevention, or U.S. Department of Veterans Affairs)
Industry (for example: pharmaceutical and device companies)
All others (including individuals, universities, and community-based organizations)
A type of eligibility criteria that indicates whether eligibility to participate in a clinical study is based on a person's self-representation of gender identity. Gender identity refers to a person's own sense of gender, which may or may not be the same as their biological sex.
A group or subgroup of participants in an observational study that is assessed for biomedical or health outcomes.
A group of people who review, approve, and monitor the clinical study's protocol. Their role is to protect the rights and welfare of people participating in a study (referred to as human research subjects), such as reviewing the informed consent form. The group typically includes people with varying backgrounds, including a community member, to make sure that research activities conducted by an organization are completely and adequately reviewed. Also called an institutional review board, or IRB, or an ethics committee.
A type of eligibility criteria. These are the reasons that a person is allowed to participate in a clinical study.
A process used by researchers to communicate to potential and enrolled participants the risks and potential benefits of participating in a clinical study.
The document used in the informed consent or process.
The general design of the strategy for assigning interventions to participants in a clinical study. Types of intervention models include: single group assignment, parallel assignment, cross-over assignment, and factorial assignment.
A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
A type of clinical study in which participants are assigned to groups that receive one or more intervention/treatment (or no intervention) so that researchers can evaluate the effects of the interventions on biomedical or health-related outcomes. The assignments are determined by the study's protocol. Participants may receive diagnostic, therapeutic, or other types of interventions.
A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
The most recent date on which the study sponsor or investigator submitted changes to a study record to ClinicalTrials.gov. There is typically a delay of a few days between the last update submitted date and when the date changes are posted on ClinicalTrials.gov (the last update posted date).
The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
Countries in which research facilities for a study are located. A country is listed only once, even if there is more than one facility in the country. The list includes all countries as of the last update submitted date; any country for which all facilities were removed from the study record are listed under removed location countries.
In the search feature, the Location terms field is used to narrow a search by location-related terms other than Country, State, and City or distance. For example, you may enter a specific facility name (such as National Institutes of Health Clinical Center) or a part of a facility name (such as Veteran for studies listing Veterans Hospital or Veteran Affairs in the facility name). Note: Not all study records include this level of detail about locations.
A clinical trial design strategy in which one or more parties involved in the trial, such as the investigator or participants, do not know which participants have been assigned which interventions. Types of masking include: open label, single blind masking, and double-blind masking.
A unique identification code given to each clinical study record registered on ClinicalTrials.gov. The format is "NCT" followed by an 8-digit number (for example, NCT00000419). Also called the ClinicalTrials.gov identifier.
An arm type in which a group of participants does not receive any intervention/treatment during the clinical trial.
A type of clinical study in which participants are identified as belonging to study groups and are assessed for biomedical or health outcomes. Participants may receive diagnostic, therapeutic, or other types of interventions, but the investigator does not assign participants to a specific interventions/treatment.
A patient registry is a type of observational study.
The general design of the strategy for identifying and following up with participants during an observational study. Types of observational study models include cohort, case-control, case-only, case-cross-over, ecologic or community studies, family-based, and other.
An adverse event that is not a serious adverse event, meaning that it does not result in death, is not life-threatening, does not require inpatient hospitalization or extend a current hospital stay, does not result in an ongoing or significant incapacity or interfere substantially with normal life functions, and does not cause a congenital anomaly or birth defect; it also does not put the participant in danger and does not require medical or surgical intervention to prevent one of the results listed above.
Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
In the search feature, the Other terms field is used to narrow a search. For example, you may enter the name of a drug or the NCT number of a clinical study to limit the search to study records that contain these words.
For clinical trials, a planned measurement described in the protocol that is used to determine the effect of an intervention/treatment on participants. For observational studies, a measurement or observation that is used to describe patterns of diseases or traits, or associations with exposures, risk factors, or treatment. Types of outcome measures include primary outcome measure and secondary outcome measure.
A type of intervention model describing a clinical trial in which two or more groups of participants receive different interventions. For example, a two-arm parallel assignment involves two groups of participants. One group receives drug A, and the other group receives drug B. So during the trial, participants in one group receive drug A "in parallel" to participants in the other group, who receive drug B.
A summary of the progress of participants through each stage of a clinical study, by study arm or group/cohort. This includes the number of participants who started, completed, and dropped out of the study.
A type of observational study that collects information about patients' medical conditions and/or treatments to better understand how a condition or treatment affects patients in the real world.
The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
A phase of research to describe clinical trials that focus on the safety of a drug. They are usually conducted with healthy volunteers, and the goal is to determine the drug's most frequent and serious adverse events and, often, how the drug is broken down and excreted by the body. These trials usually involve a small number of participants.
A phase of research to describe clinical trials that gather preliminary data on whether a drug works in people who have a certain condition/disease (that is, the drug's effectiveness). For example, participants receiving the drug may be compared to similar participants receiving a different treatment, usually an inactive substance (called a placebo) or a different drug. Safety continues to be evaluated, and short-term adverse events are studied.
A phase of research to describe clinical trials that gather more information about a drug's safety and effectiveness by studying different populations and different dosages and by using the drug in combination with other drugs. These studies typically involve more participants.
A phase of research to describe clinical trials occurring after FDA has approved a drug for marketing. They include postmarket requirement and commitment studies that are required of or agreed to by the study sponsor. These trials gather additional information about a drug's safety, efficacy, or optimal use.
Describes trials without FDA-defined phases, including trials of devices or behavioral interventions.
An inactive substance or treatment that looks the same as, and is given in the same way as, an active drug or intervention/treatment being studied.
An arm type in which a group of participants receives a placebo during a clinical trial.
The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "estimated" primary completion date is the date that the researchers think will be the primary completion date for the study.
In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
The main reason for the clinical trial. The types of primary purpose are: treatment, prevention, diagnostic, supportive care, screening, health services research, basic science, and other.
The person who is responsible for the scientific and technical direction of the entire clinical study.
The written description of a clinical study. It includes the study's objectives, design, and methods. It may also include relevant scientific background and statistical information.
National Library of Medicine (NLM) staff perform a limited review of submitted study records for apparent errors, deficiencies, or inconsistencies. NLM staff identify potential major and advisory issues and provide comments directly to the study sponsor or investigator. Major issues identified in QC review must be addressed or corrected (see First submitted that met QC criteria and Results first submitted that met QC criteria). Advisory issues are suggestions to help improve the clarity of the record. NLM staff do not verify the scientific validity or relevance of the submitted information. The study sponsor or investigator is responsible for ensuring that the studies follow all applicable laws and regulations.
A type of allocation strategy in which participants are assigned to the arms of a clinical trial by chance.
Not yet recruiting: The study has not started recruiting participants.
Recruiting: The study is currently recruiting participants.
Enrolling by invitation: The study is selecting its participants from a population, or group of people, decided on by the researchers in advance. These studies are not open to everyone who meets the eligibility criteria but only to people in that particular population, who are specifically invited to participate.
Active, not recruiting: The study is ongoing, and participants are receiving an intervention or being examined, but potential participants are not currently being recruited or enrolled.
Suspended: The study has stopped early but may start again.
Terminated: The study has stopped early and will not start again. Participants are no longer being examined or treated.
Completed: The study has ended normally, and participants are no longer being examined or treated (that is, the last participant's last visit has occurred).
Withdrawn: The study stopped early, before enrolling its first participant.
Unknown: A study on ClinicalTrials.gov whose last known status was recruiting; not yet recruiting; or active, not recruiting but that has passed its completion date, and the status has not been last verified within the past 2 years.
The process of submitting and updating summary information about a clinical study and its protocol, from its beginning to end, to a structured, public Web-based study registry that is accessible to the public, such as ClinicalTrials.gov.
Countries that appeared under listed location countries but were removed from the study record by the sponsor or investigator.
A grouping of participants in a clinical study that is used for summarizing the data collected during the study. This grouping may be the same as or different from a study arm or group.
The person responsible for submitting information about a clinical study to ClinicalTrials.gov and updating that information. Usually the study sponsor or investigator.
A structured online system, such as the ClinicalTrials.gov results database, that provides the public with access to registration and summary results information for completed or terminated clinical studies. A study with results available on ClinicalTrials.gov is described as having the results "posted."
Note: The ClinicalTrials.gov results database became available in September 2008. Older studies are unlikely to have results available in the database.
Indicates that the sponsor or investigator submitted a certification or extension request.
The date on which summary results information was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay between the date the study sponsor or investigator first submits summary results information (the results first submitted date) and the results first posted date. Some results information may be available at an earlier date if Results First Posted with QC Comments.
The date on which summary results information was first available on ClinicalTrials.gov with quality control review comments from the National Library of Medicine (NLM) identifying major issues that must be addressed by the sponsor or investigator. As of January 1, 2020, initial results submissions for applicable clinical trials (ACTs) that do not meet quality control review criteria will be publicly posted on ClinicalTrials.gov with brief standardized major comments. Accordingly, the Results First Posted with QC Comments date may be earlier than the Results First Posted date for an ACT with summary results information that is not consistent with NLM quality control review criteria.
The date on which the study sponsor or investigator first submits a study record with summary results information. There is typically a delay between the results first submitted date and when summary results information becomes available on ClinicalTrials.gov (the results first posted date).
The date on which the study sponsor or investigator first submits a study record with summary results information that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit results information one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
The date on which the National Library of Medicine provided quality control (QC) review comments to the study sponsor or investigator. The sponsor or investigator must address major issues identified in the review comments. If there is a date listed for results returned after quality control review, but there is not a subsequent date listed for results submitted to ClinicalTrials.gov, this means that the submission is pending changes by the sponsor or investigator.
Indicates that the study sponsor or investigator has submitted summary results information for a clinical study to ClinicalTrials.gov but the quality control (QC) review process has not concluded.
The results submitted date indicates when the study sponsor or investigator first submitted summary results information or submitted changes to summary results information. Submissions with changes are typically in response to QC review comments from the National Library of Medicine (NLM). If there is a date listed for results submitted to ClinicalTrials.gov, but there is not a subsequent date listed for results returned after quality control review, this means that the submission is pending review by NLM.
In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
An adverse event that results in death, is life-threatening, requires inpatient hospitalization or extends a current hospital stay, results in an ongoing or significant incapacity or interferes substantially with normal life functions, or causes a congenital anomaly or birth defect. Medical events that do not result in death, are not life-threatening, or do not require hospitalization may be considered serious adverse events if they put the participant in danger or require medical or surgical intervention to prevent one of the results listed above.
A type of eligibility criteria that indicates the sex of people who may participate in a clinical study (all, female, male). Sex is a person's classification as female or male based on biological distinctions. Sex is distinct from gender-based eligibility.
An arm type in which a group of participants receives a procedure or device that appears to be the same as the actual procedure or device being studied but does not contain active processes or components.
A type of intervention model describing a clinical trial in which all participants receive the same intervention/treatment.
In Advanced Search, the Sort studies by option is used to change the order of studies listed on the Search Results page. You can sort by Relevance or Newest First:
Relevance: Studies that best match your search terms appear higher in the search results list. This is the default display for all searches.
Newest First: Studies with the most recent First posted dates appear higher in the search results list.
The organization or person who initiates the study and who has authority and control over the study.
In the search feature, the State field is used to find clinical studies with locations in a specific state within the United States. If you choose United States in the Country field, you can search for studies with locations in a specific state.
The written description of the statistical considerations and methods for analyzing the data collected in the clinical study.
Indicates the current recruitment status or the expanded access status.
The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "estimated" study completion date is the date that the researchers think will be the study completion date.
The investigative methods and strategies used in the clinical study.
Refers to the type of documents that the study sponsor or principal investigator may add to their study record. These include a study protocol, statistical analysis plan, and informed consent form.
Identifiers that are assigned to a clinical study by the study's sponsor, funders, or others. They include unique identifiers from other trial study registries and National Institutes of Health grant numbers. Note: ClinicalTrials.gov assigns a unique identification code to each clinical study registered on ClinicalTrials.gov. Also called the NCT number, the format is "NCT" followed by an 8-digit number (for example, NCT00000419).
An entry on ClinicalTrials.gov that contains a summary of a clinical study's protocol information, including the recruitment status; eligibility criteria; contact information; and, in some cases, summary results. Each study record is assigned a ClinicalTrials.gov identifier, or NCT number.
A structured online system, such as ClinicalTrials.gov, that provides the public with access to summary information about ongoing and completed clinical studies.
A study record that includes the summary results posted in the ClinicalTrials.gov results database. Summary results information includes participant flow, baseline characteristics, outcome measures, and adverse events (including serious adverse events).
The actual date on which the first participant was enrolled in a clinical study. The "estimated" study start date is the date that the researchers think will be the study start date.
Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
The date on which the study sponsor or investigator submitted a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria.
The official title of a protocol used to identify a clinical study or a short title written in language intended for the lay public.
The acronym or initials used to identify a clinical study (not all studies have one). For example, the title acronym for the Women's Health Initiative is "WHI."
A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
An agency within the U.S. Department of Health and Human Services. AHRQ's mission is to produce evidence to make health care safer, higher quality, more accessible, equitable, and affordable, and to work within the U.S. Department of Health and Human Services and with other partners to make sure that the evidence is understood and used.
An agency within the U.S. Department of Health and Human Services. The FDA is responsible for protecting the public health by making sure that human and veterinary drugs, vaccines and other biological products, medical devices, the Nation's food supply, cosmetics, dietary supplements, and products that give off radiation are safe, effective, and secure.
A type of recruitment status. It identifies a study on ClinicalTrials.gov whose last known status was recruiting; not yet recruiting; or active, not recruiting but that has passed its completion date, and the status has not been verified within the past 2 years. Studies with an unknown status are considered closed studies.
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ClinicalTrials.gov is a website and online database of clinical research studies and information about their results. The National Library of Medicine (NLM) maintains the website. The study sponsor or investigator submits information about their study to ClinicalTrials.gov and is responsible for the safety, science, and accuracy of any study they list.
Before joining a study, talk to your health care professional about possible risks and benefits. To learn more about taking part in studies, read Learn About Studies(https://clinicaltrials.gov/study-basics/learn-about-studies).
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Randomized Double-Blind Placebo-Controlled Adaptive Design Trial Of Intrathecally Administered Autologous Mesenchymal Stem Cells In Multiple System Atrophy 鞘内注射自体间充质干细胞治疗多系统萎缩的随机双盲安慰剂对照自适应设计试验
ClinicalTrials.gov ID NCT05167721
Sponsor Mayo Clinic 赞助梅奥诊所
Information provided by Wolfgang Singer, MD, Mayo Clinic (Responsible Party) 信息由 Mayo Clinic 医学博士 Wolfgang Singer（责任方）提供
Multiple system atrophy (MSA) is a rare, rapidly progressive, and invariably fatal neurological condition characterized by autonomic failure, parkinsonism, and/or ataxia.
There is no available treatment to slow or halt disease progression. 多系统萎缩 (MSA) 是一种罕见、快速进展且总是致命的神经系统疾病，其特征为自主神经衰竭、帕金森症和/或共济失调。没有可用的治疗方法可以减缓或阻止疾病进展。
The purpose of this study is to assess optimal dosing frequency, effectiveness and safety of adipose-derived autologous mesenchymal stem cells delivered into the spinal fluid of patients with MSA. 本研究的目的是评估将脂肪来源的自体间充质干细胞输送到 MSA 患者脊髓液中的最佳给药频率、有效性和安全性。
Funding source: FDA Office of Orphan Product Development (OOPD), Mayo Clinic Executive Dean for Research Transformational Award, Mayo Clinic Regenerative Medicine, and Mayo Clinic Department of Neurology. 资金来源：FDA 孤儿产品开发办公室 (OOPD)、梅奥诊所研究转型奖执行院长、梅奥诊所再生医学和梅奥诊所神经病学部。
Multiple system atrophy (MSA) is a rare, rapidly progressive, and invariably fatal neurodegenerative disease for which there is no disease-modifying treatment. 多系统萎缩症（MSA）是一种罕见的、快速进展且总是致命的神经退行性疾病，目前尚无缓解疾病的治疗方法。
Recent insights into pathophysiologic mechanisms suggest a crucial role of deprivation of neurotrophic factors which have been shown to be secreted by mesenchymal stem cells (MSCs). 最近对病理生理机制的见解表明，神经营养因子的剥夺发挥着至关重要的作用，这些因子已被证明是由间充质干细胞（MSC）分泌的。
In a recent phase I/II study adipose-derived autologous MSCs were delivered intrathecally to patients with early MSA utilizing a dose-escalation design. 在最近的一项 I/II 期研究中，利用剂量递增设计，将脂肪来源的自体 MSC 鞘内递送至早期 MSA 患者。
At a dose of 50 million MSCs, injections were generally well tolerated, but thickening of cauda equina nerve roots was observed which was either asymptomatic or associated with low back pain. 在 5000 万个 MSC 的剂量下，注射通常具有良好的耐受性，但观察到马尾神经根增厚，这要么是无症状的，要么与腰痛相关。
The rate of disease progression assessed using the Unified MSA Rating Scale (UMSARS) was markedly slower compared to a matched control group. 与匹配的对照组相比，使用统一 MSA 评定量表 (UMSARS) 评估的疾病进展速度明显较慢。
An even more favorable side effect profile and virtually lack of disease progression was seen in an add-on cohort receiving 25 million MSCs per injection.
Neurofilament light chain, an index of central axonal degeneration, decreased in all patients receiving that dose. 每次注射接受 2500 万间充质干细胞的附加队列中观察到更有利的副作用，并且几乎没有疾病进展。所有接受该剂量的患者的神经丝轻链（中央轴突变性指标）均有所下降。
MSC administrations resulted in a marked, dose-dependent increase of neurotrophic factors in CSF.
2-year survival was significantly higher than observed in natural history cohorts. MSC 给药导致脑脊液中神经营养因子显着、剂量依赖性增加。 2 年生存率显着高于自然史队列中观察到的水平。
Based on these findings we are now conducting a double-blind, placebo-controlled, adaptive design phase II trial of adipose-derived intrathecal autologous MSCs in MSA with the goal to establish optimal treatment frequency and simultaneously derive placebo-controlled efficacy and safety data in preparation for a multicenter phase III trial. 基于这些发现，我们现在正在进行一项双盲、安慰剂对照、适应性设计的 II 期试验，对脂肪来源的鞘内自体 MSC 治疗 MSA，目的是确定最佳治疗频率，同时得出安慰剂对照的疗效和安全性数据。为多中心 III 期试验做准备。
Up to 76 adult subjects with MSA will be enrolled.
To ensure a homogenous patient population with comparable rates of disease progression, we will restrict the study to early cases but still fulfilling strictest diagnostic consensus criteria. 将招募最多 76 名患有 MSA 的成年受试者。为了确保患者群体具有可比的疾病进展率，我们将研究限制在早期病例，但仍满足最严格的诊断共识标准。
Participants will undergo a subcutaneous fat biopsy to derive autologous MSCs, which are cultured, expanded, and prepared for delivery in Mayo's Cell Therapeutics Lab. 参与者将接受皮下脂肪活检以获得自体间充质干细胞，这些干细胞在梅奥的细胞治疗实验室进行培养、扩增和准备交付。
In a first phase, subjects will be randomized 1:1:1 to receive 25 million MSCs at two different injection intervals (every 6 months or every 3 months) as the two active arms or lactated Ringer's solution as the placebo arm. 在第一阶段，受试者将按照 1:1:1 的比例随机分配，以两个不同的注射间隔（每 6 个月或每 3 个月）接受 2500 万个 MSC，作为两个活性组，或乳酸林格氏溶液作为安慰剂组。
A recruitment hold after half the subjects have been enrolled will allow for an interim futility and efficacy analysis to select the "winner" active treatment assuming futility criteria are not met. 一半受试者入组后暂停招募将允许进行临时无效性和功效分析，以选择“获胜者”积极治疗（假设不满足无效性标准）。
The study will then restart recruiting the second half of subjects utilizing 2:1 randomization ("winner" active: placebo). 然后，该研究将利用 2:1 随机化重新开始招募后半部分受试者（“获胜者”主动：安慰剂）。
Patients undergo clinical assessments at baseline, 3, 6, 9, and 12 months to derive the primary endpoint, the rate of disease progression assessed using UMSARS total and a mixed effects regression model. 患者在基线、3、6、9 和 12 个月时接受临床评估，以获得主要终点、使用 UMSARS 总数和混合效应回归模型评估的疾病进展率。
MRI of the head and lumbar spine will be completed at baseline and 12 months to expand safety data and to assess the rate of atrophy of selected brain regions using morphometric measures as surrogate markers of disease progression. 头部和腰椎的 MRI 将在基线和 12 个月内完成，以扩展安全数据并使用形态测量指标作为疾病进展的替代标志来评估选定大脑区域的萎缩率。
Spinal fluid before and after administrations, as well as stem cell product media will be collected to further explore biological properties and effects of MSCs and to explore selected spinal fluid markers as biomarkers of disease progression. 将收集给药前后的脊髓液以及干细胞产品培养基，以进一步探索 MSC 的生物学特性和作用，并探索选定的脊髓液标志物作为疾病进展的生物标志物。
This section provides the contact details for those conducting the study, and information on where this study is being conducted. 本节提供了进行研究的人员的联系方式以及有关研究进行地点的信息。
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments. 研究人员寻找符合某种描述（称为资格标准）的人。这些标准的一些例子是一个人的一般健康状况或先前的治疗。
Males or females aged 30-70 years, who are willing and able to give informed consent. 年龄30-70岁、愿意并且能够给予知情同意的男性或女性。
Clinical diagnosis of MSA, fulfilling consensus criteria for probable MSA. MSA 的临床诊断，符合可能的 MSA 的共识标准。
UMSARS I (omitting question 11) between 5 and 17, and able to walk unaided (i.e.
able to walk at least 50 yards without the use of a cane or walker, and without other support such as holding on to an arm or touching walls). UMSARS I（省略问题 11）在 5 至 17 岁之间，并且能够独立行走（即能够在不使用拐杖或助行器，并且没有其他支撑（例如扶着手臂或接触墙壁）的情况下行走至少 50 码） 。
Anticipated survival of at least 3 years in the opinion of the investigator. 研究者认为预期生存期至少为 3 年。
Normal cognition as assessed by the Montreal Cognitive Assessment (MOCA).
We will require a value ≥26. 通过蒙特利尔认知评估 (MOCA) 评估的正常认知。我们需要一个值≥26。
Exclusion Criteria: 排除标准：
Pregnant or breastfeeding women, and women of childbearing potential who do not agree to practice an acceptable method of birth control. 孕妇或哺乳期妇女，以及不同意采取可接受的节育方法的育龄妇女。
Acceptable methods of birth control in this study are: surgical sterilization, intrauterine devices, partner's vasectomy, a double-protection method (condom or diaphragm with spermicide), hormonal contraceptive drug (i.e., oral contraceptive, contraceptive patch, long-acting injectable contraceptive) with a required second mode of contraception. 本研究中可接受的节育方法有：手术绝育、宫内节育器、伴侣输精管结扎术、双重保护方法（避孕套或杀精剂隔膜）、激素避孕药（即口服避孕药、避孕贴剂、长效注射避孕药）需要采取第二种避孕方式。
Participants with a clinically significant or unstable medical or surgical condition that, in the opinion of the investigator, might preclude safe completion of the study or might affect study results. 受试者患有临床显着或不稳定的医疗或手术状况，研究者认为这些状况可能会妨碍研究的安全完成或可能影响研究结果。
These include conditions causing significant CNS or autonomic dysfunction, clinically significant peripheral neuropathy, active malignant neoplasm, amyloidosis, active autoimmune disease, immunocompromised state, active infection, congestive heart failure (NYHA III or IV), recent (<6 months) myocardial infarction, history of stoke with residual deficits, uncontrolled diabetes mellitus, alcoholism, orthopedic problems that compromise mobility and activity of daily living, significant liver or kidney disease, thrombocytopenia (<50 x 109/L), disorders affecting coagulation, and patients on active anticoagulation. 这些包括导致严重中枢神经系统或自主神经功能障碍、临床上显着的周围神经病变、活动性恶性肿瘤、淀粉样变性、活动性自身免疫性疾病、免疫功能低下状态、活动性感染、充血性心力衰竭（NYHA III 或 IV）、近期（<6 个月）心肌梗塞、有残余缺陷的中风病史、不受控制的糖尿病、酗酒、影响日常生活能力和活动的骨科问题、严重的肝脏或肾脏疾病、血小板减少症（<50 x 109/L）、影响凝血的疾病以及正在积极抗凝的患者。
Participants who have taken any investigational products within 90 days prior to baseline, or with expected effects lasting beyond 60 days prior to baseline. 参与者在基线前 90 天内服用过任何研究产品，或预期效果持续超过基线前 60 天。
Medications that could affect clinical evaluations are permitted but need to be withdrawn at least four half-lives prior to study visits.
Those include medications used to treat motor symptoms, such as levodopa and other anti-Parkinsonian medications. 允许使用可能影响临床评估的药物，但需要在研究访问前至少四个半衰期撤回。这些包括用于治疗运动症状的药物，例如左旋多巴和其他抗帕金森病药物。
Patients with contraindication to any of the study procedures, in particular MRI scanning. 对任何研究程序（尤其是 MRI 扫描）有禁忌症的患者。
30 Years to 70 Years (Adult, Older Adult )
30 岁至 70 岁（成人、老年人）
Sexes Eligible for Study 适合学习的性别
All
Accepts Healthy Volunteers 接受健康志愿者
No
Study Plan 学习计划
This section provides details of the study plan, including how the study is designed and what the study is measuring.
本节提供研究计划的详细信息，包括研究的设计方式以及研究的测量内容。
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Design Details
Primary Purpose : Treatment
Allocation : Randomized
Interventional Model : Parallel Assignment
Interventional Model Description: Randomized Double-Blind Placebo-Controlled Adaptive Design Trial
Placebo (lactated Ringer's) administered intrathecally every 3 months for 4 injections
Intervention/Treatment
Other: Placebo
Placebo administered intrathecally
Primary Outcome Measures
Outcome Measure
Measure Description
Time Frame
Change in UMSARS total (= UMSARS I + UMSARS II) score
Rate of disease progression assessed using the change in the UMSARS total (= UMSARS I + UMSARS II) score
12 months
Secondary Outcome Measures
Outcome Measure
Measure Description
Time Frame
Change in UMSARS I score
Rate of disease progression assessed using the change in the UMSARS I score
12 months
Change in UMSARS II score
Rate of disease progression assessed using the change in the UMSARS II score
12 months
Change in modified UMSARS score
Rate of disease progression assessed using a modified UMSARS scale comprising selected items of UMSARS that reflect clinically most meaningful aspects of the disease
12 months
Change in COMPASS select score
Progression in autonomic symptoms assessed using COMPASS select
12 months
Rate of atrophy of selected brain regions
Rate of atrophy and diffusivity change of selected brain regions assessed using MRI morphometry
12 months
Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
Mayo Clinic
Investigators
Principal Investigator:Wolfgang Singer, MD,Mayo Clinic
Publications
The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Login to the Protocol Registration and Results System 登录协议注册和结果系统
The ClinicalTrials.gov Protocol Registration and Results System (PRS) is used by sponsors and investigators to register studies and submit study results. Only they can enter studies and report results. Sponsors and investigators must have a PRS account to register a study. 申办者和研究者使用 ClinicalTrials.gov 方案注册和结果系统 (PRS) 来注册研究并提交研究结果。只有他们才能参与研究并报告结果。申办者和研究者必须拥有 PRS 帐户才能注册研究。 If you do not have an account, learn how to apply for a PRS account. 如果您没有账户，请了解如何申请 PRS 账户。
If you are not a sponsor or investigator, you can look for studies on the homepage. For general information on clinical research, you can learn about studies. 如果您不是赞助商或研究者，您可以在主页上查找研究。有关临床研究的一般信息，您可以了解研究。
To proceed to the PRS, please verify that you are not a robot. 要继续执行 PRS，请确认您不是机器人。
