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NCT03199469 · ClinicalTrials.gov registry record · Phase 2
A Study of AT132 in Young Children With X-Linked Myotubular Myopathy (XLMTM)
A Phase 2 study of X-Linked Myotubular Myopathy, sponsored by Astellas Gene Therapies.
- Active
- Registry status
- Phase 2
- Development phase
- 27
- Enrollment target
- 6
- Study locations
NCT03199469 is a Phase 2 study of X-Linked Myotubular Myopathy that is active but no longer recruiting, run by Astellas Gene Therapies. The registered enrollment target is 27 participants, below the 31-participant average among 3 other X-Linked Myotubular Myopathy trials with a reported enrollment target (13% lower). The trial reports 6 study locations across 5 states.
The verdict
NCT03199469, a Phase 2 study of X-Linked Myotubular Myopathy, is active but no longer recruiting, sponsored by Astellas Gene Therapies.
- ACTIVE NOT RECRUITING
- Registry status
- Phase 2
- Development phase
- 27 participants
- Enrollment target
- 6
- Study locations
Study Summary
X-linked myotubular myopathy (XLMTM) is a rare and serious condition present at birth where the muscles do not work properly. There are currently no therapies for this serious condition. The protein myotubularin is needed for muscle development and movement. A gene called MTM1 tells the body to make myotubularin. XLMTM is caused by changes, or mutations, in the MTM1 gene. Changes in the MTM1 gene causes low levels of myotubularin to be made, so the muscles do not work properly. XLMTM may also affect the liver, and in some cases, this can be dangerous and threaten the patient´s life. Gene therapy is a way of getting a healthy copy of a gene into the body. This allows the body's cells to make a normal protein that may reduce disease symptoms. AT132 is a gene therapy that gets a healthy MTM1 gene into the body to help improve muscle development and function in young children with the disease. AT132 does not treat liver disease, and because of the way the treatment works, it may make liver problems worse. AT132 was the gene therapy treatment given to children who participated in this study and is not available to the public. In this study, AT132 was given to children for the first time. Due to the occurrence of severe complications and fatalities associated with administration of AT132, the study has been stopped and no further participants will be enrolled. The main aim of the study is to check how long young children need machines to support breathing (ventilation support) after AT132. Due to the occurrence of severe complications and fatalities associated with administration of AT132, the study has been stopped and no further participants will be enrolled. This study included children with XLMTM under 5 years old who had breathing problems caused by XLMTM. They couldn't take part if they were born prematurely, recently had surgery, had liver disease or other condition or disease the study doctor thought was medically important. The study did enroll participants
Conditions Studied
Interventions
- GENETIC Resamirigene bilparvovec
Study Locations (6)
Other
- Hopital Armad Trousseau - Paris
- Kinderklinik und Kinderpoliklinik im Dr. Von Haunerschen Kinderspital Klinikum der Universitat Munchen - München
California
- UCLA Medical Center - Los Angeles
Illinois
- Ann & Robert H Lurie Children's Hospital of Chicago - Chicago
Maryland
- National Institute of Neurological Disorders and Stroke/NIH Porter - Bethesda
Ontario
- Hospital for Sick Children - Toronto
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 27 participants |
| Start Date | 2017-08-02 |
| Est. Completion | 2030-03-31 |
| Phase | Phase 2 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT03199469
The ClinicalTrials.gov registry entry for NCT03199469 describes a study currently listed as active not recruiting, categorized as Phase 2. The registered enrollment target is 27 participants, a figure that helps gauge the scale of data the investigators plan to collect, below the 31-participant average among 3 other X-Linked Myotubular Myopathy trials with a reported enrollment target (13% lower). The listed sponsor is Astellas Gene Therapies, which has 7 total studies on file at ClinicalTrials.gov.
The record links to 1 condition, with X-Linked Myotubular Myopathy appearing as the primary indexed condition, and to 1 intervention - of which Resamirigene bilparvovec is the first listed.
NCT03199469 reports 6 study locations spanning 5 distinct geographic areas - top geographies include Other, California, Illinois.
Frequently Asked Questions
What is clinical trial NCT03199469 about?
NCT03199469 is a clinical study titled "A Study of AT132 in Young Children With X-Linked Myotubular Myopathy (XLMTM)". X-linked myotubular myopathy (XLMTM) is a rare and serious condition present at birth where the muscles do not work properly. There are currently no therapies for this serious condition. The protein myotubularin is needed for muscle development and movement. A gene called MTM1 tells the body to mak...
What is the current status of trial NCT03199469?
This trial is currently active not recruiting. It is a Phase 2 study. The enrollment target is 27 participants. The study started on 2017-08-02. Estimated completion is 2030-03-31.
What conditions does trial NCT03199469 study?
This clinical trial studies the following conditions: X-Linked Myotubular Myopathy.
What interventions are being tested in trial NCT03199469?
The interventions under investigation include: Resamirigene bilparvovec (GENETIC).
Who is sponsoring clinical trial NCT03199469?
This trial is sponsored by Astellas Gene Therapies, which has 7 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT03199469 being conducted?
This trial has 6 study locations across California, Illinois, Maryland, Ontario. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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