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Study of ASP2957 in Male Participants With X-linked Myotubular Myopathy Who Need Ventilators
NCT07052929 · View on ClinicalTrials.gov ↗
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 approved therapies for XLMTM. The protein myotubularin is needed for muscle development, movement and breathing. 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 cause low or no levels of myotubularin to be made, so the muscles do not work properly. 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. Researchers have developed ASP2957 to get a healthy MTM1 gene into the body. This could help improve muscle development and function in young children with XLMTM. In this study, ASP2957 will be given to humans for the first time. ASP2957 has the healthy MTM1 gene inside a type of empty (killed) virus. The virus delivers the healthy MTM1 gene directly into cells in the body. It's possible that some boys may have antibodies to the virus if they have previously been infected with a similar virus. The antibodies could stop ASP2957 from working properly and cause an immune reaction to ASP2957. To prevent this, the boys will also be given medicines to lower the immune system. The main aims of this study are to check the safety of ASP2957, how well it is tolerated, and to find a suitable dose of ASP2957. The study was designed in 2 phases. In Phase 1, different small groups of boys will receive lower to higher doses of ASP2957. Each boy will receive a single infusion of ASP2957. Any medical problems will be recorded for each dose. This is done to find a suitable dose of ASP2957 to use in Phase 2. In Phase 2, another small group of young boys will receive a single infusion of ASP2957. The most suitable dose of ASP2957 worked out from Phase 1 will be used. The boys will be checked for up to 1 year after their single infusion of
Conditions Studied
Interventions
- DRUG Sirolimus
- GENETIC ASP2957
- DRUG Methylprednisolone
- DRUG Prednisolone
Study Locations (3)
Illinois
- Lurie Children's Hospital — Chicago
Massachusetts
- Boston Children's Hospital — Boston
Oregon
- Oregon Health & Science University — Portland
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 9 participants |
| Start Date | 2025-12-15 |
| Est. Completion | 2027-10-31 |
| Phase | Phase 1 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT07052929
The ClinicalTrials.gov registry entry for NCT07052929 describes a study currently listed as recruiting. It is categorized as Phase 1, which is the standard way researchers label where a study sits along the investigational pathway from early safety work through later efficacy and post-marketing evaluation. The registered enrollment target is 9 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Astellas Gene Therapies, which has 36 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.
The record links to 1 condition, with X-Linked Myotubular Myopathy appearing as the primary indexed condition, and to 4 interventions — of which Sirolimus is the first listed. Interventions can include drugs, devices, procedures, behavioral programs, or observational arms, and each is tracked as a separate registry field so that downstream queries can filter accurately. When a trial lists multiple interventions, it usually reflects a multi-arm design or a comparison protocol rather than a single treatment being tested in isolation. The brief summary published in the registry is the clearest source of protocol intent and should be read before drawing conclusions from any sidebar tags.
Geographic footprint matters for practical reasons: NCT07052929 reports 3 study locations spanning 3 distinct geographic areas — top geographies include Illinois, Massachusetts, Oregon. A larger site network tends to correlate with broader recruitment capacity, but it does not imply anything about study quality, and site-level enrollment status can diverge from the overall registry status shown above. Every data point on this page comes from the public ClinicalTrials.gov dataset and is reproduced here for reference only; it is not a medical recommendation, an endorsement of the sponsor, or an invitation to enroll. Verify current status, eligibility criteria, and contact details directly at ClinicalTrials.gov, and discuss any participation decision with your own healthcare provider.
Frequently Asked Questions
What is clinical trial NCT07052929 about?
NCT07052929 is a clinical study titled "Study of ASP2957 in Male Participants With X-linked Myotubular Myopathy Who Need Ventilators". 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 approved therapies for XLMTM. The protein myotubularin is needed for muscle development, movement and breathing. A gene called MTM1 tells the body to ...
What is the current status of trial NCT07052929?
This trial is currently recruiting. It is a Phase 1 study. The enrollment target is 9 participants. The study started on 2025-12-15. Estimated completion is 2027-10-31.
What conditions does trial NCT07052929 study?
This clinical trial studies the following conditions: X-Linked Myotubular Myopathy. These conditions were identified from the trial registry and reflect the primary focus areas of the research.
What interventions are being tested in trial NCT07052929?
The interventions under investigation include: Sirolimus (DRUG), ASP2957 (GENETIC), Methylprednisolone (DRUG), Prednisolone (DRUG). Each intervention is being evaluated for safety and efficacy as part of this clinical study.
Who is sponsoring clinical trial NCT07052929?
This trial is sponsored by Astellas Gene Therapies, which has 36 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.
Where is trial NCT07052929 being conducted?
This trial has 3 study locations across Illinois, Massachusetts, Oregon. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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