Study of ASP2957 in Male Participants With X-linked Myotubular Myopathy Who Need Ventilators
Public ClinicalTrials.gov record NCT07052929. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official title
A Phase 1/2, Multicenter, Open-label, Dose Escalation and Expansion Clinical Study to Evaluate the Safety, Tolerability and Preliminary Efficacy of ASP2957 in Male Participants With Invasive Ventilator-dependent X-linked Myotubular Myopathy
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
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 ASP2957. After this, there will be the option for the boys to join another study so they will continue to be checked longer term.
Study identification
- NCT ID
- NCT07052929
- Recruitment status
- Recruiting
- Study type
- Interventional
- Phase
- Phase 1, Phase 2
- Enrollment
- 9 participants
Conditions and interventions
Conditions
Interventions
- ASP2957 Genetic
- Methylprednisolone Drug
- Prednisolone Drug
- Sirolimus Drug
Genetic · Drug
Eligibility (public fields only)
- Age range
- Up to 36 Months
- Sex
- Male
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Dec 14, 2025
- Primary completion
- Oct 30, 2027
- Completion
- Oct 30, 2027
- Last update posted
- Aug 6, 2026
2025 – 2027
United States locations
- U.S. sites
- 4
- U.S. states
- 4
- U.S. cities
- 4
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| Lurie Children's Hospital | Chicago | Illinois | 60611 | Recruiting |
| Boston Children's Hospital | Boston | Massachusetts | 02115 | Recruiting |
| Oregon Health & Science University | Portland | Oregon | 97239 | Recruiting |
| The University of Texas Southwestern Medical Center | Dallas | Texas | 75390 | Recruiting |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
Non-U.S. locations
This page focuses on the U.S. directory. The official record also lists 1 non-U.S. site.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT07052929, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Aug 6, 2026 · Synced Sep 2, 2026
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Open the official record
The complete protocol, eligibility criteria, and contact information for NCT07052929 live on ClinicalTrials.gov.