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
Study identification
- NCT ID
- NCT07052929
- Recruitment status
- Recruiting
- Study type
- Interventional
- Phase
- Phase 1, Phase 2
- Lead sponsor
- Astellas Gene Therapies
- Industry
- 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
- Not listed
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
- Apr 14, 2026
2025 – 2027
United States locations
- U.S. sites
- 3
- U.S. states
- 3
- U.S. cities
- 3
| 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 |
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 Apr 14, 2026 · Synced Apr 22, 2026
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Open the official record
The complete protocol, eligibility criteria, and contact information for NCT07052929 live on ClinicalTrials.gov.