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A Study to Check Liver Health in Boys With XLMTM, a Serious Genetic Muscle Condition

Study acronym: EXCEL
StatusRecruiting
PhaseNot specified
Started
View on ClinicalTrials.gov ↗
Trial flagged as At Risk
Primary completion moved at least 7 months later May 31, 2027 → Dec 31, 2027
See other at-risk trials from Astellas Gene Therapies

Record History

See every version on ClinicalTrials.gov ↗

  1. Secondary endpoints: details revised at 2 of 9 entries
    • Site details revised at 5 of 11 sites
    • Eligibility criteria expanded, about 4 words
  2. Study sites: 10 to 11v12
  3. Primary and study completion pushed from May 31, 2027 to Dec 31, 2027v11
  4. Study sites: 9 to 10v10
  5. Trial sites expanded: 6 → 9 locationsv9
Show 7 earlier versionsHide 7 earlier versions
  1. Study sites: 5 to 6v8
  2. Study sites: 4 to 5v7
  3. Study sites: 3 to 4v6
  4. Primary and study completion dates: Aug 31, 2026 to May 31, 2027
    • Study sites: 1 to 3
    • Start date changed from estimate of Mar 31, 2025 to May 19, 2025
    • Eligibility criteria shortened, about 5 words
    v5
  5. Trial status: Not Yet Recruiting to Recruiting
    • Start date: Jan 31, 2025 to Mar 31, 2025
    • Study sites: 0 to 1
    v4
  6. Start date: Dec 31, 2024 to Jan 31, 2025
    • Primary and study completion dates: Apr 30, 2026 to Aug 31, 2026
    v3
  7. Start date: Sep 30, 2024 to Dec 31, 2024v2
  1. Study First Submitted
  • Stopped: an ongoing trial was terminated, suspended or withdrawn.
  • Notable: the trial’s plan changed such as a big move in its timeline or size, a new primary endpoint, sponsor or phase, or recruiting opened or resumed.
  • Minor: routine progress and housekeeping, such as sites added or enrollment closed.
XLMTM (X-linked myotubular myopathy) is a serious genetic muscle condition. It is caused by changes in the MTM1 gene which stops or slows down normal muscle development, causing severe muscle weakness. There is currently no cure for XLMTM. Ongoing care is needed to manage symptoms and prevent further medical problems from this condition. Recent research shows that individuals with XLMTM often have reduced bile flow which can affect liver and gallbladder health. Bile is a liquid made in the liver that helps digest fat. Ongoing liver health checks may help with the routine care of people with XLMTM. There is a need to understand liver problems that develop in individuals with XLMTM over time. The main aim of the study is to learn how many boys with XLMTM have new cases of liver problems during the study. This study is about collecting information only. This is known as an observational study. The individual's doctor decides on treatment, not the study sponsor (Astellas). In this study, boys under 18 diagnosed with XLMTM will be followed for about 1 year. The health of their liver and gallbladder will be checked about every 6 weeks. This can be done at home, if preferred. A scan called a Fibroscan (also known as transient elastography) will check for signs of scarring in the liver (fibrosis) and the build-up of lipids. It is suggested that each boy will have a Fibroscan when they start the study and another scan when they complete the study. This study will help understand liver, gallbladder, and bile duct issues in individuals with XLMTM over time. The goal is to improve their care and provide information to use in future clinical studies.
Trial Details
NCT Number NCT06581146
Lead Sponsor Astellas Gene Therapies
Conditions
(from sponsor)		 X-Linked Myotubular Myopathy
Related terms Myopathies, Structural, Congenital
Enrollment 50 participants
Start Date
Primary Completion (estimated)
Study Completion (estimated)
Updated on ClinicalTrials.gov