A Phase 3 Global Study to Evaluate AOC 1001 for the Treatment of Myotonic Dystrophy Type 1
- Conditions
- Myotonic Dystrophy Type 1MedDRA version: 27.0Level: LLTClassification code: 10090453Term: Myotonic dystrophy type 1 Class: 100000004850Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
- Registration Number
- CTIS2024-511378-60-00
- Lead Sponsor
- Avidity Biosciences Inc.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 113
1. Age: 16 to 65., 2. Clinical and genetic diagnosis (CTG repeat length = 100) of DM1., 3. Ability to walk independently (orthoses and ankle braces allowed) for at least 10 meters at screening.
1. Breastfeeding, pregnancy, or intent to become pregnant during the study., 2. Unwilling or unable to comply with contraceptive requirements., 3. Abnormal lab values, conditions or diseases that would make the participant unsuitable for the study., 4. Diabetes that is not adequately controlled., 5. History of decompensated heart failure within 3 months of screening. Participants with preexisting pacemaker/ICD are not excluded., 6. Body Mass Index > 35 kg/m2 at Screening., 7. Recently treated with an investigational drug or biological agent., 8. Treatment with anti-myotonic medication within 5 half-lives or 14 days of baseline, whichever is longer, prior to baseline., 9. Note: Additional protocol defined inclusion and exclusion criteria apply.
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method