Open
Actively Recruiting
Study of AOC 1001 in Adult Myotonic Dystrophy Type 1 (DM1) Patients
About
Brief Summary
AOC 1001-CS1 is a randomized, double-blind, placebo-controlled, Phase 1/2 study to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of single and multiple-doses of AOC 1001 Administered Intravenously to Adult Myotonic Dystrophy Type 1 (DM1) patients (MARINA).
Part A is a single dose design with 1 cohort (dose level). In Part A, the patient duration is 6 months as the treatment period is 1 day followed by a 6 month follow-up period. Part B is a multiple-ascending dose design with 3 cohorts (dose levels). In Part B, the patient duration is 6 months as the treatment period is 3 months followed by a 3 month follow-up period.
Primary Purpose
Study Type
Phase
Eligibility
Gender
Healthy Volunteers
Minimum Age
Maximum Age
Key Inclusion Criteria:
- Genetic diagnosis of DM1 (CTG repeat length ≥ 100)
- Clinician assessed signs of DM1
- Ability to walk independently (orthoses and ankle braces allowed) for at least 10 meters at screening
Key Exclusion Criteria:
- Diabetes that is not adequately controlled
- BMI > 35 kg/m2
- Uncontrolled hypertension
- Congenital DM1
- History of tibialis anterior (TA) biopsy within 3 months of Day 1 or planning to undergo TA biopsies during study period
- Recently treated with an investigational drug
- Treatment with anti-myotonic medication within 14 days of Day 1
Note: Other protocol defined Inclusion/Exclusion criteria may apply.
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Study Stats
Protocol No.
21-001658
Category
Neurology
Urology
Principal Investigator
Contact
Location
- UCLA Westwood