DM1, Dystrophy Myotonic, Myotonic Disorders, Myotonic Dystrophy, Myotonic Dystrophy 1, Myotonic Dystrophy Type 1 (DM1), Myotonic Muscular Dystrophy, Steinert Disease
Conditions
Keywords
DM1, Myotonic Dystrophy 1, Myotonic Dystrophy Type 1 (DM1), Myotonic Dystrophy, DM, Dystrophy Myotonic, Myotonic Disorders, Steinert Disease, MARINA, Avidity Biosciences, Avidity, AOC 1001, Myotonic Muscular Dystrophy
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 2 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.
Interventions
DRUGAOC 1001
AOC 1001 will be administered by intravenous (IV) infusion.
DRUGPlacebo
Placebo will be administered by intravenous (IV) infusion.
Sponsors
Avidity Biosciences, Inc.
Study design
Allocation
RANDOMIZED
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
18 Years to 65 Years
Healthy volunteers
No
Inclusion criteria
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/
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Frequency of treatment emergent adverse events (TEAEs) | Through study completion, up to Day 183 |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Plasma pharmacokinetic (PK) parameters | Through study completion, up to Day 183 | Maximum plasma concentration (Cmax) |
| Urine pharmacokinetic (PK) parameters | Through study completion, up to Day 183 | fraction excreted (fe) in urine |
| AOC 1001 levels in muscle tissue | Through study completion, up to Day 183 | — |
| Change and percentage change from baseline in DMPK mRNA knockdown | Through study completion, up to Day 183 | — |
| Change and percentage change from baseline in Spliceopathy | Through study completion, up to Day 183 | — |
Countries
United States
Contacts
STUDY_DIRECTORLi Tai, MD
Avidity Biosciences, Inc.
Outcome results
None listed