Study of AOC 1001 in Adult Myotonic Dystrophy Type 1 (DM1) Patients (MARINA)

Avidity Biosciences

Status and phase: Completed; Phase 2; Phase 1

Conditions: Myotonic Dystrophy Type 1 (DM1); Myotonic Dystrophy 1; DM1; Myotonic Disorders; Myotonic Muscular Dystrophy; Dystrophy Myotonic; Steinert Disease; Myotonic Dystrophy

Treatments: Drug: Placebo; Drug: AOC 1001

Study type: Interventional

Funder types: Industry

Identifiers

NCT05027269

AOC 1001-CS1

Details and patient eligibility

About

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.

Enrollment: 38 patients

Sex: All

Ages: 18 to 65 years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion 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/Exclusion criteria may apply.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Sequential Assignment

Masking: Quadruple Blind

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38 participants in 4 patient groups, including a placebo group

Part A Single Dose: AOC 1001 Dose Level 1

Experimental group

Description:

AOC 1001 will be administered once.

Treatment:

Drug: AOC 1001

Part A Single Dose: Placebo

Placebo Comparator group

Description:

Saline will be administered once.

Treatment:

Drug: Placebo

Part B Multiple Ascending Dose: AOC 1001 Dose Levels 2 & 3

Experimental group

Description:

AOC 1001 will be administered three times.

Treatment:

Drug: AOC 1001

Part B Multiple Ascending Dose: Placebo

Placebo Comparator group

Description:

Saline will be administered three times.

Treatment:

Drug: Placebo