Extension of AOC 1001-CS1 (MARINA) Study in Adult Myotonic Dystrophy Type 1 (DM1) Patients (MARINA-OLE)

Avidity Biosciences

Status and phase: Completed; Phase 2

Conditions: Muscular Diseases; Neuromuscular Diseases; Neurodegenerative Diseases; Genetic Diseases, Inborn; DM1; Musculoskeletal Diseases; Myotonic Disorders; Nervous System Diseases; Heredodegenerative Disorders, Nervous System; Myotonic Dystrophy; Myotonic Dystrophy 1; Muscular Disorders, Atrophic; Muscular Dystrophies

Treatments: Drug: Placebo; Drug: AOC 1001

Study type: Interventional

Funder types: Industry

Identifiers

NCT05479981

AOC 1001-CS2

Details and patient eligibility

About

AOC 1001-CS2 (MARINA-OLE) is a Phase 2 extension of the AOC 1001-CS1 (MARINA) study to evaluate the safety, tolerability, efficacy, pharmacokinetics and pharmacodynamics of multiple-doses of AOC 1001 Administered Intravenously to Adult Myotonic Dystrophy Type 1 (DM1) patients

Full description

This study will continue to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics, and efficacy of AOC 1001 in participants that enrolled in the randomized, placebo-controlled, First-In-Human Phase 1/2 AOC 1001-CS1 (MARINA) clinical study.

Participants from AOC 1001-CS1 are eligible to enroll in AOC 1001-CS2 if they have satisfactorily completed AOC 1001-CS1.

After completion of Q8W Visit 6 assessments, participants will have the option to enroll in another AOC 1001 open-label extension (OLE) study. Participants who do not enroll in an AOC 1001 OLE study will be monitored for safety for a total of 8 weeks from the last dose of study drug. Total duration on study is up to 37 months (3 years 1 month).

As of September 2024, the dosing regimen was updated to every 8 weeks.

Enrollment: 37 patients

Sex: All

Ages: 18 to 66 years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

Key Inclusion Criteria:

• Completion of AOC 1001-CS1 (MARINA) study with satisfactory compliance and no significant tolerability issues

Key Exclusion Criteria:

• Pregnancy, intent to become pregnant, or active breastfeeding
• Unwilling or unable to continue to comply with contraceptive requirements
• Any new conditions or worsening of existing conditions that in the opinion of the investigator or sponsor would make the participant unsuitable for the study or could interfere with participation or completion of the study

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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37 participants in 2 patient groups

AOC 1001

Experimental group

Description:

AOC 1001 will initially be administered quarterly. On Day 43 patients will receive an additional dose. Treatment assignment will be based on treatment received in AOC 1001-CS1. If participant did not receive AOC 1001 on Day 43 in AOC 1001-CS1, participant will receive AOC 1001 treatment on Day 43 in AOC 1001-CS2. Beginning in September 2024, AOC 1001 will be administered every 8 weeks.

Treatment:

Drug: AOC 1001

AOC 1001 (with Placebo at Day 43)

Experimental group

Description:

AOC 1001 will initially be administered quarterly. On Day 43 patients will receive an additional dose. Treatment assignment will be based on treatment received in AOC 1001-CS1. If participant received AOC 1001 on Day 43 in AOC 1001-CS1, participant will receive blinded placebo treatment on Day 43 in AOC 1001-CS2. Beginning in September 2024, AOC 1001 will be administered every 8 weeks.

Treatment:

Drug: AOC 1001

Drug: Placebo