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

Phase 1

Completed

• Conditions: Dystrophy Myotonic; Myotonic Disorders; DM1; Steinert Disease; Myotonic Dystrophy 1; Myotonic Muscular Dystrophy; Myotonic Dystrophy Type 1 (DM1); Myotonic Dystrophy
• Interventions: Drug: Placebo; Drug: AOC 1001

• Registration Number: NCT05027269
• Lead Sponsor: Avidity Biosciences, Inc.

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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2021-08-24
• Study First Submitted QC: 2021-08-24
• Study First Posted: 2021-08-30
• Study Start: 2021-10-28
• Primary Completion: 2023-02-14
• Study Completion: 2023-02-14
• Status Verified: 2024-02
• Disposition First Submitted: 2024-02-01
• Last Update Submitted: 2024-02-01
• Disposition First Posted: 2024-02-05
• Last Update Posted: 2024-02-05

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 38

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

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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 & Design

• Study Type: INTERVENTIONAL
• Study Design: SEQUENTIAL

Arm && Interventions

Group	Intervention	Description
Part B Multiple Ascending Dose: Placebo	Placebo	Saline will be administered three times.
Part A Single Dose: Placebo	Placebo	Saline will be administered once.
Part B Multiple Ascending Dose: AOC 1001 Dose Levels 2 & 3	AOC 1001	AOC 1001 will be administered three times.
Part A Single Dose: AOC 1001 Dose Level 1	AOC 1001	AOC 1001 will be administered once.

Primary Outcome Measures

Name	Time	Method
Frequency of treatment emergent adverse events (TEAEs)	Through study completion, up to Day 183

Secondary Outcome Measures

Name	Time	Method
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
Plasma pharmacokinetic (PK) parameters	Through study completion, up to Day 183	Area Under the Concentration-time Curve (AUC)
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

Trial Locations

Locations (8)

University of California Los Angeles; 🇺🇸 Los Angeles, California, United States

Stanford University; 🇺🇸 Palo Alto, California, United States

Kansas University Medical Center; 🇺🇸 Kansas City, Kansas, United States

University of Rochester Medical Center; 🇺🇸 Rochester, New York, United States

University of Colorado; 🇺🇸 Denver, Colorado, United States

University of Florida; 🇺🇸 Gainesville, Florida, United States

Ohio State University; 🇺🇸 Columbus, Ohio, United States

Virginia Commonwealth University; 🇺🇸 Richmond, Virginia, United States