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

Nervous System Diseases Clinical Trial

— MARINA-OLE  

Official title:

A Phase 2 Extension Study to Evaluate the Long-Term Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of AOC 1001 Administered Intravenously to Adult Myotonic Dystrophy Type 1 (DM1) Patients

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT05479981
• Other study ID #: AOC 1001-CS2
• Status: Active, not recruiting
• Phase: Phase 2
• Start date: August 4, 2022
• Est. completion date: June 2025

Study information

• Verified date: July 2023
• Source: Avidity Biosciences, Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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

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. The total duration of active treatment in AOC 1001-CS2 is approximately 24 months. Once participants have completed active treatment, they will be followed through a 9-month safety follow-up period. The sponsor may extend active treatment beyond 24 months at a future timepoint.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 37
• Est. completion date: June 2025
• Est. primary completion date: June 2025
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 66 Years

Eligibility

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

Related Conditions & MeSH terms

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

Intervention

Drug:
• Placebo
Placebo will be administered by intravenous (IV) infusion.
• AOC 1001
AOC 1001 will be administered by intravenous (IV) infusion.

Locations

Country	Name	City	State
United States	Ohio State University	Columbus	Ohio
United States	University of Colorado	Denver	Colorado
United States	University of Florida	Gainesville	Florida
United States	Kansas University Medical Center	Kansas City	Kansas
United States	University of California Los Angeles	Los Angeles	California
United States	Stanford University	Palo Alto	California
United States	Virginia Commonwealth University	Richmond	Virginia
United States	University of Rochester Medical Center	Rochester	New York

Sponsors (1)

Lead Sponsor	Collaborator
Avidity Biosciences, Inc.

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Number and severity of treatment-emergent adverse events (TEAEs)		Through study completion, up to Day 729
Secondary	Plasma pharmacokinetic (PK) parameters	Maximum and trough plasma concentration	Through study completion, up to Day 729
Secondary	AOC 1001 levels in muscle tissue		Through Day 183
Secondary	Change and percentage change from baseline in DMPK mRNA knockdown		Through Day 183
Secondary	Change and percentage change from baseline in Spliceopathy		Through Day 183