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Clinical Trial Details — Status: Active, not recruiting

Administrative data

NCT number NCT05479981
Other study ID # AOC 1001-CS2
Secondary ID
Status Active, not recruiting
Phase Phase 2
First received
Last updated
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
Health authority
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

Study Design


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
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