Nervous System Diseases Clinical Trial
— MARINA-OLEOfficial 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
Verified date | July 2023 |
Source | Avidity Biosciences, Inc. |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Interventional |
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
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 |
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 |
Lead Sponsor | Collaborator |
---|---|
Avidity Biosciences, Inc. |
United States,
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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