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Extension of AOC 1001-CS1 (MARINA) Study in Adult Myotonic Dystrophy Type 1 (DM1) Patients
NCT05479981 · View on ClinicalTrials.gov ↗
Study 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
Conditions Studied
Interventions
- DRUG Placebo
- DRUG AOC 1001
Study Locations (8)
California
- University of California Los Angeles — Los Angeles
- Stanford University — Palo Alto
Colorado
- University of Colorado — Denver
Florida
- University of Florida — Gainesville
Kansas
- Kansas University Medical Center — Kansas City
New York
- University of Rochester Medical Center — Rochester
Ohio
- Ohio State University — Columbus
Virginia
- Virginia Commonwealth University — Richmond
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 37 participants |
| Start Date | 2022-08-04 |
| Est. Completion | 2025-09-10 |
| Phase | Phase 2 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT05479981
The ClinicalTrials.gov registry entry for NCT05479981 describes a study currently listed as completed. It is categorized as Phase 2, which is the standard way researchers label where a study sits along the investigational pathway from early safety work through later efficacy and post-marketing evaluation. The registered enrollment target is 37 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Avidity Biosciences, which has 6 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.
The record links to 10 conditions, with Nervous System Diseases appearing as the primary indexed condition, and to 2 interventions — of which Placebo is the first listed. Interventions can include drugs, devices, procedures, behavioral programs, or observational arms, and each is tracked as a separate registry field so that downstream queries can filter accurately. When a trial lists multiple interventions, it usually reflects a multi-arm design or a comparison protocol rather than a single treatment being tested in isolation. The brief summary published in the registry is the clearest source of protocol intent and should be read before drawing conclusions from any sidebar tags.
Geographic footprint matters for practical reasons: NCT05479981 reports 8 study locations spanning 7 distinct geographic areas — top geographies include California, Colorado, Florida. A larger site network tends to correlate with broader recruitment capacity, but it does not imply anything about study quality, and site-level enrollment status can diverge from the overall registry status shown above. Every data point on this page comes from the public ClinicalTrials.gov dataset and is reproduced here for reference only; it is not a medical recommendation, an endorsement of the sponsor, or an invitation to enroll. Verify current status, eligibility criteria, and contact details directly at ClinicalTrials.gov, and discuss any participation decision with your own healthcare provider.
Frequently Asked Questions
What is clinical trial NCT05479981 about?
NCT05479981 is a clinical study titled "Extension of AOC 1001-CS1 (MARINA) Study in Adult Myotonic Dystrophy Type 1 (DM1) Patients". 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
What is the current status of trial NCT05479981?
This trial is currently completed. It is a Phase 2 study. The enrollment target is 37 participants. The study started on 2022-08-04. Estimated completion is 2025-09-10.
What conditions does trial NCT05479981 study?
This clinical trial studies the following conditions: Nervous System Diseases, Neuromuscular Diseases, Muscular Dystrophies, Musculoskeletal Diseases, Myotonic Dystrophy. These conditions were identified from the trial registry and reflect the primary focus areas of the research.
What interventions are being tested in trial NCT05479981?
The interventions under investigation include: Placebo (DRUG), AOC 1001 (DRUG). Each intervention is being evaluated for safety and efficacy as part of this clinical study.
Who is sponsoring clinical trial NCT05479981?
This trial is sponsored by Avidity Biosciences, which has 6 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.
Where is trial NCT05479981 being conducted?
This trial has 8 study locations across California, Colorado, Florida, Kansas, New York. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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