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NCT05479981 · ClinicalTrials.gov registry record · Phase 2

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

A Phase 2 study of Nervous System Diseases and Neuromuscular Diseases, sponsored by Avidity Biosciences.

Completed
Registry status
Phase 2
Development phase
37
Enrollment target
8
Study locations

NCT05479981 is a Phase 2 study of Nervous System Diseases and Neuromuscular Diseases that has completed, run by Avidity Biosciences. The registered enrollment target is 37 participants, below the 322-participant average among 23 other Nervous System Diseases trials with a reported enrollment target (89% lower). The trial reports 8 study locations across 7 states.

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The verdict

NCT05479981, a Phase 2 study of Nervous System Diseases and Neuromuscular Diseases, has completed, sponsored by Avidity Biosciences.

COMPLETED
Registry status
Phase 2
Development phase
37 participants
Enrollment target
8
Study locations

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

Nervous System Diseases Neuromuscular Diseases Muscular Dystrophies Musculoskeletal Diseases Myotonic Dystrophy Muscular Diseases Myotonic Dystrophy 1 DM1 Muscular Disorders, Atrophic Myotonic Disorders

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

FieldValue
Enrollment Target 37 participants
Start Date 2022-08-04
Est. Completion 2025-09-10
Phase Phase 2

Sponsor

Avidity Biosciences

8 total trials

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What the Registry Record Tells You About NCT05479981

The ClinicalTrials.gov registry entry for NCT05479981 describes a study currently listed as completed, categorized as Phase 2. The registered enrollment target is 37 participants, a figure that helps gauge the scale of data the investigators plan to collect, below the 322-participant average among 23 other Nervous System Diseases trials with a reported enrollment target (89% lower). The listed sponsor is Avidity Biosciences, which has 8 total studies on file at ClinicalTrials.gov.

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.

NCT05479981 reports 8 study locations spanning 7 distinct geographic areas - top geographies include California, Colorado, Florida.

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.

What interventions are being tested in trial NCT05479981?

The interventions under investigation include: Placebo (DRUG), AOC 1001 (DRUG).

Who is sponsoring clinical trial NCT05479981?

This trial is sponsored by Avidity Biosciences, which has 8 total clinical trials registered on ClinicalTrials.gov.

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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Data sourced from official public datasets. See our methodology for details. Retrieved and formatted by PlainTrial Editorial

Every figure on PlainTrial is rendered directly from the ClinicalTrials.gov registry, no number is typed in by an editor. This page mirrors this trial's own ClinicalTrials.gov registry record, live from the dataset. See our editorial standards & corrections policy, the methodology behind these numbers, or report a data error.