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COMPLETED Phase 1

Study of AOC 1044 in Healthy Adult Volunteers and Participants With Duchenne Muscular Dystrophy (DMD) Mutations Amenable to Exon 44 Skipping

NCT05670730 · View on ClinicalTrials.gov ↗

Study Summary

AOC 1044-CS1 (EXPLORE44) is a Phase 1/2 study to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamic effects of single and multiple ascending doses of AOC 1044 in healthy adult volunteers and participants with DMD mutations amenable to exon 44 skipping. Part A is a single dose design with multiple cohorts (dose levels) in healthy adult volunteers. Part B is a multiple-ascending dose design with 3 cohorts (dose levels) in participants with Duchenne.

Interventions

  • DRUG Placebo
  • DRUG AOC 1044

Study Locations (10)

California

  • UCSD — La Jolla
  • Stanford University — Palo Alto

Arkansas

  • Arkansas Children's — Little Rock

Georgia

  • Rare Disease Research - Atlanta — Atlanta

Iowa

  • University of Iowa — Iowa City

Massachusetts

  • University of Massachusetts Chan Medical School — Worcester

Minnesota

  • Gillette Children's — Saint Paul

North Carolina

  • Rare Disease Research NC — Hillsborough

Ohio

  • Abigail Research Institute at Nationwide Children's Hospital — Columbus

Trial Details

FieldValue
Enrollment Target 70 participants
Start Date 2022-11-09
Est. Completion 2024-11-25
Phase Phase 1

Sponsor

Avidity Biosciences

6 total trials

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Full Details on ClinicalTrials.gov ↗

What the Registry Record Tells You About NCT05670730

The ClinicalTrials.gov registry entry for NCT05670730 describes a study currently listed as completed. It is categorized as Phase 1, 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 70 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 2 conditions, with Duchenne Muscular Dystrophy 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: NCT05670730 reports 10 study locations spanning 9 distinct geographic areas — top geographies include California, Arkansas, Georgia. 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 NCT05670730 about?

NCT05670730 is a clinical study titled "Study of AOC 1044 in Healthy Adult Volunteers and Participants With Duchenne Muscular Dystrophy (DMD) Mutations Amenable to Exon 44 Skipping". AOC 1044-CS1 (EXPLORE44) is a Phase 1/2 study to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamic effects of single and multiple ascending doses of AOC 1044 in healthy adult volunteers and participants with DMD mutations amenable to exon 44 skipping. Part A is a single dose ...

What is the current status of trial NCT05670730?

This trial is currently completed. It is a Phase 1 study. The enrollment target is 70 participants. The study started on 2022-11-09. Estimated completion is 2024-11-25.

What conditions does trial NCT05670730 study?

This clinical trial studies the following conditions: Duchenne Muscular Dystrophy, Exon 44. These conditions were identified from the trial registry and reflect the primary focus areas of the research.

What interventions are being tested in trial NCT05670730?

The interventions under investigation include: Placebo (DRUG), AOC 1044 (DRUG). Each intervention is being evaluated for safety and efficacy as part of this clinical study.

Who is sponsoring clinical trial NCT05670730?

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 NCT05670730 being conducted?

This trial has 10 study locations across Arkansas, California, Georgia, Iowa, Massachusetts. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.

Related

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