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NCT05670730 · ClinicalTrials.gov registry record · Phase 1
Study of AOC 1044 in Healthy Adult Volunteers and Participants With Duchenne Muscular Dystrophy (DMD) Mutations Amenable to Exon 44 Skipping
A Phase 1 study of Duchenne Muscular Dystrophy and Exon 44, sponsored by Avidity Biosciences.
- Completed
- Registry status
- Phase 1
- Development phase
- 70
- Enrollment target
- 10
- Study locations
NCT05670730: Completed Phase 1 study of Duchenne Muscular Dystrophy and Exon 44, sponsored by Avidity Biosciences.
NCT05670730 is a Phase 1 study of Duchenne Muscular Dystrophy and Exon 44 that has completed, run by Avidity Biosciences. The registered enrollment target is 70 participants, below the 411-participant average among 48 other Duchenne Muscular Dystrophy trials with a reported enrollment target (83% lower). The trial reports 10 study locations across 9 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT05670730, a Phase 1 study of Duchenne Muscular Dystrophy and Exon 44, has completed, sponsored by Avidity Biosciences.
- COMPLETED
- Registry status
- Phase 1
- Development phase
- 70 participants
- Enrollment target
- 10
- Study locations
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.
Conditions Studied
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
| Field | Value |
|---|---|
| Enrollment Target | 70 participants |
| Start Date | 2022-11-09 |
| Est. Completion | 2024-11-25 |
| Phase | Phase 1 |
What the finished NCT05670730 record still lists
NCT05670730 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 70 participants, a relatively small participant target, below the 411-participant average among 48 other Duchenne Muscular Dystrophy trials with a reported enrollment target (83% lower).
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.
NCT05670730 lists 10 locations in 9 states (California, Arkansas, Georgia).
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.
What interventions are being tested in trial NCT05670730?
The interventions under investigation include: Placebo (DRUG), AOC 1044 (DRUG).
Who is sponsoring clinical trial NCT05670730?
This trial is sponsored by Avidity Biosciences, which has 8 total clinical trials registered on ClinicalTrials.gov.
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.
Learn More About Clinical Trials
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Where NCT05670730's enrollment target sits among peer trials
70 19th of 48 higher than 30 of 48 other Duchenne Muscular Dystrophy trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Duchenne Muscular Dystrophy trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
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