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NCT05027269 · ClinicalTrials.gov registry record · Phase 1
Study of AOC 1001 in Adult Myotonic Dystrophy Type 1 (DM1) Patients
A Phase 1 study, sponsored by Avidity Biosciences.
- Completed
- Registry status
- Phase 1
- Development phase
- 39
- Enrollment target
NCT05027269: Completed Phase 1 study, sponsored by Avidity Biosciences.
NCT05027269 is a Phase 1 study that has completed, run by Avidity Biosciences. The registered enrollment target is 39 participants, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (35% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT05027269, a Phase 1 study, has completed, sponsored by Avidity Biosciences.
- COMPLETED
- Registry status
- Phase 1
- Development phase
- 39 participants
- Enrollment target
Study Summary
AOC 1001-CS1 is a randomized, double-blind, placebo-controlled, Phase 1/2 study to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of single and multiple-doses of AOC 1001 Administered Intravenously to Adult Myotonic Dystrophy Type 1 (DM1) patients (MARINA). Part A is a single dose design with 1 cohort (dose level). In Part A, the patient duration is 6 months as the treatment period is 1 day followed by a 6 month follow-up period. Part B is a multiple-ascending dose design with 2 cohorts (dose levels). In Part B, the patient duration is 6 months as the treatment period is 3 months followed by a 3 month follow-up period.
Interventions
- DRUG Placebo
- DRUG AOC 1001
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 39 participants |
| Start Date | 2021-10-28 |
| Est. Completion | 2023-02-14 |
| Phase | Phase 1 |
What the finished NCT05027269 record still lists
NCT05027269 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 39 participants, a relatively small participant target, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (35% lower).
The record links to 0 conditions, and to 2 interventions - of which Placebo is the first listed.
NCT05027269 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT05027269 about?
NCT05027269 is a clinical study titled "Study of AOC 1001 in Adult Myotonic Dystrophy Type 1 (DM1) Patients". AOC 1001-CS1 is a randomized, double-blind, placebo-controlled, Phase 1/2 study to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of single and multiple-doses of AOC 1001 Administered Intravenously to Adult Myotonic Dystrophy Type 1 (DM1) patients (MARINA). Part A is a sin...
What is the current status of trial NCT05027269?
This trial is currently completed. It is a Phase 1 study. The enrollment target is 39 participants. The study started on 2021-10-28. Estimated completion is 2023-02-14.
What interventions are being tested in trial NCT05027269?
The interventions under investigation include: Placebo (DRUG), AOC 1001 (DRUG).
Who is sponsoring clinical trial NCT05027269?
This trial is sponsored by Avidity Biosciences, which has 8 total clinical trials registered on ClinicalTrials.gov.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT05027269's enrollment target sits among peer trials
39 1191st of 2000 higher than 804 of 2,000 other Phase 1 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 1 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.
Source ClinicalTrials.gov registry export · 2026-08-08
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