Study of AOC 1001 in Adult Myotonic Dystrophy Type 1 (DM1) Patients
Public ClinicalTrials.gov record NCT05027269. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official title
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
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
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.
Study identification
- NCT ID
- NCT05027269
- Recruitment status
- Completed
- Study type
- Interventional
- Phase
- Phase 1, Phase 2
- Enrollment
- 39 participants
Conditions and interventions
Conditions
Eligibility (public fields only)
- Age range
- 18 Years to 65 Years
- Sex
- All
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Oct 28, 2021 (Actual)
- Primary completion
- Feb 14, 2023 (Actual)
- Completion
- Feb 14, 2023 (Actual)
- Last update posted
- Sep 28, 2026
2021 – 2023
United States locations
- U.S. sites
- 8
- U.S. states
- 7
- U.S. cities
- 8
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| University of California Los Angeles | Los Angeles | California | 90095 | — |
| Stanford University | Palo Alto | California | 94304 | — |
| University of Colorado | Denver | Colorado | 80045 | — |
| University of Florida | Gainesville | Florida | 32608 | — |
| Kansas University Medical Center | Kansas City | Kansas | 66205 | — |
| University of Rochester Medical Center | Rochester | New York | 14642 | — |
| Ohio State University | Columbus | Ohio | 43221 | — |
| Virginia Commonwealth University | Richmond | Virginia | 23298 | — |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT05027269, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Sep 28, 2026 · Synced Oct 22, 2026
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Open the official record
The complete public registry record, listed eligibility criteria, and available contact information for NCT05027269 live on ClinicalTrials.gov.