Extension of AOC 1001-CS1 (MARINA) Study in Adult Myotonic Dystrophy Type 1 (DM1) Patients
Key facts
- Who runs it
- Avidity Biosciences, Inc.
- Where
- Columbus, Denver, Gainesville, Kansas City, Los Angeles, Palo Alto, Richmond, and Rochester, United StatesSee the 8 sites
- Trial code
- Status
- Completed
- Phase
- Phase 2
- People taking part
- 37 people enrolled
- Started
- 4 Aug 2022
- Ended
- 10 Sept 2025
- Type of study
- Interventional, 2 groups
- Treatments tested
- Placebo
- AOC 1001
- Record updated
- 11 September 2026
Who can take part
- Age
- 18 years to 66 years
- Sex
- Women and men
- Healthy volunteers
- Cannot take part
The registry criteria, word for word
Show all 4 criteriaShow less
Who can take part
- Completion of AOC 1001-CS1 (MARINA) study with satisfactory compliance and no significant tolerability issues
Who cannot take part
- Any new conditions or worsening of existing conditions that in the opinion of the investigator or sponsor would make the participant unsuitable for the study or could interfere with participation or completion of the study
- Pregnancy, intent to become pregnant, or active breastfeeding
- Unwilling or unable to continue to comply with contraceptive requirements
Where in United States
- Ohio State UniversityColumbus, United States
- University of ColoradoDenver, United States
- University of FloridaGainesville, United States
- Kansas University Medical CenterKansas City, United States
- University of California, Los AngelesLos Angeles, United States
- Stanford UniversityPalo Alto, United States
- Virginia Commonwealth UniversityRichmond, United States
- University of Rochester Medical CenterRochester, United States
About the trial
What the trial wants to find out
AOC 1001-CS2 (MARINA-OLE) (NCT05479981) is a Phase 2 extension of the AOC 1001-CS1 (MARINA) (NCT05027269) 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
The 2 groups in the trial
- AOC 1001Treatment being tested
AOC 1001 will initially be administered quarterly. On Day 43 patients will receive an additional dose. Treatment assignment will be based on treatment received in AOC 1001-CS1. If participant did not receive AOC 1001 on Day 43 in AOC 1001-CS1, participant will receive AOC 1001 treatment on Day 43 in AOC 1001-CS2. Beginning in September 2024, AOC 1001 will be administered every 8 weeks.
- AOC 1001 (with Placebo at Day 43)Treatment being tested
AOC 1001 will initially be administered quarterly. On Day 43 patients will receive an additional dose. Treatment assignment will be based on treatment received in AOC 1001-CS1. If participant received AOC 1001 on Day 43 in AOC 1001-CS1, participant will receive blinded placebo treatment on Day 43 in AOC 1001-CS2. Beginning in September 2024, AOC 1001 will be administered every 8 weeks.
Treatment: Placebo and AOC 1001
Data and sources
- Registry record
- Listed on ClinicalTrials.gov.
- Official title
- A Phase 2 Extension Study to Evaluate the Long-Term Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of AOC 1001 Administered Intravenously to Adult Myotonic Dystrophy Type 1 (DM1) Patients
- Record updated
- 11 September 2026