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Antisense Oligonucleotide

AOC 1001 for Myotonic Dystrophy (MARINA-OLE Trial)

Phase 2
Waitlist Available
Research Sponsored by Avidity Biosciences, Inc.
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Be older than 18 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up through study completion, up to day 729
Awards & highlights

MARINA-OLE Trial Summary

This trial is testing a possible new treatment for myotonic dystrophy type 1, which is a rare genetic disease that causes muscle weakness.

Who is the study for?
This trial is for adults with Myotonic Dystrophy Type 1 who completed the MARINA study without major issues. They must be able to continue using contraception and not be pregnant or breastfeeding. Those with new or worsening conditions that could affect their participation are excluded.Check my eligibility
What is being tested?
The trial is an extension of a previous study, testing multiple doses of AOC 1001 administered intravenously against a placebo to assess its safety, tolerability, effectiveness, and how it's processed by the body in DM1 patients.See study design
What are the potential side effects?
Specific side effects aren't listed here but generally may include reactions at the infusion site, potential allergic responses to AOC 1001, general discomforts like headaches or nausea, and any other unexpected health changes.

MARINA-OLE Trial Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~through study completion, up to day 729
This trial's timeline: 3 weeks for screening, Varies for treatment, and through study completion, up to day 729 for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Number and severity of treatment-emergent adverse events (TEAEs)
Secondary outcome measures
AOC 1001 levels in muscle tissue
Change and percentage change from baseline in DMPK mRNA knockdown
Change and percentage change from baseline in Spliceopathy
+1 more

MARINA-OLE Trial Design

2Treatment groups
Experimental Treatment
Group I: AOC 1001 (with Placebo at Day 43)Experimental Treatment2 Interventions
AOC 1001 will 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.
Group II: AOC 1001Experimental Treatment1 Intervention
AOC 1001 will 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.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Placebo
1995
Completed Phase 3
~2670
AOC 1001
2021
Completed Phase 2
~40

Find a Location

Who is running the clinical trial?

Avidity Biosciences, Inc.Lead Sponsor
5 Previous Clinical Trials
648 Total Patients Enrolled
Li Tai, MDStudy DirectorAvidity Biosciences, Inc.
1 Previous Clinical Trials
38 Total Patients Enrolled

Media Library

AOC 1001 (Antisense Oligonucleotide) Clinical Trial Eligibility Overview. Trial Name: NCT05479981 — Phase 2
Muscular Dystrophy Research Study Groups: AOC 1001, AOC 1001 (with Placebo at Day 43)
Muscular Dystrophy Clinical Trial 2023: AOC 1001 Highlights & Side Effects. Trial Name: NCT05479981 — Phase 2
AOC 1001 (Antisense Oligonucleotide) 2023 Treatment Timeline for Medical Study. Trial Name: NCT05479981 — Phase 2
Muscular Dystrophy Patient Testimony for trial: Trial Name: NCT05479981 — Phase 2

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Are there any available berths for new participants in this study?

"This particular clinical trial is no longer recruiting patients, as reflected on the most recent update to clinicaltrials.gov from October 13th, 2022. The study was first posted on August 4th, 2022. While this specific study may not be looking for candidates, there are 1,318 other trials that are actively recruiting patients at this time."

Answered by AI

Is this research only available to adults over the age of 20?

"This study includes individuals that are aged 18-66, as specified in the eligibility requirements."

Answered by AI

Is AOC 1001 generally well-tolerated by human subjects?

"AOC 1001 falls into the Phase 2 category, which means that while there is data supporting its safety, there is no indication of it being an effective treatment. Our team has given it a 2."

Answered by AI

What are the requirements for patients who want to enroll in this clinical trial?

"Patients that have oppenheim disease and are of legal age (18+) can be accepted into this trial. Currently, the 44th person is being enrolled."

Answered by AI

Is this clinical trial accessible to patients in different parts of the city?

"So far, this experiment is being conducted in 8 different places, which include University of Florida in Gainesville, Virginia Commonwealth University in Richmond and University of California Los Angeles."

Answered by AI

Who else is applying?

How old are they?
18 - 65
What site did they apply to?
University of California Los Angeles
Stanford University
University of Florida
Other
What portion of applicants met pre-screening criteria?
Met criteria
What state do they live in?
California
How many prior treatments have patients received?
0

Why did patients apply to this trial?

I hope this clinical trials address my medical condition. I’m so much interested to try medications that can help me walk better.
PatientReceived 1 prior treatment
Recent research and studies
clinicaltrials.govStudy
Extension of AOC 1001-CS1 (MARINA) Study in Adult Myotonic Dystrophy Type 1 (DM1) Patients
2022. "Extension of AOC 1001-CS1 (MARINA) Study in Adult Myotonic Dystrophy Type 1 (DM1) Patients". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT05479981.
All > Autonomic Dysfunction > Muscular Dystrophy
~15 spots leftby Jun 2025
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