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AOC 1001 for Myotonic Dystrophy (MARINA Trial)

Phase 1 & 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 183
Awards & highlights

MARINA Trial Summary

This trial is testing a new drug for safety and efficacy in patients with myotonic dystrophy type 1. The trial has two parts, A and B. Part A is testing a single dose of the drug in one group of patients, while Part B is testing multiple doses of the drug in three different groups of patients. The trial will last for six months for each patient, with follow-up periods after treatment.

Eligible Conditions
  • Myotonic Dystrophy
  • Myotonic Disorders
  • Myotonic Muscular Dystrophy

MARINA Trial Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~through study completion, up to day 183
This trial's timeline: 3 weeks for screening, Varies for treatment, and through study completion, up to day 183 for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Frequency 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
+2 more

MARINA Trial Design

4Treatment groups
Experimental Treatment
Placebo Group
Group I: Part B Multiple Ascending Dose: AOC 1001 Dose Levels 2 & 3Experimental Treatment1 Intervention
AOC 1001 will be administered three times.
Group II: Part A Single Dose: AOC 1001 Dose Level 1Experimental Treatment1 Intervention
AOC 1001 will be administered once.
Group III: Part B Multiple Ascending Dose: PlaceboPlacebo Group1 Intervention
Saline will be administered three times.
Group IV: Part A Single Dose: PlaceboPlacebo Group1 Intervention
Saline will be administered once.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
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
647 Total Patients Enrolled
1 Trials studying Myotonic Dystrophy
37 Patients Enrolled for Myotonic Dystrophy
Li Tai, MDStudy DirectorAvidity Biosciences, Inc.
1 Previous Clinical Trials
37 Total Patients Enrolled
1 Trials studying Myotonic Dystrophy
37 Patients Enrolled for Myotonic Dystrophy

Frequently Asked Questions

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

Does this investigation include geriatric candidates?

"This study is recruiting individuals between 18 and 65 years old."

Answered by AI

Are there still spots available for this experiment?

"According to the data provided on clinicaltrials.gov, this medical trial is no longer actively seeking patients - it was first posted in October 28th 2021 and last edited on October 13 2022. However, there are plenty of alternative trials currently recruiting participants across the United States."

Answered by AI

Who meets the criteria for participation in this clinical trial?

"The study requires 44 adults, aged between 18 and 65 years old, who have been diagnosed with myotonic dystrophy. Additionally, all patients must demonstrate the ability to walk 10 meters unassisted (with agreed-upon braces or orthotics) during screening."

Answered by AI

How many healthcare facilities are currently participating in this research project?

"This clinical trial is being run from Stanford University in Palo Alto, California, the University of Colorado in Denver, and the University of Florida located in Gainesville as well as 8 other medical centres."

Answered by AI

Who else is applying?

How old are they?
18 - 65
What site did they apply to?
Stanford University
What portion of applicants met pre-screening criteria?
Met criteria

How responsive is this trial?

Average response time
  • < 2 Days
Most responsive sites:
  1. Stanford University: < 48 hours
Typically responds via
Email
Recent research and studies
clinicaltrials.govStudy
Study of AOC 1001 in Adult Myotonic Dystrophy Type 1 (DM1) Patients
2021. "Study of AOC 1001 in Adult Myotonic Dystrophy Type 1 (DM1) Patients". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT05027269.
~11 spots leftby Apr 2025
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