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AOC 1001 for Paramyotonia Congenita (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

Summary

This trial tests a new drug called AOC 1001 in adults with Myotonic Dystrophy Type 1. The drug is given through an IV, and researchers will check if it is safe and how it affects the body over time.

Eligible Conditions
  • Myotonic Dystrophy
  • Paramyotonia Congenita
  • Muscular Dystrophy
  • Type 1 Diabetes
  • Myotonic Disorders

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

Study objectives can provide a clearer picture of what you can expect from a treatment.
Secondary study objectives
Plasma pharmacokinetic (PK) parameters
Urine pharmacokinetic (PK) parameters

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
7 Previous Clinical Trials
908 Total Patients Enrolled
Li Tai, MDStudy DirectorAvidity Biosciences, Inc.
1 Previous Clinical Trials
37 Total Patients Enrolled
~9 spots leftby Nov 2025
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