AOC 1001 for Atrophy, Disuse

Phase-Based Progress Estimates
University of Florida, Gainesville, FL
Atrophy, Disuse+13 More
AOC 1001 - Drug
All Sexes
What conditions do you have?

Study Summary

AOC 1001-CS2 (MARINA-OLE) is a Phase 2 extension of the AOC 1001-CS1 (MARINA) 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

Eligible Conditions

  • Atrophy, Disuse
  • Muscular Diseases
  • Myotonic Dystrophy
  • Muscular Dystrophies
  • Oppenheim Disease
  • Myotonic Disorders
  • Musculoskeletal Diseases
  • Neurodegenerative Diseases
  • DM1
  • Nervous System Diseases
  • Heredodegenerative Disorders, Nervous System
  • Hereditary Diseases

Treatment Effectiveness

Effectiveness Progress

1 of 3

Other trials for Atrophy, Disuse

Study Objectives

1 Primary · 4 Secondary · Reporting Duration: Through study completion, up to Day 729

Through Day 183
AOC 1001 levels in muscle tissue
Change and percentage change from baseline in DMPK mRNA knockdown
Change and percentage change from baseline in Spliceopathy
Day 729
Number and severity of treatment-emergent adverse events (TEAEs)
Plasma pharmacokinetic (PK) parameters

Trial Safety

Safety Progress

2 of 3
This is further along than 68% of similar trials

Other trials for Atrophy, Disuse

Trial Design

2 Treatment Groups

AOC 1001
1 of 2
AOC 1001 (with Placebo at Day 43)
1 of 2
Experimental Treatment

44 Total Participants · 2 Treatment Groups

Primary Treatment: AOC 1001 · Has Placebo Group · Phase 2

AOC 1001
Experimental Group · 1 Intervention: AOC 1001 · Intervention Types: Drug
AOC 1001 (with Placebo at Day 43)Experimental Group · 2 Interventions: Placebo, AOC 1001 · Intervention Types: Drug, Drug
First Studied
Drug Approval Stage
How many patients have taken this drug
Completed Phase 3

Trial Logistics

Trial Timeline

Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: through study completion, up to day 729
Closest Location: University of Florida · Gainesville, FL
Photo of University of Florida 1Photo of University of Florida 2Photo of University of Florida 3
2007First Recorded Clinical Trial
0 TrialsResearching Atrophy, Disuse
783 CompletedClinical Trials

Who is running the clinical trial?

Avidity Biosciences, Inc.Lead Sponsor
2 Previous Clinical Trials
494 Total Patients Enrolled
Li Tai, MDStudy DirectorAvidity Biosciences, Inc.
1 Previous Clinical Trials
44 Total Patients Enrolled

Eligibility Criteria

Age 18+ · All Participants · 2 Total Inclusion Criteria

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About The Reviewer

Michael Gill preview

Michael Gill - B. Sc.

First Published: October 9th, 2021

Last Reviewed: August 12th, 2022

Michael Gill holds a Bachelors of Science in Integrated Science and Mathematics from McMaster University. During his degree he devoted considerable time modeling the pharmacodynamics of promising drug candidates. Since then, he has leveraged this knowledge of the investigational new drug ecosystem to help his father navigate clinical trials for multiple myeloma, an experience which prompted him to co-found Power Life Sciences: a company that helps patients access randomized controlled trials.