AOC 1001 for Atrophy, Disuse
Phase-Based Progress Estimates
Effectiveness
Safety
University of Florida, Gainesville, FL
Atrophy, Disuse+13 More
AOC 1001 - DrugEligibility
18+
All Sexes
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Eligibility
18+
All Sexes
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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
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
This is further along than 68% of similar trials
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
Drug
Experimental Group · 1 Intervention: AOC 1001 · Intervention Types: DrugAOC 1001 (with Placebo at Day 43)Experimental Group · 2 Interventions: Placebo, AOC 1001 · Intervention Types: Drug, Drug
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Placebo
1995
Completed Phase 3
~2670
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
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
Mark “yes” if the following statements are true for you:About The Reviewer
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.
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