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MARINA: AOC 1001 in Adult DM1 Patients
NCT05027269Phase 2
Key facts
- Status
- Completed
- Start
- Oct 2021
- Primary completion
- Feb 2023
- Countries
- 1
- Sponsor
- Avidity Biosciences
- Conditions
- myotonic dystrophy type 1 (DM1)
01
Overview
Phase 2, randomised, double-blind trial of delpacibart etedesiran in myotonic dystrophy type 1 (DM1) (4 arms), sponsored by Avidity Biosciences. Primary endpoint: Frequency of Treatment Emergent Adverse Events at Day 183. Started Oct 2021; status: completed.
02
Design
4 arms- Study design
- RandomizedDouble-blind
Part A Single Dose: AOC 1001 Dose Level 1
AOC 1001 will be administered once.
Part A Single Dose: Placebo
Saline will be administered once.
Part B Multiple Ascending Dose: AOC 1001 Dose Levels 2 & 3
AOC 1001 will be administered three times.
Part B Multiple Ascending Dose: Placebo
Saline will be administered three times.
03
Who it enrols
Adults with a genetic diagnosis of DM1 (CTG repeat >= 100), clinical signs of DM1, and ability to walk independently. Excludes patients with uncontrolled diabetes, BMI > 35, uncontrolled hypertension, congenital DM1, or recent anti-myotonic medication.
04
Endpoints
Primary
Frequency of Treatment Emergent Adverse Events
Secondary
Plasma Pharmacokinetic Parameters
Urine Pharmacokinetic Parameters
Change From Baseline in DMPK mRNA Knockdown
Absolute Change From Baseline in 4 Gene Spliceopathy
AOC 1001 Concentration Levels in Plasma, Urine, and Muscle Tissue
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