Study of AOC 1001 in Adult Myotonic Dystrophy Type 1 (DM1) Patients

Myotonic Dystrophy 1 Clinical Trial

— MARINA  

Official title:

A Randomized, Double-Blind, Placebo-Controlled, Phase 1/2 Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Single and Multiple-Doses of AOC 1001 Administered Intravenously to Adult Myotonic Dystrophy Type 1 (DM1) Patients

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT05027269
• Other study ID #: AOC 1001-CS1
• Status: Completed
• Phase: Phase 1/Phase 2
• Start date: October 28, 2021
• Est. completion date: February 14, 2023

Study information

• Verified date: February 2024
• Source: Avidity Biosciences, Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

AOC 1001-CS1 is a randomized, double-blind, placebo-controlled, Phase 1/2 study to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of single and multiple-doses of AOC 1001 Administered Intravenously to Adult Myotonic Dystrophy Type 1 (DM1) patients (MARINA). Part A is a single dose design with 1 cohort (dose level). In Part A, the patient duration is 6 months as the treatment period is 1 day followed by a 6 month follow-up period. Part B is a multiple-ascending dose design with 2 cohorts (dose levels). In Part B, the patient duration is 6 months as the treatment period is 3 months followed by a 3 month follow-up period.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 38
• Est. completion date: February 14, 2023
• Est. primary completion date: February 14, 2023
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 65 Years

Eligibility

Key Inclusion Criteria:

• Genetic diagnosis of DM1 (CTG repeat length = 100)
• Clinician assessed signs of DM1
• Ability to walk independently (orthoses and ankle braces allowed) for at least 10 meters at screening

Key Exclusion Criteria:

• Diabetes that is not adequately controlled
• BMI > 35 kg/m2
• Uncontrolled hypertension
• Congenital DM1
• History of tibialis anterior (TA) biopsy within 3 months of Day 1 or planning to undergo TA biopsies during study period
• Recently treated with an investigational drug
• Treatment with anti-myotonic medication within 14 days of Day 1 Note: Other protocol defined Inclusion/Exclusion criteria may apply.

Related Conditions & MeSH terms

• DM1
• Muscular Dystrophies
• Myotonic Disorders
• Myotonic Dystrophy
• Myotonic Dystrophy 1
• Myotonic Dystrophy Type 1 (DM1)
• Myotonic Muscular Dystrophy
• Steinert Disease

Intervention

Drug:
• AOC 1001
AOC 1001 will be administered by intravenous (IV) infusion.
• Placebo
Placebo will be administered by intravenous (IV) infusion.

Locations

Country	Name	City	State
United States	Ohio State University	Columbus	Ohio
United States	University of Colorado	Denver	Colorado
United States	University of Florida	Gainesville	Florida
United States	Kansas University Medical Center	Kansas City	Kansas
United States	University of California Los Angeles	Los Angeles	California
United States	Stanford University	Palo Alto	California
United States	Virginia Commonwealth University	Richmond	Virginia
United States	University of Rochester Medical Center	Rochester	New York

Sponsors (1)

Lead Sponsor	Collaborator
Avidity Biosciences, Inc.

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Frequency of treatment emergent adverse events (TEAEs)		Through study completion, up to Day 183
Secondary	Plasma pharmacokinetic (PK) parameters	Maximum plasma concentration (Cmax)	Through study completion, up to Day 183
Secondary	Plasma pharmacokinetic (PK) parameters	Time to maximum plasma concentration (Tmax)	Through study completion, up to Day 183
Secondary	Plasma pharmacokinetic (PK) parameters	Terminal Half-life (t1/2)	Through study completion, up to Day 183
Secondary	Plasma pharmacokinetic (PK) parameters	Area Under the Concentration-time Curve (AUC)	Through study completion, up to Day 183
Secondary	Urine pharmacokinetic (PK) parameters	fraction excreted (fe) in urine	Through study completion, up to Day 183
Secondary	AOC 1001 levels in muscle tissue		Through study completion, up to Day 183
Secondary	Change and percentage change from baseline in DMPK mRNA knockdown		Through study completion, up to Day 183
Secondary	Change and percentage change from baseline in Spliceopathy		Through study completion, up to Day 183

External Links

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