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

A

Avidity Biosciences

Status and phase

Completed
Phase 2
Phase 1

Conditions

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

Treatments

Drug: Placebo
Drug: AOC 1001

Study type

Interventional

Funder types

Industry

Identifiers

NCT05027269
AOC 1001-CS1

Details and patient eligibility

About

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.

Enrollment

38 patients

Sex

All

Ages

18 to 65 years old

Volunteers

No Healthy Volunteers

Inclusion and exclusion criteria

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.

Trial design

Primary purpose

Treatment

Allocation

Randomized

Interventional model

Sequential Assignment

Masking

Quadruple Blind

38 participants in 4 patient groups, including a placebo group

Part A Single Dose: AOC 1001 Dose Level 1
Experimental group
Description:
AOC 1001 will be administered once.
Treatment:
Drug: AOC 1001
Part A Single Dose: Placebo
Placebo Comparator group
Description:
Saline will be administered once.
Treatment:
Drug: Placebo
Part B Multiple Ascending Dose: AOC 1001 Dose Levels 2 & 3
Experimental group
Description:
AOC 1001 will be administered three times.
Treatment:
Drug: AOC 1001
Part B Multiple Ascending Dose: Placebo
Placebo Comparator group
Description:
Saline will be administered three times.
Treatment:
Drug: Placebo

Trial contacts and locations

0

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Data sourced from clinicaltrials.gov

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