Clinical trial NCT05027269: Study of AOC 1001 in Adult Myotonic Dystrophy Type 1 (DM1) Patients
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
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
PHASE2
Conditions: DM1
Myotonic Dystrophy 1
Myotonic Dystrophy
Myotonic Dystrophy Type 1 (DM1)
Dystrophy Myotonic
Myotonic Disorders
Steinert Disease
Myotonic Muscular Dystrophy
Keywords: DM1
Myotonic Dystrophy 1
Myotonic Dystrophy Type 1 (DM1)
Myotonic Dystrophy
DM
Dystrophy Myotonic
Myotonic Disorders
Steinert Disease
MARINA
Avidity Biosciences
Avidity
AOC 1001
Myotonic Muscular Dystrophy
Lead sponsor: Avidity Biosciences, Inc.
Sponsor class: INDUSTRY
First posted: 2021-08-30
Enrollment: 38
Sex: ALL
Minimum age (years): 18
Maximum age (years): 65
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: SEQUENTIAL
Primary purpose: TREATMENT

Brief 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.


Eligibility 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.

Primary outcomes:
measure: Frequency of treatment emergent adverse events (TEAEs)
timeFrame: Through study completion, up to Day 183
Secondary outcomes:
measure: Plasma pharmacokinetic (PK) parameters
timeFrame: Through study completion, up to Day 183
measure: Plasma pharmacokinetic (PK) parameters
timeFrame: Through study completion, up to Day 183
measure: Plasma pharmacokinetic (PK) parameters
timeFrame: Through study completion, up to Day 183
measure: Plasma pharmacokinetic (PK) parameters
timeFrame: Through study completion, up to Day 183
measure: Urine pharmacokinetic (PK) parameters
timeFrame: Through study completion, up to Day 183
measure: AOC 1001 levels in muscle tissue
timeFrame: Through study completion, up to Day 183
measure: Change and percentage change from baseline in DMPK mRNA knockdown
timeFrame: Through study completion, up to Day 183
measure: Change and percentage change from baseline in Spliceopathy
timeFrame: Through study completion, up to Day 183
Other outcomes:
Locations:
city: Los Angeles
country: United States
facility: University of California Los Angeles
state: California
city: Palo Alto
country: United States
facility: Stanford University
state: California
city: Denver
country: United States
facility: University of Colorado
state: Colorado
city: Gainesville
country: United States
facility: University of Florida
state: Florida
city: Kansas City
country: United States
facility: Kansas University Medical Center
state: Kansas
city: Rochester
country: United States
facility: University of Rochester Medical Center
state: New York
city: Columbus
country: United States
facility: Ohio State University
state: Ohio
city: Richmond
country: United States
facility: Virginia Commonwealth University
state: Virginia
Study officials:
affiliation: Avidity Biosciences, Inc.
name: Li Tai, MD
role: STUDY_DIRECTOR
References: