Clinical trial NCT05479981: Extension of AOC 1001-CS1 (MARINA) Study in Adult Myotonic Dystrophy Type 1 (DM1) Patients
Official title: A Phase 2 Extension Study to Evaluate the Long-Term Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of AOC 1001 Administered Intravenously to Adult Myotonic Dystrophy Type 1 (DM1) Patients
Study type: INTERVENTIONAL
Overall status: ACTIVE_NOT_RECRUITING
Phase: PHASE2
Conditions: DM1
Muscular Dystrophies
Myotonic Dystrophy
Myotonic Dystrophy 1
Myotonic Disorders
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, Inborn
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Keywords: DM1
Myotonic Dystrophy 1
Myotonic Dystrophy Type 1 (DM1)
Myotonic Dystrophy
DM
Dystrophy Myotonic
Myotonic Disorders
Steinert Disease
MARINA-OLE
MARINA
Avidity Biosciences
Avidity
AOC 1001
Myotonic Muscular Dystrophy
Lead sponsor: Avidity Biosciences, Inc.
Sponsor class: INDUSTRY
First posted: 2022-07-29
Enrollment: 37
Sex: ALL
Minimum age (years): 18
Maximum age (years): 66
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief 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

Detailed description: This study will continue to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics, and efficacy of AOC 1001 in participants that enrolled in the randomized, placebo-controlled, First-In-Human Phase 1/2 AOC 1001-CS1 (MARINA) clinical study.

Participants from AOC 1001-CS1 are eligible to enroll in AOC 1001-CS2 if they have satisfactorily completed AOC 1001-CS1.

The total duration of active treatment in AOC 1001-CS2 is up to 58 months. Once participants have completed active treatment, they will be followed through an 8-week safety follow-up period. The sponsor may extend active treatment beyond 58 months at a future timepoint.

As of September 2024, the dosing regimen was updated to every 8 weeks.

Eligibility criteria: Key Inclusion Criteria:

* Completion of AOC 1001-CS1 (MARINA) study with satisfactory compliance and no significant tolerability issues

Key Exclusion Criteria:

* Pregnancy, intent to become pregnant, or active breastfeeding
* Unwilling or unable to continue to comply with contraceptive requirements
* Any new conditions or worsening of existing conditions that in the opinion of the investigator or sponsor would make the participant unsuitable for the study or could interfere with participation or completion of the study

Primary outcomes:
measure: Number and severity of treatment-emergent adverse events (TEAEs)
timeFrame: Through study completion, up to Week 248
Secondary outcomes:
measure: AOC 1001 levels in muscle tissue
timeFrame: Through Day 183
measure: Change and percentage change from baseline in DMPK mRNA knockdown
timeFrame: Through Day 183
measure: Change and percentage change from baseline in Spliceopathy
timeFrame: Through Day 183
measure: Plasma pharmacokinetic (PK) parameters
timeFrame: Through study completion, up to Week 248
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:
References: