Clinical trial NCT06547216: Phase 2 Open-label Extension Study of AOC 1020 in Participants with Facioscapulohumeral Muscular Dystrophy (FSHD)
Official title: A Phase 2 Open-label Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of AOC 1020 Administered Intravenously to Participants with Facioscapulohumeral Muscular Dystrophy (FSHD)
Study type: INTERVENTIONAL
Overall status: ENROLLING_BY_INVITATION
Phase: PHASE2
Conditions: FSHD
FSHD1
FSHD2
FMD
FMD2
Fascioscapulohumeral Muscular Dystrophy
Fascioscapulohumeral Muscular Dystrophy Type 1
Fascioscapulohumeral Muscular Dystrophy Type 2
Dystrophies, Facioscapulohumeral Muscular
Dystrophy, Facioscapulohumeral Muscular
Facioscapulohumeral Muscular Dystrophy 1
Facioscapulohumeral Muscular Dystrophy 2
Facio-Scapulo-Humeral Dystrophy
Atrophy, Facioscapulohumeral
Atrophies, Facioscapulohumeral
Facioscapulohumeral Atrophy
Muscular Dystrophies
Muscular Dystrophy, Facioscapulohumeral
FSH Muscular Dystrophy
Landouzy Dejerine Dystrophy
Landouzy-Dejerine Muscular Dystrophy
Dystrophies, Landouzy-Dejerine
Dystrophy, Landouzy-Dejerine
Landouzy-Dejerine Syndrome
Muscular Dystrophy, Landouzy Dejerine
Progressive Muscular Dystrophy
FSH
Keywords: FORTITUDE
Avidity
Avidity Biosciences
AOC 1020
FORTITUDE-OLE
FORTITUDE OLE
Lead sponsor: Avidity Biosciences, Inc.
Sponsor class: INDUSTRY
First posted: 2024-08-09
Enrollment: 84
Sex: ALL
Minimum age (years): 16
Maximum age (years): 70
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: A Phase 2 Open-label Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of AOC 1020 Administered Intravenously to Participants with Facioscapulohumeral Muscular Dystrophy (FSHD)

Detailed description: This study will continue to evaluate the safety, tolerability, and efficacy of AOC 1020 in participants who were treated in the randomized, placebo-controlled, Phase 1/2 AOC 1020-CS1 clinical study.

Participants from AOC 1020-CS1 are eligible to enroll in AOC 1020-CS2 if they have satisfactorily completed AOC 1020-CS1. All participants who enroll in AOC 1020-CS2 will receive AOC 1020 regardless of whether they received AOC 1020 or placebo in AOC 1020-CS1. Dosing will occur every 6 to 7 weeks (twice quarterly).

The total duration of active treatment in AOC 1020-CS2 is approximately 22 months. Once participants have completed active treatment, they will be followed through a 12-week safety follow-up period to monitor for adverse events (AEs), concomitant medications, and pregnancy status (as applicable). Total duration on study is up to approximately 25 months (2 years 1 month), excluding the potential 8-week screening period.

Eligibility criteria: Inclusion Criteria:

1. Ability to provide written informed consent (signed and dated) and any authorizations required by local law and be willing and able to comply with all study requirements. When enrolling participants who are minors, it is necessary to also obtain consent from a legally designated representative and the participant will receive information in a way adapted to their age and mental maturity.
2. Completion of AOC 1020-CS1 with the following as judged by the Investigator and Sponsor:

   1. No significant tolerability issues
   2. Satisfactory compliance with the AOC 1020-CS1 protocol requirements

Exclusion Criteria:

1. Pregnancy, intent to become pregnant during the clinical study, or active breastfeeding.
2. Unwilling or unable to continue to comply with contraceptive requirements for the length of AOC 1020-CS2.
3. Any new conditions or worsening of existing condition(s) 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. (Note: FSHD progression is not exclusionary, even if the participant no longer has the ability to walk 10 meters without a walker or two canes.)

Primary outcomes:
measure: Incidence of treatment-emergent adverse events
timeFrame: Through study completion, up to Day 729
Secondary outcomes:
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: San Diego
country: United States
facility: University of California San Diego
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: Atlanta
country: United States
facility: Rare Disease Research
state: Georgia
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: Durham
country: United States
facility: Duke University
state: North Carolina
city: Columbus
country: United States
facility: Ohio State University
state: Ohio
city: Philadelphia
country: United States
facility: University of Pennsylvania
state: Pennsylvania
city: Dallas
country: United States
facility: University of Texas Southwestern
state: Texas
city: Richmond
country: United States
facility: Virginia Commonwealth University
state: Virginia
city: Seattle
country: United States
facility: University of Washington
state: Washington
city: Ottawa
country: Canada
facility: University of Ottawa
state: Ontario
city: London
country: United Kingdom
facility: University College London
city: Sheffield
country: United Kingdom
facility: University of Sheffield
Study officials:
affiliation: Avidity Biosciences, Inc.
name: Amy Halseth, Ph.D.
role: STUDY_DIRECTOR
References: