Clinical trial NCT06411288: Global Study of Del-desiran for the Treatment of DM1
Official title: A Phase 3 Randomized, Double-Blind, Placebo-Controlled, Global Study to Evaluate the Efficacy and Safety of Intravenous AOC 1001 for the Treatment of Myotonic Dystrophy Type 1
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE3
Conditions: DM1
Myotonic Dystrophy
Myotonic Dystrophy 1
Myotonia
Myotonic Dystrophy Type 1 (DM1)
Dystrophy Myotonic
Myotonic Disorders
Steinert Disease
Steinert
Myotonic Muscular Dystrophy
Keywords: DM1
Myotonic Dystrophy
Myotonic Dystrophy 1
Myotonia
Myotonic Dystrophy Type 1 (DM1)
Dystrophy Myotonic
Myotonic Disorders
Steinert Disease
Steinert
Myotonic Muscular Dystrophy
HARBOR
Avidity Biosciences
Avidity
AOC 1001
Del-desiran
Delpacibart etedesiran
Lead sponsor: Avidity Biosciences, Inc.
Sponsor class: INDUSTRY
First posted: 2024-05-13
Enrollment: 150
Sex: ALL
Minimum age (years): 16
Maximum age (years): 65
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: A Phase 3 Randomized, Double-Blind, Placebo-Controlled, Global Study to Evaluate the Efficacy and Safety of Intravenous Delpacibart Etedesiran (abbreviated del-desiran, formerly AOC 1001) for the Treatment of Myotonic Dystrophy Type 1

Detailed description: The study consists of a Screening Period of up to 6 weeks and 54-week Treatment Period. The anticipated duration is approximately 60 weeks.

Participants will be randomized to receive an intravenous infusion of either del-desiran or placebo at the clinical study site every 8 weeks for a total of 7 doses. The final dose will occur at Week 48, followed by a final assessment at Week 54.

After completion of Week 54 assessments, eligible participants will have the option to enroll into an open label extension (OLE) study, pending regulatory approval.

An Independent Data Monitoring Committee (IDMC) comprised of members independent and external to the Sponsor will review safety, tolerability, and efficacy (as needed) data of this study at regular intervals.

Eligibility criteria: Key Inclusion Criteria:

* Clinical and genetic diagnosis (CTG repeat ≥ 100) of DM1
* Ability to walk independently (orthoses and ankle braces allowed) for at least 10 meters at screening

Key Exclusion Criteria:

* Breastfeeding, pregnancy, or intent to become pregnant during the study
* Unwilling or unable to comply with contraceptive requirements
* Abnormal lab values, conditions or diseases that would make the participant unsuitable for the study
* Diabetes that is not adequately controlled
* History of decompensated heart failure within 3 months of screening. Participants with preexisting pacemaker/ICD are not excluded.
* Body Mass Index \> 35 kg/m2 at Screening
* Recently treated with an investigational drug or biological agent
* Treatment with anti-myotonic medication within 5 half-lives or 14 days of baseline, whichever is longer, prior to baseline.

Note: Additional protocol defined Inclusion and Exclusion criteria apply

Primary outcomes:
measure: Hand function
timeFrame: Through Week 30
Secondary outcomes:
measure: Hand grip strength
timeFrame: Through Week 30
measure: Quantitative Muscle Testing composite score
timeFrame: Through Week 30
measure: Myotonic Dystrophy Type 1 activity and participation scale c
timeFrame: Through Week 30
Other outcomes:
Locations:
city: Stanford
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: Tampa
country: United States
facility: University Research Center of South Florida
state: Florida
city: Indianapolis
country: United States
facility: Indiana University (IU)
state: Indiana
city: Kansas City
country: United States
facility: Kansas University Medical Center
state: Kansas
city: Baltimore
country: United States
facility: Kennedy Krieger Institute
state: Maryland
city: Boston
country: United States
facility: Beth Israel Deaconess Medical Center
state: Massachusetts
city: Minneapolis
country: United States
facility: University of Minnesota
state: Minnesota
city: Rochester
country: United States
facility: University of Rochester Medical Center
state: New York
city: Durham
country: United States
facility: Duke University Medical Center
state: North Carolina
city: Winston-Salem
country: United States
facility: Wake Forest
state: North Carolina
city: Cincinnati
country: United States
facility: University of Cincinnati Gardner Neuroscience Institute
state: Ohio
city: Columbus
country: United States
facility: Ohio State University
state: Ohio
city: Philadelphia
country: United States
facility: University of Pennsylvania
state: Pennsylvania
city: Houston
country: United States
facility: Houston Methodist Neurological Institute
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: The Ottawa Hospital
state: Ontario
city: Montreal
country: Canada
facility: Montreal Neurological Institute
state: Quebec
city: Aarhus N
country: Denmark
facility: Aarhus University Hospital
city: Kobenhavn
country: Denmark
facility: Rigshospitalet
city: Paris
country: France
facility: AP-HP Hopital Pitie-Salpetriere
city: Munich
country: Germany
facility: Klinikum der Ludwig-Maximilians-Universitaet Muenchen
city: Milano
country: Italy
facility: Fondazione Serena Onlus - Centro Clinico NeMO Milano
city: Aomori-shi
country: Japan
facility: Aomori Hospital
state: Aomori
city: Osaka
country: Japan
facility: National Hospital Organization Osaka Toneyama Medical Center
city: Osaka
country: Japan
facility: Osaka University Hospital
city: Tokyo
country: Japan
facility: National Center of Neurology and Psychiatry
city: Maastricht
country: Netherlands
facility: Maastricht University Medical Center
city: Nijmegen
country: Netherlands
facility: Stichting Radboud Universitair Medisch Centrum
city: San Sebastian
country: Spain
facility: Hospital Universitario Donostia
city: London
country: United Kingdom
facility: University College London Hospital
city: London
country: United Kingdom
facility: St. Georges University Hospitals NHS Foundation Trust
Study officials:
References: