Long-Term Safety & Efficacy of Apitegromab in Patients With SMA Who Completed Previous Trials of Apitegromab

Part of paid clinical trials in Phoenix, Arizona.

Sponsor
Scholar Rock, Inc.
Study ID
NCT05626855
Phase
PHASE3
Status
Active Not Recruiting

Conditions

  • Anti-myostatin
  • Atrophy
  • Muscular Atrophy
  • Muscular Atrophy, Spinal
  • Neuromuscular Diseases
  • Neuromuscular Manifestations
  • SMA
  • Spinal Muscular Atrophy
  • Spinal Muscular Atrophy Type 2
  • Spinal Muscular Atrophy Type 3

Eligibility Criteria

Sex
ALL
Age
2 Years - N/A
Healthy Volunteers
Not accepted

Interventions

  • Apitegromab — DRUG
    Apitegromab (SRK-015) is an investigational, fully human immunoglobulin G4 monoclonal antibody that specifically binds to human proforms (i.e., inactive precursor forms) of myostatin, pro- and latent- myostatin, with high affinity, inhibiting activation of myostatin, a negative regulator of muscle growth and strength.

Study Details

The ONYX study is an Open-Label, Multicenter, Extension study that will evaluate the long-term safety and efficacy of Apitegromab in Patients with Type 2 and Type 3 SMA who have completed TOPAZ or SAPPHIRE.

Key Dates

Start date
Apr 17, 2023
Status verified
May 2025
Primary completion
Nov 1, 2026
Completion
May 2, 2029

Study Design

Enrollment
238 participants (actual)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: Treatment Period
    Patients who are ≥2 years of age with Type 2 and Type 3 SMA will receive apitegromab 20 mg/kg every 4 weeks by intravenous (IV) infusion during the 104-week Treatment Period

Primary Outcome Measure

Evaluate the long-term safety and tolerability of apitegromab in patients with Type 2 and Type 3 SMA [ Time Frame: Up to 6 years ]

Locations (25)

FacilityCityStateZIPSite coordinators
Phoenix Childrens HospitalPhoenixArizona85016-
UCSD Altman Clinical and Translational ResearchLa JollaCalifornia92037-
Children's Hospital Los AngelesLos AngelesCalifornia90027-
Stanford Neuroscience Health CenterPalo AltoCalifornia94304-
Children's Hospital ColoradoAuroraColorado80045-
Nemours Biomedical ResearchOrlandoFlorida32827-
Lurie Children's Hospital of ChicagoChicagoIllinois60611-
University of IowaIowa CityIowa52242-
University of Kansas Medical CenterFairwayKansas66205-
Johns HopkinsHospitalBaltimoreMaryland21287-
Boston Children's HospitalBostonMassachusetts02115-
Helen DeVos Children's HospitalGrand RapidsMichigan49503-
Gillette Children's Specialty HealthcareSaint PaulMinnesota55101-
Washington University Medical CampusSt LouisMissouri63110-
Columbia University Medical CenterNew YorkNew York10032-
Wake Forest University School of MedicineWinston-SalemNorth Carolina27157-
Nationwide Children's HospitalColumbusOhio43215-
Oregon Health & Science UniversityPortlandOregon97239-
The Children's Hospital of PhiladelphiaPhiladelphiaPennsylvania19104-
St. Jude Children's Research HospitalMemphisTennessee38105-
University of Texas Southwestern - Pediatric NeurologyDallasTexas75207-
University of UtahSalt Lake CityUtah84112-
Children's Specialty Group PLLC (Children's Hospital of The King's Daughters)Newport NewsVirginia23606-
Seattle Children's HospitalSeattleWashington98115-
University of Wisconsin HospitalMadisonWisconsin53792-

Find similar trials in Phoenix, AZ

By research site
Phoenix Childrens Hospital· Phoenix, AZUCSD Altman Clinical and Translational Research· La Jolla, CAChildren's Hospital Los Angeles· Los Angeles, CAStanford Neuroscience Health Center· Palo Alto, CAChildren's Hospital Colorado· Aurora, CONemours Biomedical Research· Orlando, FL

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