Efficacy and Safety of Apitegromab in Patients With Later-Onset Spinal Muscular Atrophy Treated With Nusinersen or Risdiplam

Part of paid clinical trials in Birmingham, Alabama.

Sponsor
Scholar Rock, Inc.
Study ID
NCT05156320
Phase
PHASE3
Status
Completed

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 - 21 Years
Healthy Volunteers
Not accepted

Interventions

  • Apitegromab — DRUG
    Apitegromab is a fully human anti-proMyostatin monoclonal antibody (mAb) of the immunoglobulin G4 (IgG4)/lambda isotype that specifically binds to human pro/latent myostatin with high affinity inhibiting myostatin activation. SRK-015 was administered every 4 weeks by intravenous (IV) infusion.
  • Placebo — DRUG
    Placebo was administered every 4 weeks by intravenous (IV) infusion.

Study Details

This Phase 3 trial (Study SRK-015-003) was conducted in patients ≥2 years old at Screening, who were previously diagnosed with later-onset spinal muscular atrophy (SMA) (i.e., Type 2 and Type 3 SMA) and were receiving an approved survival motor neuron (SMN) upregulator therapy (i.e., either nusinersen or risdiplam), to confirm the efficacy and safety of apitegromab as an adjunctive therapy to nusinersen and evaluate the efficacy and safety of apitegromab as an adjunctive therapy to risdiplam.

Key Dates

Start date
Apr 14, 2022
Status verified
Jan 2026
Primary completion
Dec 18, 2024
Completion
Dec 18, 2024

Study Design

Enrollment
188 participants (actual)
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT

Arms

  • Experimental: Main Efficacy Population (Apitegromab 10 mg/kg)
    Aged 2-12 years at Screening. Participants were randomized to receive apitegromab 10 mg/kg for up to 52 weeks.
  • Experimental: Main Efficacy Population (Apitegromab 20 mg/kg)
    Aged 2-12 years at Screening. Participants were randomized to receive apitegromab 20 mg/kg for up to 52 weeks.
  • Placebo Comparator: Main Efficacy Population (Placebo)
    Aged 2-12 years at Screening. Participants were randomized to receive placebo for up to 52 weeks.
  • Experimental: Exploratory Subpopulation (Apitegromab)
    Aged 13-21 years at Screening. Participants were randomized to receive apitegromab 20 mg/kg for up to 52 weeks.
  • Placebo Comparator: Exploratory Subpopulation (Placebo)
    Aged 13-21 years at Screening. Participants were randomized to receive placebo for up to 52 weeks.

Primary Outcome Measure

Main Efficacy Population: Change from Baseline in Hammersmith Functional Motor Scale Expanded (HFMSE) total score. [ Time Frame: Baseline up to 12 months. ]

Locations (26)

FacilityCityStateZIPSite coordinators
Children's of AlabamaBirminghamAlabama35233-
Phoenix Children's HospitalPhoenixArizona85016-
Children's Hospital of Los AngelesLos AngelesCalifornia90027-
Stanford University Medical CenterPalo AltoCalifornia94304-
Rady's Children's Hospital/UCSDSan DiegoCalifornia92123-
Children's Hospital ColoradoAuroraColorado80045-
Nemours Children's HospitalOrlandoFlorida32827-
Ann & Robert H. Lurie Children's Hospital of ChicagoChicagoIllinois60611-
University of IowaIowa CityIowa52242-
University of Kansas Medical CenterKansas CityKansas66160-
The Johns Hopkins UniversityBaltimoreMaryland21287-
Boston Children's HospitalBostonMassachusetts02115-
Helen DeVos Children's HospitalGrand RapidsMichigan49503-
Gillette Children's Specialty HealthcareSaint PaulMinnesota55101-
Washington University School of Medicine in St. LouisSt LouisMissouri63110-
Columbia University, SMA Clinical Research CenterNew YorkNew York10032-
Wake Forest Baptist Medical CenterWinston-SalemNorth Carolina27157-
Nationwide Children's HospitalColumbusOhio43205-
Oregon Health & Sciences UniversityPortlandOregon97239-
Childrens Hospital of PhiladelphiaPhiladelphiaPennsylvania19104-
St. Jude Children's Research HospitalMemphisTennessee38105-
Children's Medical Center DallasDallasTexas75235-
University of UtahSalt Lake CityUtah84112-
Children's Hospital of The King's DaughtersNorfolkVirginia23507-
Seattle Children's HospitalSeattleWashington98105-
University of Wisconsin School of Medicine and Public HealthMadisonWisconsin53705-

Find similar trials in Birmingham, AL

By research site
Children's of Alabama· Birmingham, ALPhoenix Children's Hospital· Phoenix, AZChildren's Hospital of Los Angeles· Los Angeles, CAStanford University Medical Center· Palo Alto, CARady's Children's Hospital/UCSD· San Diego, CAChildren's Hospital Colorado· Aurora, CO

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