A Study to Evaluate How Apitegromab Works in Subjects Who Are Less Than 2 Years Old and Have Spinal Muscular Atrophy

Study Identifier:
SRK-015-005
ClinicalTrials.gov Identifier:
NCT07047144
EudraCT Identifier:
N/A
EU CT Number:
N/A
Sponsor:
Scholar Rock, Inc.
Recruiting

Study Details

Medical Condition
  • All Spinal Muscular Atrophies
  • Spinal Muscular Atrophy Type II
  • Spinal Muscular Atrophy Type III
Study Drug
  • Drug: Apitegromab
  • Drug: Nusinersen
  • Drug: Risdiplam
Date
Sep 2025 - Nov 2028
Phase 1
Phase 2
Phase 3
Phase 4
Patient Requirements
Sex: Female & Male
Age: N/A - 2 Years
Requirements Information
Inclusion and Exclusion Criteria
Inclusion Criteria
  • Is \<2 years old at the time of the informed consent
  • Had a gestational age of ≥35 weeks and gestational body weight ≥2.0 kg at birth
  • Has confirmed diagnosis of 5q autosomal recessive SMA
  • Has confirmed presence of SMN2 gene copy(ies)
  • Must have been treated with an approved SMN1-targeted therapy (ie, onasemnogene abeparvovec-xioi) or are continuing to be treated with an approved SMN2-targeted therapy (ie, nusinersen or risdiplam)
  • Body weight for age is no less than 1st percentile based on the WHO Child Growth Standards at the Screening Visit
  • Has delayed motor milestones for age attributed to SMA at the discretion of the Investigator or a CHOP-INTEND score \<55
Exclusion Criteria
  • Nutritional status that is not anticipated to be stable throughout the study or medical necessity for a gastric feeding tube, where most feeds are administered by this route
  • Major orthopedic issues such as severe scoliosis or severe contractures or interventional procedure, including spine or hip surgery, which is considered to have the potential to substantially limit the ability of the subject to be evaluated on any motor function outcome measures, within 6 months before Screening or anticipated during the study
  • Any other physical limitations (eg, the subject requires cast for contractures) that would prevent the subject from undergoing motor function outcome measures throughout the study.

Protocol Summary

This double-blind, Phase 2, multiple-dose study will be conducted to evaluate the PK/PD, efficacy, safety, and tolerability of apitegromab in subjects \<2 years old with 5q autosomal recessive SMA who have delayed motor milestones for their age attributed to SMA at the discretion of the Investigator or a Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP-INTEND) score \<55.

Trial Locations

Location
Status
Location
Phoenix Children's Hospital
Phoenix, Arizona, United States, 85016
Status
Recruiting
Location
Children's Hospital of Orange County (CHOC)
Orange, California, United States, 92868
Status
Recruiting
Location
Stanford Neuroscience Health Center (SNHC)
Palo Alto, California, United States, 94304
Status
Recruiting
Location
Children's Hospital Colorado
Aurora, Colorado, United States, 80045
Status
Recruiting
Location
Children's Healthcare of Atlanta
Atlanta, Georgia, United States, 30329
Status
Recruiting
Location
University of Iowa
Iowa City, Iowa, United States, 52242
Status
Recruiting
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