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

RecruitingPhase 2

A Phase 2, Double-Blind Study to Evaluate the Pharmacokinetics, Pharmacodynamics, Efficacy, and Safety of Apitegromab in Subjects <2 Years Old With Spinal Muscular Atrophy (SMA)

Who can join

Up to age 2 · All sexes

Full eligibility criteria
Inclusion Criteria:

1. Is \<2 years old at the time of the informed consent
2. Had a gestational age of ≥35 weeks and gestational body weight ≥2.0 kg at birth
3. Has confirmed diagnosis of 5q autosomal recessive SMA
4. Has confirmed presence of SMN2 gene copy(ies)
5. 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)
6. Body weight for age is no less than 1st percentile based on the WHO Child Growth Standards at the Screening Visit
7. Has delayed motor milestones for age attributed to SMA at the discretion of the Investigator or a CHOP-INTEND score \<55

Exclusion Criteria:

1. 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
2. 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
3. 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.

About the study

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.

What is being tested

Sponsor: Scholar Rock, Inc. · Participants: 52 · Started: Sep 15, 2025

Contact the study team

Official record on ClinicalTrials.gov — NCT07047144

Locations in the U.S.

ArizonaPhoenix Children's Hospital, Phoenix
CaliforniaChildren's Hospital of Orange County (CHOC), Orange
Stanford Neuroscience Health Center (SNHC), Palo Alto
ColoradoChildren's Hospital Colorado, Aurora
GeorgiaChildren's Healthcare of Atlanta, Atlanta
IowaUniversity of Iowa, Iowa City
MichiganHelen DeVos Children's Hospital at Spectrum Health, Grand Rapids
PennsylvaniaThe Children's Hospital of Philadelphia, Philadelphia
TennesseeLe Bonheur Children's Hospital, Memphis (Not yet recruiting)
TexasUniversity of Texas Southwestern Medical Center, Dallas
Neurology Rare Disease Center, Flower Mound
Cook Children's Medical Center, Fort Worth
Texas Children's Hospital (TCH)-Clinical Care Center (CCC), Houston

Conditions

From ClinicalTrials.gov, data retrieved Sep 29, 2026. Each study sets its own eligibility; the study team decides who can join.