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

Recruiting now · Phase 2

Conditions studied: Spinal Muscular Atrophy, SMA, Spinal Muscular Atrophy Type 2, Spinal Muscular Atrophy Type 3, Neuromuscular Manifestations, Anti-myostatin

In brief

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.

Key facts

Study ID

NCT07047144

Run by

Scholar Rock, Inc.

People needed

52

Starts

2025-09-15

Expected to finish

2029-03-01

Last updated by the study team

2026-05-01

Who can join

Age: any, up to 2. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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

You may not qualify if…

• 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.

Where it is running

• Leeds General Infirmary Children's Research Department — Leeds, West Yorkshire, United Kingdom (enrolling)
• Children's Hospital of Orange County (CHOC) — Orange, California, United States (enrolling)
• Stanford Neuroscience Health Center (SNHC) — Palo Alto, California, United States (enrolling)
• Children's Hospital Colorado — Aurora, Colorado, United States (enrolling)
• Children's Healthcare of Atlanta — Atlanta, Georgia, United States (enrolling)
• University of Iowa — Iowa City, Iowa, United States (enrolling)
• Helen DeVos Children's Hospital at Spectrum Health — Grand Rapids, Michigan, United States (enrolling)
• Atrium Health Wake Forest Baptist — Winston-Salem, North Carolina, United States (enrolling)
• The Children's Hospital of Philadelphia — Philadelphia, Pennsylvania, United States (enrolling)
• Phoenix Children's Hospital — Phoenix, Arizona, United States (enrolling)
• University of Texas Southwestern Medical Center — Dallas, Texas, United States (enrolling)
• Neurology Rare Disease Center — Flower Mound, Texas, United States (enrolling)
• Cook Children's Medical Center — Fort Worth, Texas, United States (enrolling)
• Texas Children's Hospital (TCH)-Clinical Care Center (CCC) — Houston, Texas, United States (enrolling)
• UZ Gent — Ghent, East-Flanders, Belgium (enrolling)
• UZ Leuven - Campus Gasthuisberg — Leuven, Belgium (enrolling)
• CHR Citadelle — Liège, Belgium (enrolling)
• Hopital Trousseau - I-Motion — Paris, France (enrolling)
• Fondazione I.R.C.C.S. - Istituto Neurologico Carlo Besta — Milan, Italy (enrolling)
• Centro Clinico NeMO Milano - Fondazione Serena Onlus — Milan, Italy (enrolling)
• Fondazione Policlinico Universitario Agostino Gemelli IRCCS — Rome, Italy (enrolling)
• Universitair Medisch Centrum Utrecht — Utrecht, Netherlands (enrolling)
• Hospital Sant Joan de Déu Barcelona — Barcelona, Spain (enrolling)
• Hospital Universitari i Politecnico La Fecnic La Fe — Valencia, Spain (enrolling)
• Le Bonheur Children's Hospital — Memphis, Tennessee, United States

Full record on ClinicalTrials.gov

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