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NCT07047144PHASE2Recruiting

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

Scholar Rock, Inc.

Start Date

9/15/2025

Completion Date

3/1/2029

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.

Eligibility Criteria

Age Range: No minimum to 2 years

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.

Interventions

DRUG

Apitegromab

DRUG

Nusinersen

DRUG

Risdiplam

Interested in This Trial?

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Conditions

Spinal Muscular AtrophySMASpinal Muscular Atrophy Type 2Spinal Muscular Atrophy Type 3Neuromuscular ManifestationsAnti-myostatin

Locations

Phoenix Children's Hospital

Phoenix, Arizona 85016

United States

Children's Hospital of Orange County (CHOC)

Orange, California 92868

United States

Stanford Neuroscience Health Center (SNHC)

Palo Alto, California 94304

United States

Children's Hospital Colorado

Aurora, Colorado 80045

United States

Children's Healthcare of Atlanta

Atlanta, Georgia 30329

United States

University of Iowa

Iowa City, Iowa 52242

United States

Helen DeVos Children's Hospital at Spectrum Health

Grand Rapids, Michigan 49503

United States

Atrium Health Wake Forest Baptist

Winston-Salem, North Carolina 27157

United States

The Children's Hospital of Philadelphia

Philadelphia, Pennsylvania 19104

United States

Le Bonheur Children's Hospital

Memphis, Tennessee 38103

United States

University of Texas Southwestern Medical Center

Dallas, Texas 75207

United States

Neurology Rare Disease Center

Flower Mound, Texas 75028

United States

Cook Children's Medical Center

Fort Worth, Texas 76104

United States

Texas Children's Hospital (TCH)-Clinical Care Center (CCC)

Houston, Texas 77030

United States

UZ Gent

Ghent, East-Flanders 9000

Belgium

UZ Leuven - Campus Gasthuisberg

Leuven, 3000

Belgium

CHR Citadelle

Liège, 4000

Belgium

Hopital Trousseau - I-Motion

Paris, 75012

France

Fondazione I.R.C.C.S. - Istituto Neurologico Carlo Besta

Milan, 20133

Italy

Centro Clinico NeMO Milano - Fondazione Serena Onlus

Milan, 20162

Italy

Fondazione Policlinico Universitario Agostino Gemelli IRCCS

Rome, 00168

Italy

Universitair Medisch Centrum Utrecht

Utrecht, 3584 CX

Netherlands

Hospital Sant Joan de Déu Barcelona

Barcelona, 08950

Spain

Hospital Universitari i Politecnico La Fecnic La Fe

Valencia, 46026

Spain

Leeds General Infirmary Children's Research Department

Leeds, West Yorkshire LS1 3EX

United Kingdom