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RecruitingPhase 2

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

Scholar Rock, Inc. · NCT07047144

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Opens the official ClinicalTrials.gov record, with the study team’s contacts. Bored Studies doesn’t enroll anyone.

Key facts

Compensation
Not posted. Ask the study team before screening.
Who can join
Up to age 2 · All sexes
Healthy volunteers
Not accepted (a condition is required)
Study type
Interventional · Phase 2
Target enrollment
52
US sites recruiting
12
Study start
September 15, 2025
Record last updated
October 2, 2026

Status re-checked against ClinicalTrials.gov within the last 12 hours.

What the study is about

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.

Being studied: Apitegromab; Nusinersen; Risdiplam

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

Eligibility criteria

Copied from the official record. The study team makes the final decision at screening.

Show full 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.

Recruiting US locations

  • Phoenix Children's Hospital

    Phoenix, AZ 85016

    Site contact: Melony Valdez · 602-933-2017 · mvaldez8@phoenixchildrens.com

  • Children's Hospital of Orange County (CHOC)

    Orange, CA 92868

    Site contact: Sabrine Serrano · 714-509-7926 · Sabrina.serrano@choc.org

  • Stanford Neuroscience Health Center (SNHC)

    Palo Alto, CA 94304

    Site contact: Wendy Ao · 650-725-4341 · NeuromuscularResearch@stanford.edu

  • Children's Hospital Colorado

    Aurora, CO 80045

    Site contact: Hannah Kleiner · 720-222-1377 · hannah.kleiner@childrenscolorado.org

  • Children's Healthcare of Atlanta

    Atlanta, GA 30329

    Site contact: Julia Hunt · 404-785-4677 · julia.hunt@choa.org

  • University of Iowa

    Iowa City, IA 52242

    Site contact: Carrie Stephan · 319-356-2673 · carrie-stephan@iowa.edu

  • Helen DeVos Children's Hospital at Spectrum Health

    Grand Rapids, MI 49503

    Site contact: Rosa Sifontes-Escamilla · 616-486-6342 · rosa.sifontes-escamilla@helendevoschildrens.org

  • The Children's Hospital of Philadelphia

    Philadelphia, PA 19104

    Site contact: Karen Monono · 267-425-0158 · mononok@chop.edu

  • University of Texas Southwestern Medical Center

    Dallas, TX 75207

    Site contact: Holly Lawrence · 214-456-9561 · Holly.Lawrence@utsouthwestern.edu

  • Neurology Rare Disease Center

    Flower Mound, TX 75028

    Site contact: Jennifer Avelar · 972-999-1011 · jennifer.avelar@neuromdcenter.com

  • Cook Children's Medical Center

    Fort Worth, TX 76104

    Site contact: Kayla Blough · 682-303-1489 · kayla.blough@cookchildrens.org

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

    Houston, TX 77030

    Site contact: Kim Walker · 832-824-0253 · Kim.Walker@bcm.edu

Study contacts (from the official record)

  • Scholar Rock · 857-259-3860 · MedicalInformation@scholarrock.com
A Study to Evaluate How Apitegromab Works in Subjects Who Are Less… (NCT07047144) | Bored Studies