RECRUITING

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

Conditions

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

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

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.

Official Title

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)

Quick Facts

Study Start:2025-09

Study Completion:2029-03

Study Type:Not specified

Phase:Not Applicable

Enrollment:Not specified

Status:RECRUITING

Study ID

NCT07047144

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:Not specified to 2 Years

Sexes Eligible for Study:ALL

Accepts Healthy Volunteers:No

Standard Ages:CHILD

Inclusion Criteria	Exclusion 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	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.

Inclusion Criteria

Exclusion 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

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.

Contacts and Locations

Study Contact

Scholar Rock

CONTACT

857-259-3860

clinicaltrials@scholarrock.com

Study Locations (Sites)

Atrium Health Wake Forest Baptist

Winston-Salem, North Carolina, 27157

United States

Neurology Rare Disease Center

Denton, Texas, 76208

United States

Collaborators and Investigators

Sponsor: Scholar Rock, Inc.

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2025-09

Study Completion Date2029-03

Study Record Updates

Study Start Date2025-09

Study Completion Date2029-03

Terms related to this study

Additional Relevant MeSH Terms

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