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

NCT07047144 · tracked via the Priya Life Science UK tracker

Sponsor

Scholar Rock, Inc.

Phase

Phase 2

Started

2025-09-15

Last updated

2026-09-14

Condition(s) studied

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

Investigational drug(s) / intervention(s)

ApitegromabNusinersenRisdiplam

Apitegromab: Apitegromab is a fully human anti-proMyostatin monoclonal antibody (mAb) of the immunoglobulin G4 (IgG4)/lambda isotype that specifically binds to human pro/latent myostatin with high affinity inhibiting myostatin activation. SRK-015 will be administered every 4 weeks by intravenous (IV) infusion.

Nusinersen: Nusinersen is a current standard-of-care SMN therapy that targets the SMN2 gene. It will be administered intrathecally per the prescribing information.

Risdiplam: Risdiplam is a current standard-of-care SMN therapy that targets the SMN2 gene. It will be administered orally per the prescribing information.

Study 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

Sex

ALL

Min age

—

Max age

2 Years

Healthy volunteers

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.

Primary outcome measure(s)

Evaluate the PK of apitegromab in subjects <2 years old with SMA — 52 Weeks
Apitegromab concentrations in serum
Evaluate the PD of apitegromab in subjects <2 years old with SMA — 52 Weeks
Total latent myostatin concentrations in serum
Evaluate the motor function outcomes (ie, efficacy) due to apitegromab treatment — 48 Weeks
Change from baseline in the raw score of the Bayley Scale of Infant and Toddler Development, Fourth Edition - Gross Motor Subscale (BSID-4 GMS) at 48 weeks. BSID-4 GMS is a standardized assessment commonly used to evaluate development across 5 domains in infants and young children. It consists of 58 items, scored from 0 to 2 for each item, with higher scores indicating better gross motor development.

Trial sites (24)

Facility	City	Region	Status
Phoenix Children's Hospital	Phoenix	Arizona	Recruiting
Children's Hospital of Orange County (CHOC)	Orange	California	Recruiting
Stanford Neuroscience Health Center (SNHC)	Palo Alto	California	Recruiting
Children's Hospital Colorado	Aurora	Colorado	Recruiting
Children's Healthcare of Atlanta	Atlanta	Georgia	Recruiting
University of Iowa	Iowa City	Iowa	Recruiting
Helen DeVos Children's Hospital at Spectrum Health	Grand Rapids	Michigan	Recruiting
The Children's Hospital of Philadelphia	Philadelphia	Pennsylvania	Recruiting
Le Bonheur Children's Hospital	Memphis	Tennessee	Not Yet Recruiting
University of Texas Southwestern Medical Center	Dallas	Texas	Recruiting
Neurology Rare Disease Center	Flower Mound	Texas	Recruiting
Cook Children's Medical Center	Fort Worth	Texas	Recruiting
Texas Children's Hospital (TCH)-Clinical Care Center (CCC)	Houston	Texas	Recruiting
UZ Gent	Ghent	East-Flanders	Recruiting
UZ Leuven - Campus Gasthuisberg	Leuven	Belgium	Recruiting
CHR Citadelle	Liège	Belgium	Recruiting
Hopital Trousseau - I-Motion	Paris	France	Recruiting
Fondazione I.R.C.C.S. - Istituto Neurologico Carlo Besta	Milan	Italy	Recruiting
Centro Clinico NeMO Milano - Fondazione Serena Onlus	Milan	Italy	Recruiting
Fondazione Policlinico Universitario Agostino Gemelli IRCCS	Rome	Italy	Recruiting
Universitair Medisch Centrum Utrecht	Utrecht	Netherlands	Recruiting
Hospital Sant Joan de Déu Barcelona	Barcelona	Spain	Recruiting
Hospital Universitari i Politecnico La Fecnic La Fe	Valencia	Spain	Recruiting
Leeds General Infirmary Children's Research Department	Leeds	West Yorkshire	Recruiting

On this site

📄 Spinraza (nusinersen) drug profile → 📄 Evrysdi (risdiplam) drug profile →

More Scholar Rock, Inc. trials in the UK

Long-Term Safety & Efficacy of Apitegromab in Patients With SMA Who Completed Previous Trials of Apitegromab Active, not recruiting

Other trials for the same condition

Long-term Follow-up Study of Patients Receiving Onasemnogene Abeparvovec-xioi Active, not recruiting

Long-Term Safety & Efficacy of Apitegromab in Patients With SMA Who Completed Previous Trials of Apitegromab Active, not recruiting

Study of an Intrathecal Port and Catheter System for Subjects With Spinal Muscular Atrophy Recruiting

A Study to Evaluate the Efficacy and Safety of Taldefgrobep Alfa in Participants With Spinal Muscular Atrophy Active, not recruiting

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT07047144 on ClinicalTrials.gov ↗ ← All trials in the UK