Skip to main content
UK clinical trials - updated daily from ClinicalTrials.gov
TrialConnect
← Back to Search
Looking for participantsPhase2

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

Sponsor: Scholar Rock, Inc.

NCT ID: NCT07047144

View on ClinicalTrials.gov ↗

At a glance

What the study gets you
Access to the study treatment being tested
Type of study
Interventional (receives a drug or procedure)
Time in hospital
In-person visits at study sites — visit count not specified by the sponsor
Drug or intervention
Apitegromab (drug), Nusinersen (drug), Risdiplam (drug)
How long the study runs
Study runs about 42 months (dates as stated)
About the drug or intervention
Apitegromab — drug: 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. · Nusinersen — drug: Nusinersen is a current standard-of-care SMN therapy that targets the SMN2 gene. · Risdiplam — drug: Risdiplam is a current standard-of-care SMN therapy that targets the SMN2 gene.
Patient visit burden
Not specified by the sponsor
Type of study
Testing a treatment
Ages
Up to 2 Years
Who
All
Number of participants
52
Started
2025-09-15
Last checked
2026-10

Plain English Summary

What is this study?

  • • Testing a new treatment for spinal muscular atrophy
  • • Phase2 - 52 participants
  • • 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

Who can take part?

  • • Ages Up to 2 Years
  • • Diagnosed with spinal muscular atrophy

Where?

  • • Leeds - Leeds General Infirmary Children's Research Department

This is a simplified summary. Always discuss with your doctor before making any decisions.

About This Trial

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.

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

How this trial compares with your answers

Answer 2 more questions to improve match

What we know so far

Condition· Matched your search
Age· Tell us your age for better matching
Gender· Tell us your sex for better matching

Still need:

  • • Tell us your age for better matching
  • • Tell us your sex for better matching

Preliminary match based on your answers. Full eligibility requires on-site assessment including medical history, physical exam, and lab tests. This does not guarantee enrolment.

Eligibility at a Glance

Key info

  • Age: Up to 2 Years
  • Who can join: All genders

What the study is looking for

  • ✓Is \<2 years old at the time of the agreement to take part
  • ✓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-treatment that targets specific changes in the cancer (ie, onasemnogene abeparvovec-xioi) or are continuing...

Who cannot take part

  • ✗Nutritional status that is not anticipated to be stable throughout the study or medical necessity for a gastric...
  • ✗Any other physical limitations (eg, the subject requires cast for contractures) that would prevent the subject from...
See the full criteria
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.

Where Is This Study? (1 UK site)

Leeds General Infirmary Children's Research Department

Leeds LS1 3EX, United Kingdom

Recruiting
Site contact (verified)

How to Get in Touch

Scholar Rock

Sponsor contact

CONTACT

857-259-3860 MedicalInformation@scholarrock.com
Data sourced from ClinicalTrials.gov · Last verified: 2026-10