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.
How this trial compares with your answers
Answer 2 more questions to improve match
What we know so far
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
Where Is This Study? (1 UK site)
Leeds General Infirmary Children's Research Department
Leeds LS1 3EX, United Kingdom
How to Get in Touch
Scholar Rock
Sponsor contactCONTACT
