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

Recruiting Phase 2 Interventional Study
Spinal Muscular Atrophy SMA Spinal Muscular Atrophy Type 2 Spinal Muscular Atrophy Type 3 Neuromuscular Manifestations Anti-myostatin
No Placebo Group Every participant receives an active treatment — no one gets a placebo. Prior Safety Data This treatment has already been tested in at least one earlier human trial.
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At a Glance
Age
Up to 2
Sex
Any
Trial phase
Phase 2
Study type
Interventional
Purpose
Treatment
Participants needed
52 (estimated)
Sponsor
Scholar Rock, Inc. · Industry
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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…
Trial Locations
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Eligibility 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 …
Contacts

Scholar Rock

857-259-3860

MedicalInformation@scholarrock.com

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