A Phase 2, Double-Blind Study to Evaluate the Pharmacokinetics, Pharmacodynamics, Efficacy, and Safety of Apitegromab in Subjects Under 2 Years Old With Spinal Muscular Atrophy

What this Trial Is About

Researchers are evaluating apitegromab in children younger than 2 years old who have 5q autosomal recessive Spinal Muscular Atrophy (SMA) and delayed motor milestones linked to SMA or a Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP-INTEND) score below 55. This Phase 2, double-blind study aims to assess how the drug acts in the body (pharmacokinetics and pharmacodynamics), its effects on motor function, and its safety and tolerability. Participants will receive apitegromab through intravenous (IV) infusion every 4 weeks during a 48-week treatment period. All enrolled children must have been treated with an approved SMN1-targeted therapy like onasemnogene abeparvovec-xioi or be currently receiving an approved SMN2-targeted therapy such as nusinersen or risdiplam. The study compares low and high doses of apitegromab alongside these standard SMN therapies. During the study, researchers will monitor apitegromab levels in the body, its biological effects, and motor function improvements over 48 to 52 weeks. Safety and tolerability will also be assessed throughout this time. The trial involves regular assessments including motor function tests and safety evaluations to track progress and any adverse effects. Participation lasts approximately one year, with detailed follow-up to understand the treatment's impact.

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