This study is looking at how a drug called apitegromab works in children under 2 years old with Spinal Muscular Atrophy (SMA). Apitegromab is given through an IV every four weeks. Participants in this study will also be receiving standard SMA treatments like nusinersen or risdiplam. Researchers want to understand how apitegromab moves through the body, what effects it has, and if it helps improve motor skills. To join, your child must be under 2 years old, have a confirmed diagnosis of 5q autosomal recessive SMA, and have received or be receiving an approved SMA treatment. The study aims to enroll 52 participants, but the current recruitment status is unclear.

Study design

This is a double-blind, Phase 2 study, meaning neither you nor the study team will know if your child is receiving the active drug or a placebo. It plans to enroll 52 participants.

What's involved

Not specified in the trial record.

Compensation

Not stated in the trial record.

Follow-up

The study will evaluate outcomes for apitegromab for up to 52 weeks and motor function for up to 48 weeks.

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.