Phase 3 Spinal Muscular Atrophy Trial, Recruiting

Written by an AI model from ClinicalTrials.gov eligibility criteria and checked on a sample basis. Please confirm against the original criteria.

Who might be able to join this trial:

• The baby must be 42 days old or younger when they receive their first dose of the study drug.
• The baby must have a genetic test confirming they have Spinal Muscular Atrophy (SMA) caused by a specific change or deletion on chromosome 5.
• The baby must have either 2 or 3 copies of a specific gene called SMN2.
• The baby must have a nerve signal reading (measured by a test on the arm) at or above a certain level at the start of the trial.
• The baby must weigh at least as much as the 3rd percentile for their age, based on World Health Organization growth charts, at the time of sign-up.

Who may not be able to join:

• Babies who are already showing clear signs or symptoms of SMA, as judged by the trial doctor.
• Babies who have no reflexes at the elbow, knee, or ankle when checked by a doctor.
• Babies whose blood oxygen levels are too low (below a certain level while awake or asleep, without any extra oxygen or breathing support).
• Babies who were diagnosed with a serious breathing condition at birth that required special medication (surfactant) or a breathing machine.
• Babies for whom the spinal fluid collection procedure (needed for this trial) would carry an increased risk of complications, for any reason including blood test results.
• Babies who have already received any approved SMA treatment (such as nusinersen, onasemnogene abeparvovec-xioi, or risdiplam), a myostatin inhibitor therapy, or any other experimental SMA drug.

Important: Always verify eligibility with the trial site directly before applying.