Phase 2 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:

• You must be under 2 years old at the time of signing up for the trial
• Your baby must have been born at 35 weeks of pregnancy or later, and weighed at least 2.0 kg (about 4.4 lbs) at birth
• Your child must have a confirmed diagnosis of Spinal Muscular Atrophy (SMA) caused by a change in the 5q gene (a specific inherited form of SMA)
• Your child must have a confirmed copy or copies of a gene called SMN2
• Your child must have already been treated with an approved SMA gene therapy (onasemnogene abeparvovec-xioi), or must currently be receiving an approved SMA medication (nusinersen or risdiplam)
• Your child's weight must be within a healthy range for their age (at or above the 1st percentile on World Health Organization growth charts) at the screening visit
• Your child must be showing delays in movement and physical development for their age due to SMA, as judged by the study doctor, or must score below 55 on a specific movement ability test (confirm with trial site)

Who may not be able to join:

• Children whose nutrition is not expected to remain stable throughout the study, or who rely mainly on a feeding tube inserted into the stomach for most of their feeds
• Children who have serious bone or joint problems — such as severe curvature of the spine or severe stiffness in the joints — or who have had, or are expected to need, major bone or joint surgery (including spine or hip surgery) within 6 months before screening or during the study
• Children who have any other physical limitations (for example, needing a cast on their body) that would stop them from completing the movement and physical ability tests required throughout the study

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