Phase 4 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 joining the trial
• Your child must have a confirmed diagnosis of spinal muscular atrophy (SMA) caused by a specific genetic change affecting the SMN1 gene
• Your child must have exactly two copies of the SMN2 gene, confirmed by a lab test
• Your child must have already received a gene therapy treatment called onasemnogene abeparvovec (also known as Zolgensma) for SMA, whether given before or after symptoms appeared
• The gene therapy must have been given at least 13 weeks ago, but no more than 30 weeks ago, before joining this trial
• If your child received a medication called risdiplam before the gene therapy, they must have taken it for no more than 3 weeks, and stopped it one day before receiving the gene therapy
• In the doctor's opinion, your child must not have had any significant loss of abilities or function since receiving the gene therapy

Who may not be able to join:

• Children who are currently in, or have previously been part of, another research trial
• Children with certain abnormal lab results related to standard care guidelines for the gene therapy
• Children who have received a different type of SMA treatment called an antisense oligonucleotide (for example, nusinersen/Spinraza), either currently or in the past
• Children who have used a medication known as an anti-myostatin agent at any point
• Children who need a breathing machine connected through a tube in the throat (invasive ventilation or tracheostomy)
• Children who need breathing support while awake, or whose blood oxygen levels are too low while awake (confirm with trial site)
• Children who have a feeding tube and also score zero on a specific swallowing assessment called OrSAT (confirm with trial site)
• Children who were hospitalised for a lung or breathing problem in the last 2 months, or who have a planned hospitalisation coming up
• Children who had a serious illness needing hospitalisation in the past month, or any fever-causing illness in the week before screening or up until the first study dose

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