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:

• You have been diagnosed with Type 2 Spinal Muscular Atrophy (SMA) caused by a change in the 5q gene, confirmed by genetic testing and symptoms.
• You have been regularly receiving the medication nusinersen for more than one year before the screening visit.
• You have not taken the medication risdiplam in the past 2 months, and do not plan to take it within 12 months of joining the trial.
• You are able to sit up on your own but cannot walk on your own, and you score 10 points or higher on a standard motor function test called the HFMSE.
• You (and/or your legal guardian, if applicable) understand what the trial involves, are willing to follow its requirements, and agree to take part by signing a consent form.

Who may not be able to join:

• You have a high level of antibodies in your blood that could interfere with the gene therapy being tested (anti-AAV9 neutralizing antibody level above 1:50).
• You have received nusinersen within the 2 months before enrolling in the trial.
• You have certain other health conditions that could affect the study results or your safety, such as organ problems, active infections, immune system disorders, serious heart or brain conditions, digestive disorders, diabetes, epilepsy, a history of seizures or meningitis, or a family history of psychiatric illness (confirm with trial site).
• You have significant liver problems, shown by certain blood test levels being too high (ALT or AST more than 3 times the normal limit, or total bilirubin more than 1.5 times the normal limit).
• You have health conditions that would make it unsafe for you to take steroid medications, such as severe high blood pressure, diabetes, active infections, glaucoma, weak bones, stomach ulcers, or tuberculosis.
• You have a condition that makes it unsafe to have a lumbar puncture (spinal tap) or an injection into the spinal canal.
• You have a physical condition that could affect how your movement and motor skills are measured, such as severe spinal curvature, severe joint stiffness, very weak bones, a history of fractures, or planned spinal surgery during the trial.
• You have tested positive for Hepatitis B, Hepatitis C, HIV, or syphilis.
• You have received any vaccines within 2 weeks before receiving the trial treatment.
• You have previously had gene therapy, or have taken part in another clinical trial within the past 3 months.
• The trial doctor has determined for any other reason that the trial is not right for you.

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