ASsessing The REAl-world Safety & Effectiveness of Spinal Muscular Atrophy Participants Treated With Intrathecal Onasemnogene Abeparvovec-brve (OAV101B) (ITVISMA®): A U.S. Pragmatic Multicenter Study (STREAM)

Korábbi kezelés

• An uncontrolled acute illness, active infection, febrile illness or acute medical condition, within 30 days prior to dosing
• Inability to tolerate corticosteroids administered by mouth or gastrostomy tube

Más betegség

• A participant will be excluded if any of the following conditions apply:

Egyéb

• Contraindication for lumbar puncture as determined by the investigator (e.g., spinal anatomy that precludes safe lumbar puncture)
• Current participation in another interventional clinical trial that would interfere with the objectives or endpoints of STREAM. Note: participation in observational cohort studies or non-interventional studies in which the participant does not receive treatment or undergo procedures which may compromise this study data integrity may be allowed following Sponsor approval
• Planned relocation or any circumstance that is likely to prevent completion of the minimum 12-month follow-up.
  • Other protocol inclusion, exclusion criteria may apply.

• A participant will be enrolled in STREAM only if all the following conditions are met:
• The participant has a genetically confirmed diagnosis of SMA (biallelic SMN1 deletion or mutation).
• He or she is ≥ 2 years of age on the day of the intrathecal injection.
• The treating Investigator intends to administer within the current episode of care, a single dose of ITVISMA® (1.2 × 10¹⁴ vg) in routine U.S. practice after written informed consent obtained.
• One of the following functional categories applies at screening:
  • independently ambulatory child/adolescent 2 - <18 years; or
  • independently ambulatory adult (≥ 18 years).
  • non-ambulatory (includes walkers with assistance) adult > 18 years with entry level RULM at baseline
• At least 1 year of pre-treatment medical history, including motor-function documentation, can be retrieved from the site electronic record.