A Phase IV Open-Label Study Evaluating the Effectiveness and Safety of Risdiplam Administered in Pediatric Patients with Spinal Muscular Atrophy who Experienced a Plateau or Decline in Function After Gene Therapy - BN44621

F. Hoffmann-La Roche AG0 个研究点目标入组 46 人开始时间: 2023年10月25日最近更新: 2024年7月22日

适应症

试验速览

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•Confirmed diagnosis of 5q-autosomal recessive SMA, including genetic confirmation of homozygous deletion or compound heterozygosity predictive of loss of function of the survival of motor neuron 1 (SMN1) telomeric gene, Confirmed presence of two survival of motor neuron 2 (SMN2) centromeric gene copies as documented through laboratory testing, Administration of onasemnogene abeparvovec pre-symptomatically or post-symptomatically (<=3 months of symptoms), Has received onasemnogene abeparvovec for SMA no less than 3 months, Parent or caregiver of patient willing to consider nasogastric, naso-jejunal, or gastrostomy tube placement during the study to maintain safe hydration, nutrition, and treatment delivery, as recommended by the investigator, In the opinion of the investigator, has demonstrated a plateau or decline in function post-gene therapy (with a duration of 6 months or less) documented by 2 individual time points in the functions as follows: - Swallowing AND - One (1) additional function/ability (respiratory, motor function, other) per appropriate expectation

•Treatment with investigational therapy prior to initiation of study treatment, Any unresolved standard-of-care laboratory abnormalities per the onasemnogene abeparvovec prescribing information, Concomitant or previous administration of a SMN2-targeting antisense oligonucleotide or SMN2 splicing modifier either in a clinical study or as part of medical care, Patients requiring invasive ventilation or tracheostomy, Any major illness requiring hospitalization within 1 month before the screening examination or any febrile illness within 1 week prior to screening and up to first dose administration, History of respiratory failure or severe pneumonia and pulmonary function has not fully recovered at the time of screening

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