Escalating Dose and Randomized, Controlled Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy - 232SM203 (Devote)

Biogen Idec Research Limited0 个研究点目标入组 4 人开始时间: 待定最近更新: 2024年4月15日

适应症

试验速览

暂无简介。

•- Genetic documentation of 5q SMA (homozygous gene deletion, mutation, or
•compound heterozygote)
•- Participants with SMA symptom onset <= 6 months (<= 180 days) of age (infantile
•onset) should have age > 1 week to <= 7 months (<= 210 days) at the time of
•informed consent
•- Participants with SMA symptom onset > 6 months (> 180 days) of age (later
•- Age 2 to < 10 years at the time of informed consent
•- Can sit independently but has never had the ability to walk independently
•- HFMSE score >= 10 and <= 54 at Screening

•- Presence of an untreated or inadequately treated active infection requiring
•systemic antiviral or antimicrobial therapy at any time during the Screening
•- Presence of an implanted shunt for the drainage of CSF or of an implanted
•central nervous system (CNS) catheter
•- Hospitalization for surgery, pulmonary event, or nutritional support within 2
•months prior to Screening or planned within 12 months after the participant's
•- Treatment with an investigational drug including but not limited to the
•treatment of SMA, biological agent, or device within 30 days or 5 half-lives of
•the agent, whichever is longer, prior to Screening or anytime during the study;
•any prior or current treatment with any survival motor neuron-2 (SMN2)-
•splicing modifier or gene therapy; or prior antisense oligonucleotide
•treatment, or cell transplantation
•- Participants with SMA symptom onset > 6 months (> 180 days) of age (later
•- Respiratory insufficiency, defined by the medical necessity for invasive or
•noninvasive ventilation for > 6 hours during a 24-hour period, at Screening
•- Medical necessity for a gastric feeding tube
•- Participants with SMA symptom onset <= 6 months (<= 180 days) of age (infantile
•onset): Signs or symptoms of SMA present at birth or within the first week
•after birth

发起方