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

Berger Zdenek0 个研究点目标入组 152 人开始时间: 2020年12月25日最近更新: 2023年10月17日

适应症

试验速览

阶段: 2 期
状态: 进行中（未招募）
发起方
入组人数: 152

研究概览入排标准研究者记录与标识符相似试验

研究概览

简要总结

暂无简介。

研究设计

研究类型: Interventional

入排标准

年龄范围: ot applicable 至 ot applicable（—）
性别: All

入选标准

•Part A, B and C:
•- Genetic documentation of 5q SMA (homozygous gene deletion, mutation, or compound heterozygote)
•- Onset of clinical signs and symptoms consistent with SMA at > 6 months (> 180 days) of age (i.e., later-onset SMA)
•- Age 2 to <= 15 years, inclusive, at the time of informed consent
•- 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 onset):
•* 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
•- Participants >= 18 years of age at Screening must be ambulatory
•- Currently on nusinersen treatment at the time of Screening, with the first dose being at least 1 year prior to Screening
•NOTE: Other protocol defined Inclusion criteria may apply.

排除标准

•Part A, B and C:
•- Presence of an untreated or inadequately treated active infection requiring systemic antiviral or antimicrobial therapy at any time during the Screening period
•- Presence of an implanted shunt for the drainage of cerebrospinal fluid (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 first dose
•- 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
•- Treatment with an investigational drug given for 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
•- Treatment with an investigational drug given for 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 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 onset):
•* 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.
•- Concurrent or previous participation and/or administration of nusinersen in another clinical study
•NOTE: Other protocol defined Exclusion criteria may apply.

研究者

发起方

Berger Zdenek

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