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

Biogen Idec Research Limited0 个研究点目标入组 172 人开始时间: 2022年2月5日最近更新: 2022年5月30日

适应症

试验速览

阶段: 1 期
状态: 进行中（未招募）
发起方: Biogen Idec Research Limited
入组人数: 172

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 clinical trial of medicinal product

入排标准

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 = 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
•- Currently on nusinersen treatment at the time of Screening, with the first dose being at least 1 year prior to Screening
•Part C Cohort 1:
•- Participants of any age (individuals =18 years of age at Screening must be ambulatory)
•Part C Cohort 2:
•- Participants =18 years of age at Screening (can be ambulatory or nonambulatory)
•- HFMSE total score =4 points at Screening
•- RULM entry item A score =3 points at Screening
•Are the trial subjects under 18? yes
•Number of subjects for this age range: 140
•F.1.2 Adults (18-64 years) yes
•F.1.2.1 Number of subjects for this age range 32
•F.1.3 Elderly (>=65 years) no
•F.1.3.1 Number of subjects for this age range

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 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 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 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
•- Concomitant or previous administration of any SMN2-splicing modifier (excluding nusinersen) or gene therapy, either in a clinical study or as part of medical care.
•- Concurrent or previous participation in any interventional investigational study for any other drug or device within 30 days or 5 half-lives of the agent, whichever is longer, prior to Screening
•NOTE: Other protocol defined Inclusion/Exclusion criteria may apply

研究者

发起方

Biogen Idec Research Limited

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