EUCTR2019-002663-10-FR进行中（未招募）1 期
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
适应症
相关药物
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 152
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
- •- 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
- •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 12
- •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
- •- 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
- •- 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
- •- Concurrent or previous participation and/or administration of nusinersen in another clinical study
- •NOTE: Other protocol defined Inclusion/Exclusion criteria may apply
研究者
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