NCT02240355终止1 期
A Multicenter, Randomized, Double Blind, Placebo Controlled, Multiple Dose Study to Investigate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of RO6885247 Following 12 Weeks of Treatment in Adult and Pediatric Patients With Spinal Muscular Atrophy (MOONFISH).
适应症
干预措施
相关药物
试验速览
- 阶段
- 1 期
- 状态
- 终止
- 入组人数
- 9
- 试验地点
- 9
- 主要终点
- Safety: Incidence of adverse events (AEs)
研究概览
简要总结
This multicenter, randomized, double-blind, 12-week, placebo-controlled multiple dose study will investigate the safety and tolerability of RO6885247 in adult and pediatric patients with spinal muscular atrophy (SMA).
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- Double (Participant, Investigator)
入排标准
- 年龄范围
- — 至 55 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Males and females, aged 2 to 55 years inclusive or below 7 months inclusive
- •Confirmed diagnosis of 5q-autosomal recessive SMA (Types 1 to 3), for patients aged 7 months or below clinical symptoms attributable to type 1 SMA and 2 SMN2 copies
- •Able and willing to provide informed consent and to comply with the study protocol. Alternatively, a legally authorized representative must be able to consent for the patient and assent must be given by the subject wherever possible.
- •Female patients of childbearing potential and male patients with a female partner of childbearing potential must agree with the required contraceptive methods as defined per protocol.
- •For patients aged 7 months or below, Gestational age of 37 to 42 weeks and not considered small for gestational age at birth
排除标准
- •Concomitant or previous participation in any investigational drug or device study within 90 days prior to screening
- •Concomitant or previous participation in a SMN2-targeting antisense oligonucleotide study within 12 months prior to screening
- •Concomitant or previous participation at any time in a gene therapy study
- •For patients aged 2-55 years, hospitalization for pulmonary event within the last 2 months or planned at the time of screening
- •Surgery for scoliosis in the last 6 months from screening or planned within 6 months from screening
- •Unstable gastrointestinal, renal, hepatic, endocrine or cardiovascular system disease
- •Clinically relevant ECG abnormalities at screening or baseline; personal or family history (first degree relatives) of congenital long QT syndrome
- •Clinically significant abnormalities in laboratory test results at screening
- •Any concomitant disease or condition that could interfere with the conduct of the study, or pose an unacceptable risk to the subject in this study
- •Use of prohibited medications as per protocol within 90 days prior to randomization. Patients who are on inhaled corticosteroids, administered either through a nebulizer or an inhaler, are allowed.
- •Recently initiated treatment (within <6 months prior to randomization) with oral salbutamol or another beta2-adrenergic agonist taken orally is not allowed. Patients who have been on oral salbutamol (or another beta2-adrenergic agonist) for at least 6 months before randomization are allowed. Use of inhaled beta2-adrenergic agonists is allowed.
- •For patients aged 7 months or below, patients requiring invasive ventilation or tracheostomy, presence of non-SMA related morbidities
研究组 & 干预措施
Part 1
Experimental
Up to 2 cohorts of patients, within each cohort patients will receive either RO6885247 or placebo once daily for 12 weeks
干预措施: RO6885247 (Drug)
Part 1
Experimental
Up to 2 cohorts of patients, within each cohort patients will receive either RO6885247 or placebo once daily for 12 weeks
干预措施: placebo (Drug)
Part 2
Experimental
1 cohort of patients, within each cohort patients will receive either RO6885247 or placebo once daily for 12 weeks
干预措施: RO6885247 (Drug)
Part 2
Experimental
1 cohort of patients, within each cohort patients will receive either RO6885247 or placebo once daily for 12 weeks
干预措施: placebo (Drug)
Part 3
Experimental
1 cohort of patients, within each cohort patients will receive RO6885247 once daily for 12 weeks or 20 weeks
干预措施: RO6885247 (Drug)
结局指标
主要结局
Safety: Incidence of adverse events (AEs)
时间窗: Up to 20 weeks
次要结局
- Pharmacodynamics: SMN protein levels in blood(Up to 20 weeks)
- Pharmacokinetics: RO6885247 plasma concentrations(Up to 16 weeks)
- Pharmacodynamics: In vivo splicing modification of SMN2 mRNA in blood(Up to 20 weeks)
- Effect of RO6885247 on muscle electrophysiology, as assessed by Compound Muscle Action Potential (CMAP)(Up to 20 weeks)
- Effect of RO6885247 on Electrical Impedance Myography(Up to 20 weeks)
- Pharmacokinetics: RO6885247 exposure, area under the concentration-time curve (AUC-tau, over the 24-hour dosing interval)(Up to 12 weeks)
研究者
研究点 (9)
Loading locations...
相似试验
已完成
2 期
A 12-Week Study in Adult Subjects With AsthmaAsthmaNCT01516086Dey498
终止
1 期
Safety Study of AMG 557 in Subjects With Subacute Cutaneous Lupus ErythematosusCutaneous LupusLupusNCT01389895Amgen1
已完成
1 期
Safety Study of AMG 557 in Subjects With Lupus ArthritisLupus Arthritis, Systemic Lupus ErythematosusNCT01683695Amgen20
已完成
1 期
ALK33BUP-201: Safety and Tolerability Study of ALKS 33-BUP (ALKS 5461) Administration in Subjects With MDDMajor Depressive DisorderNCT01381107Alkermes, Inc.32
终止
2 期
Study of RO6807952 in Patients With Diabetes Mellitus Type 2Diabetes Mellitus Type 2NCT01516476Hoffmann-La Roche2