EUCTR2014-002246-41-ES进行中（未招募）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) - MOONFISH
Roche Farma S.A., en nombre de F. Hoffmann-La Roche Ltd.0 个研究点目标入组 64 人开始时间: 2015年3月3日最近更新:
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 发起方
- 入组人数
- 64
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
- •- Males and females aged below 7 months (inclusive) or 2 to 55 years (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 two 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 patient 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.
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 52
- •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
- •- 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
- •- History of cell therapy
- •- Hospitalization for pulmonary event within the last 2 months or planned at the time of screening (for patients aged 2-55 years)
- •- 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 patient 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.
- •- Ascertained or presumptive hypersensitivity (e.g. anaphylactic reaction) to RO6885247 or to the constituents of its formulation
- •- Patients aged 7 months or below requiring invasive ventilation, tracheotomy, day-time non-invasive ventilation or patients with nocturnal hypoxemia; patients having had severe pneumonia and have not fully recovered their pulmonary function at the time of screening; patients with contractures or hip subluxation/dislocation at birth; patients with non-SMA related morbidities.
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