A Long-term Follow-up Study of Patients in the Clinical Trials for Spinal Muscular Atrophy Receiving AVXS-101
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 入组人数
- 22
- 试验地点
- 6
- 主要终点
- Change from baseline in height measurements
研究概览
简要总结
To collect long-term follow-up safety and efficacy data in patients with spinal muscular atrophy (SMA) who were treated with AVXS-101 (also known as OAV101) in a Novartis Pharma AG -sponsored clinical trial, including but not limited to AVXS-101-CL-102 (Phase 1), AVXS-101-CL302 (Phase 3), AVXS-101-CL-303 (Phase 3), AVXS-101-CL-304 (Phase 3) or AVXS-101-CL-306 (Phase 3)
研究设计
- 分配方式
- Not Applicable
- 主要目的
- Long-term Follow-up Study without IMP - observational phase
- 盲法
- None
入排标准
- 年龄范围
- 0 years 至 64 years（18-64 Years, 0-17 Years）
- 接受健康志愿者
- 是
入选标准
- •Any patient with SMA who received AVXS-101 gene replacement therapy in a Novartis Pharma AG -sponsored clinical study
- •Patient/parent/legal guardian willing and able to complete the informed consent process and comply with study procedures and visit schedule
排除标准
- •Patient/parent/legal guardian unable or unwilling to participate in the long-term follow-up safety study
结局指标
主要结局
Change from baseline in height measurements
Change from baseline in height measurements
Number of participants who reach developmental milestones
Number of participants who reach developmental milestones
Change from baseline in Hammersmith Functional Motor Scale - Expanded (HFMSE) score
Change from baseline in Hammersmith Functional Motor Scale - Expanded (HFMSE) score
Change from baseline in Revised Upper Limb Module (RULM)
Change from baseline in Revised Upper Limb Module (RULM)
Change from baseline in Bayley Scales of Infant and Toddler Development (Bayley-III)
Change from baseline in Bayley Scales of Infant and Toddler Development (Bayley-III)
Change from baseline in Cogstate Computerized Cognitive Battery
Change from baseline in Cogstate Computerized Cognitive Battery
Change from baseline in Clinical Evaluation of Language Fundamentals (CELF-5)
Change from baseline in Clinical Evaluation of Language Fundamentals (CELF-5)
Change from baseline in Assessment of Caregiver Experience with Neuromuscular Disease (ACEND)
Change from baseline in Assessment of Caregiver Experience with Neuromuscular Disease (ACEND)
Number of participants who experience a clinically significant change from baseline in pulmonary assessment results and require ventilatory support
Number of participants who experience a clinically significant change from baseline in pulmonary assessment results and require ventilatory support
Number of participants who experience swallowing dysfunction and require nutritional support
Number of participants who experience swallowing dysfunction and require nutritional support
Number of participants who experience a clinically significant change from baseline in physical examination findings
Number of participants who experience a clinically significant change from baseline in physical examination findings
Number of participants who experience a clinically significant change from baseline in vital signs measurements
Number of participants who experience a clinically significant change from baseline in vital signs measurements
Change from baseline in weight measurements
Change from baseline in weight measurements
Number of participants who experience a clinically significant change from baseline in clinical laboratory assessments
Number of participants who experience a clinically significant change from baseline in clinical laboratory assessments
Number of participants who experience a clinically significant change from baseline in cardiac assessments
Number of participants who experience a clinically significant change from baseline in cardiac assessments
Number of participants who experience a clinically significant change from baseline in observational phase questionnaire results
Number of participants who experience a clinically significant change from baseline in observational phase questionnaire results
Concomitant Medications
Concomitant Medications
Any other treatment for SMA for the observational phase
Any other treatment for SMA for the observational phase
Number of participants who experience at least one serious adverse event (SAE)
Number of participants who experience at least one serious adverse event (SAE)
Number of participants who experience at least one adverse event of special interest (AESI)
Number of participants who experience at least one adverse event of special interest (AESI)
次要结局
未报告次要终点
研究者
Andre Dorochevsky
Scientific
Novartis Pharma AG