Long Term Safety Study of Amifampridine Phosphate in Ambulatory Patients With Spinal Muscular Atrophy (SMA) Type 3
试验速览
- 阶段
- 2 期
- 状态
- 终止
- 入组人数
- 13
- 试验地点
- 1
- 主要终点
- Long-term Safety and Tolerability of Amifampridine
研究概览
简要总结
A long term safety study of amifampridine phosphate in patients with spinal muscular atrophy (SMA) Type 3.
详细描述
This open-label outpatient extension study was designed to evaluate the long-term safety and tolerability of amifampridine in patients diagnosed with SMA Type 3. In addition, the evaluation of the effects of amifampridine on the QoL was performed. The study was planned to enroll those patients who had completed the SMA-001 study and after all final evaluations for that study had been completed, or those who demonstrated benefit after completing the dose titration period but failed to meet the randomization criteria on Day 0 of SMA-001.
The duration of participation for each patient was expected to be at least 12 months as patients could continue in the study until amifampridine was approved by Regulatory Agencies or the clinical development of amifampridine was terminated for this indication. In addition to amifampridine, patients continued to receive previous concomitant medications, as needed.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 6 Years 至 50 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Individuals eligible to participate in this study must meet all the following inclusion criteria:
- •Participated in the SMA-001 study
- •Willing and able to provide written informed consent after the nature of the study has been explained and before the start of any research-related procedures.
- •Female patients of childbearing potential must have a negative pregnancy test (urine human chorionic gonadotropin [HCG] at the end of SMA-001 study); and must practice an effective, reliable contraceptive regimen during the study and for up to 30 days following discontinuation of treatment.
- •Ability to participate in the study based on overall health of the patient and disease prognosis, as applicable, in the opinion of the Investigator; and able to comply with all requirements of the protocol, including completion of study questionnaires.
排除标准
- •Individuals who met any of the exclusion criteria in the original protocol or those listed below are not eligible to participate in the study:
- •Epilepsy and currently on medication.
- •Uncontrolled asthma.
- •Concomitant use with sultopride.
- •Concomitant use with medicinal products with a narrow therapeutic window.
- •Concomitant use with medicinal products with a known to cause QTc prolongation.
- •Clinically significant abnormalities in 12 lead ECG, in the opinion of the Investigator.
- •Subjects with congenital QT syndromes.
- •Breastfeeding or pregnant at Screening or planning to become pregnant at any time during the study.
- •Intolerable amifampridine-related side effects
- •Treatment with an investigational drug (other than amifampridine) or device while participating in this study.
- •Any medical condition that, in the opinion of the Investigator, might interfere with the patient's participation in the study, poses an added risk for the patient, or confound the assessment of the patient.
- •History of drug allergy to any pyridine-containing substances or any amifampridine excipient(s).
研究组 & 干预措施
amifampridine phosphate
The dose of amifampridine was based on optimal neuromuscular benefit determined from the Run-in Period from SMA-001 or could be modified as the discretion of the Investigator. The maximum single dose was 20 mg. The dose range for patients 6 to 16 years of age was 15 to 60 mg, and for those 17 years and older the range was from 30 to 80 mg daily.
干预措施: Amifampridine Phosphate 10 MG Oral Tablet (Drug)
结局指标
主要结局
Long-term Safety and Tolerability of Amifampridine
时间窗: 18 months
Number of subjects with treatment emergent adverse events (TEAE).
次要结局
- To Assess the Clinical Efficacy of Amifampridine Phosphate Over Time in Patients With SMA Type 3 Based on Changes in Quality of Life (QoL).(Screening to end of study.)