NCT04706013招募中3 期
Study of Pyridoxal 5'-Phosphate for the Treatment of Patients With PNPO Deficiency
适应症
干预措施
相关药物
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 发起方
- Medicure
- 入组人数
- 15
- 试验地点
- 7
- 主要终点
- Overall survival time (time to death), including incidence of death at 12 months
研究概览
简要总结
The proposed clinical study is intended to evaluate oral P5P for the treatment of patients confirmed to have Pyridox(am)ine 5'-Phosphate Oxidase (PNPO) deficiency via genetic analysis. There is an unmet clinical need for pharmaceutical grade P5P, as to date none has been made commercially available. Patients will receive pharmaceutical grade P5P according to their normal oral P5P dosing regimen, as previously established by their physicians.
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
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients with confirmed PNPO deficiency via genetic analysis, whose seizures are typically controlled on P5P (oral) therapy.
- •a. Typically controlled is defined as receiving multiple doses of P5P daily to control seizures. Receiving P5P for a minimum of 30 days.
- •Male and/or female patients.
- •Aged ≥2 years
- •(3) Patients with previous failed treatment on pyridoxine are eligible for the study (patient should be off pyridoxine for at least 24 hours).
- •(4) Written informed consent (by parent or guardian if under the age of 18).
排除标准
- •The patient has any condition or abnormality which may, in the opinion of the Investigator, compromise the safety of the patient, or influence their ability to comply with study procedures.
- •Known or suspected allergy to the trial drug or the relevant drugs given in the trial.
- •Involvement in a clinical research study within 4 weeks prior to screening and/or prior enrollment in the study. Participation in observational registry studies is permitted.
研究组 & 干预措施
Single Arm Active
Experimental
Pyridoxal 5'-Phosphate
干预措施: Pyridoxal Phosphate (Drug)
结局指标
主要结局
Overall survival time (time to death), including incidence of death at 12 months
时间窗: 12 months
The study group will be compared to a historical control group (without active treatment) for overall survival by using the method of survival analysis
次要结局
- Frequency of seizures (including but not limited to status epilepticus)(up to 12 months)
研究者
研究点 (7)
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