Phenylbutyrate in SCA3: a Double-blind, Placebo-controlled Study to Determine Safety and Efficacy of Sodium Phenylbutyrate in Patients With SCA3
试验速览
- 阶段
- 2 期
- 状态
- 撤回
- 试验地点
- 1
- 主要终点
- Safety and tolerability of phenylbutyrate in patients with SCA3
研究概览
简要总结
DESIGN: Pilot, Phase II, double-blind, placebo-controlled study
JUSTIFICATION: In the literature one does not find a pharmacological treatment that changes the natural history of Spinocerebellar ataxtia type 3 (SCA3). Patients with this disease invariably become dependent.
OBJECTIVES I. To determine safety and tolerability of phenylbutyrate in patients with SCA3.
II. To provide early subsidies on the efficacy of phenylbutyrate in SCA3.
DURATION: 12 months of a double-blind study.
PLACE OF REALIZATION: Hospital de Clínicas de Porto Alegre, Brazil.
NUMBER OF PATIENTS: 20 patients.
CONCOMITANT MEDICATIONS: There are no concomitant medications that are prohibited unless they affect safety parameters of this study (hemogram and platelets; fasting serum glucose, AST, ALT, Gamma-GT, Bilirubins, Prothrombin time, Creatinine, Urea, Na, K, chlorides and arterial gasometry; electrocardiogram and echocardiogram).
MEDICATIONS UNDER INVESTIGATION: Powdered sodium phenylbutyrate in sachets containing each 3g. At the start of the study, the dose will be 15g/day (five sachets) and may be reduced in case of mild adverse events.
OUTCOMES Primary safety outcome: The number of adverse events, interruptions and dose reductions in the two groups (cases and controls).
Efficacy outcomes: Efficacy outcomes are the following scores in both groups: NESSCA, SARA, Barthel, BDI, and WHOQol.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 16 Years 至 80 Years（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •All patients shall have their molecular diagnosis confirmed and expanded polyglutamine tract (CAG) measures already determined.
- •Patients still able to walk with until 8 years of disease duration, and
- •Patients aged 16 years or over will be invited to participate in the study.
排除标准
- •they show electrocardiogram ou echocardiographic alterations suggestive of heart insufficiency at baseline;
- •their serum creatinine levels are higher than 1.2 mg/dL, with the confirmation of renal insufficiency due to the rate of glomerular filtration;
- •they show a history of hypersensibility to sodium phenylbutyrate, and if
- •they (men and women) do not agree to use a reliable contraceptive method during the entire study period and for three months after its end.
研究组 & 干预措施
Placebo
Placebo
干预措施: Placebo (Drug)
Sodium phenylbutyrate
Active drug
干预措施: Sodium Phenylbutyrate (Drug)
结局指标
主要结局
Safety and tolerability of phenylbutyrate in patients with SCA3
时间窗: 6 months - 12 months
The number of adverse events, interruptions and dose reductions in the two groups (cases and controls).
次要结局
- Efficacy of phenylbutyrate in SCA3 on neurological dysfunction and quality of life(6 months - 12 months)