Chinese Medicine WT for Elevating IGF-1 of Patients With Spinocerebellar Ataxia Type 3 - Pilot Study
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 33
- 试验地点
- 1
- 主要终点
- Change from baseline in Insulin-like Growth Factor 1 at week 12, or from week 16 to week 28
研究概览
简要总结
Spinocerebellar ataxia type 3 (SCA3) is one of autosomal dominant hereditary ataxias. Standing imbalance, unsteady gait, dysmetria, fatigue, and depression would occur gradually. There are no effective treatment or palliative methods for patients in the present days. However, low-dose growth hormone, or its downstream product, insulin-like growth factor I (IGF-1), may deter the progress of SCA3 in transgenic mice. The main bioactive constituent among the Chinese medicine WT possesses neuroprotective function against glutamate-induced toxicity, which is one major pathology of SCA3. It promotes neurogenesis, and increases the protein expression of IGF-1 in ischemic brains of rats. Thus, we designed a randomized, double-blind trial for patients with SCA3, if WT is a possible neuroprotective medicine.
All the subjects will be recruited from Changhua Christian Hospital. Diagnosis is confirmed by gene test and magnetic resonance image by a neurologist. They will be assigned in random and double blind, prescribed with 3 grams concentrated powder of WT or placebo, twice a day, for 12 weeks. After the washout period of 4 weeks, there will be a crossover of placebo or WT for another 12 weeks. After that, another 4-week rest will be followed by the end of trial. Check items in five check points include: 1. Blood examination (serum IGF-1, Neurofilament light chain, mitochondria copy number, 8_OHdG, delta-Ct), 2.Neurological exam (Scale for the Assessment and Rating of Ataxia), 3. Questionnaires (Modified Fatigue Impact Scale, Epworth Sleepiness Scale), 4. Handgrip strength test (which is correlated to IGF-1 value in elderly), and 5. serum metabolites, . All the data will be disclosed after the end of trial. Paired-T test or Wilcoxon Ranked Sign Test will be operated in SPSS.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Crossover
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 20 Years 至 80 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •diagnosed as Spinocerebellar Ataxia type 3
排除标准
- •using other Chinese medicine or herbal medicine
- •allergic to Chinese medicine or herbal medicine
- •pregnancy or breast feeding
- •with other major diseases, such as cancer, stroke, heart failure, or renal failure
研究组 & 干预措施
Chinese Medicine
干预措施: Chinese medicine WT (Drug)
Placebo
干预措施: Chinese medicine WT (Drug)
结局指标
主要结局
Change from baseline in Insulin-like Growth Factor 1 at week 12, or from week 16 to week 28
时间窗: baseline and 12 weeks, or 16 weeks and 28 weeks, 32 weeks
Insulin-like growth factor I is appropriate to observe the change for a period instead of the absolute value.
Change from baseline in Neurofilament light Chain at week 12, or from week 16 to week 28
时间窗: baseline and 12 weeks, or 16 weeks and 28 weeks, 32 weeks
Neurofilament light Chain is appropriate to observe the change for a period instead of the absolute value
次要结局
- Change from baseline in Epworth Sleepiness Scale (ESS) at week 12, or from week16 to week 28(baseline and 12 weeks, or 16 weeks and 28 weeks)
- Change from baseline in Hand-grip strength test (HST) at week 12, or from week16 to week 28(baseline and 12 weeks, or 16 weeks and 28 weeks)
- Change from baseline in mitochondria DNA (mtDNA) at week 12, or from week16 to week 28(baseline and 12 weeks, or 16 weeks and 28 weeks)
- Change from baseline in scale for the assessment and rating of ataxia(SARA) at week 12, or from week 16 to week 28(baseline and 12 weeks, or 16 weeks and 28 weeks)
- Change from baseline in Modified Fatigue Impact Scale (MFIS) at week 12, or from week 16 to week 28(baseline and 12 weeks, or 16 weeks and 28 weeks)
- Change from baseline in 8_OHdG at week 12, or from week16 to week 28(baseline and 12 weeks, or 16 weeks and 28 weeks)
- Change from baseline in △Ct at week 12, or from week16 to week 28(baseline and 12 weeks, or 16 weeks and 28 weeks)
- Change from baseline in serum metabolites at week 12, or from week 16 to week 28(baseline and 12 weeks, or 16 weeks and 28 weeks)
研究者
Chiu Chung Min
Adjunct Physician
Changhua Christian Hospital