Chinese Medicine WT for Spinocerebellar Ataxia Type 3

Phase 2

Completed

• Conditions: Ataxia, Spinocerebellar; Insulin-Like Growth Factor I; Medicine, Chinese Traditional
• Interventions: Drug: Chinese medicine WT

• Registration Number: NCT05038306
• Lead Sponsor: Changhua Christian Hospital

Brief Summary

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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2021-05-27
• Study Start: 2021-05-31
• Study First Submitted QC: 2021-09-05
• Study First Posted: 2021-09-09
• Primary Completion: 2022-09-26
• Study Completion: 2022-09-26
• Status Verified: 2023-01
• Last Update Submitted: 2023-01-28
• Last Update Posted: 2023-01-31

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 33

Inclusion Criteria

• diagnosed as Spinocerebellar Ataxia type 3

Exclusion Criteria

• 1. using other Chinese medicine or herbal medicine 2. allergic to Chinese medicine or herbal medicine 3. pregnancy or breast feeding 4. with other major diseases, such as cancer, stroke, heart failure, or renal failure

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: CROSSOVER

Arm && Interventions

Group	Intervention	Description
Placebo	Chinese medicine WT	-
Chinese Medicine	Chinese medicine WT	-

Primary Outcome Measures

Name	Time	Method
Change from baseline in Insulin-like Growth Factor 1 at week 12, or from week 16 to week 28	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	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

Secondary Outcome Measures

Name	Time	Method
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	scores of ESS are from 0 to 24, with higher scores means worse outcome
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	HST is appropriate to observe the change for a period instead of the absolute value
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	mitochondrial DNA HST is appropriate to observe the change for a period instead of the absolute value
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	scores of SARA are from 0 to 40, with higher scores means worse outcome
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	scores of MFIS are from 0 to 84, with higher scores means worse outcome
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	8_OHdG is appropriate to observe the change for a period instead of the absolute value
Change from baseline in △Ct at week 12, or from week16 to week 28	baseline and 12 weeks, or 16 weeks and 28 weeks	△Ct is appropriate to observe the change for a period instead of the absolute value
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	serum metabolites is appropriate to observe the change for a period instead of the absolute value. Till now, there was no sufficient data about the metabolites of Chinese WT, so we would like to measure the difference of metabolites before and after taking the Chinese medicine WT.

Trial Locations

Locations (1)

Changhua Christian Hospital; 🇨🇳 Changhua, Taiwan