Clinical trial NCT05038306: Chinese Medicine WT for Spinocerebellar Ataxia Type 3
Official title: Chinese Medicine WT for Elevating IGF-1 of Patients With Spinocerebellar Ataxia Type 3 - Pilot Study
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Ataxia, Spinocerebellar
Insulin-Like Growth Factor I
Medicine, Chinese Traditional
Lead sponsor: Changhua Christian Hospital
Sponsor class: OTHER
First posted: 2021-09-09
Enrollment: 33
Sex: ALL
Minimum age (years): 20
Maximum age (years): 80
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: CROSSOVER
Primary purpose: TREATMENT

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.


Eligibility criteria: 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

Primary outcomes:
measure: Change from baseline in Insulin-like Growth Factor 1 at week 12, or from week 16 to week 28
timeFrame: baseline and 12 weeks, or 16 weeks and 28 weeks, 32 weeks
measure: Change from baseline in Neurofilament light Chain at week 12, or from week 16 to week 28
timeFrame: baseline and 12 weeks, or 16 weeks and 28 weeks, 32 weeks
Secondary outcomes:
measure: Change from baseline in scale for the assessment and rating of ataxia(SARA) at week 12, or from week 16 to week 28
timeFrame: baseline and 12 weeks, or 16 weeks and 28 weeks
measure: Change from baseline in Modified Fatigue Impact Scale (MFIS) at week 12, or from week 16 to week 28
timeFrame: baseline and 12 weeks, or 16 weeks and 28 weeks
measure: Change from baseline in Epworth Sleepiness Scale (ESS) at week 12, or from week16 to week 28
timeFrame: baseline and 12 weeks, or 16 weeks and 28 weeks
measure: Change from baseline in Hand-grip strength test (HST) at week 12, or from week16 to week 28
timeFrame: baseline and 12 weeks, or 16 weeks and 28 weeks
measure: Change from baseline in mitochondria DNA (mtDNA) at week 12, or from week16 to week 28
timeFrame: baseline and 12 weeks, or 16 weeks and 28 weeks
measure: Change from baseline in 8_OHdG at week 12, or from week16 to week 28
timeFrame: baseline and 12 weeks, or 16 weeks and 28 weeks
measure: Change from baseline in △Ct at week 12, or from week16 to week 28
timeFrame: baseline and 12 weeks, or 16 weeks and 28 weeks
measure: Change from baseline in serum metabolites at week 12, or from week 16 to week 28
timeFrame: baseline and 12 weeks, or 16 weeks and 28 weeks
Other outcomes:
Locations:
city: Changhua
country: Taiwan
facility: Changhua Christian Hospital
Study officials:
References: