Chinese Medicine WT for Spinocerebellar Ataxia Type 3

NCT05038306 | Completed Phase 2

• 1 other identifier: 200703
• Study Type: interventional
• Target: 33
• Locations: 1 country
• Sites: 1

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.

Conditions

Ataxia, Spinocerebellar; Insulin-Like Growth Factor I; Medicine, Chinese Traditional

Outcome Measures

Primary Outcomes (2)

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

  Insulin-like growth factor I is appropriate to observe the change for a period instead of the absolute value.

  baseline and 12 weeks, or 16 weeks and 28 weeks, 32 weeks

• Change from baseline in Neurofilament light Chain at week 12, or from week 16 to week 28

  Neurofilament light Chain is appropriate to observe the change for a period instead of the absolute value

  baseline and 12 weeks, or 16 weeks and 28 weeks, 32 weeks

Secondary Outcomes (8)

• 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 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

Study Arms (2)

Chinese Medicine

ACTIVE COMPARATOR

Drug: Chinese medicine WT

Placebo

PLACEBO COMPARATOR

Drug: Chinese medicine WT

Interventions

Chinese medicine WT DRUG

The Chinese medicine WT is thought to be neurotrophic, and its major constituent may benefit IGF-1 in animal studies, or certain healthy human group. WT is safe in clinical use in Asia.

Chinese Medicine; Placebo

Eligibility Criteria

• Age: 20 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• diagnosed as Spinocerebellar Ataxia type 3

You may not qualify if:

• \. 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

Sponsors & Collaborators

• Changhua Christian Hospital: lead

Study Sites (1)

Changhua Christian Hospital

Changhua, 500, Taiwan

Location

MeSH Terms

Conditions

Spinocerebellar Ataxias

Condition Hierarchy (Ancestors)

Cerebellar Ataxia; Cerebellar Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Spinocerebellar Degenerations; Spinal Cord Diseases; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Ataxia; Dyskinesias; Neurologic Manifestations; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Adjunct Physician

Study Record Dates

• First Submitted: May 27, 2021
• First Posted: September 9, 2021
• Study Start: May 31, 2021
• Primary Completion: September 26, 2022
• Study Completion: September 26, 2022
• Last Updated: January 31, 2023

Record last verified: 2023-01

Data Sharing

• IPD Sharing: Will not share

Locations

TW (1)