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NCT Number: NCT05038306

Chinese Medicine WT for Spinocerebellar Ataxia Type 3

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.

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Key information

Conditions

Ataxia, Spinocerebellar Ataxia Brain Diseases Central Nervous System Diseases Cerebellar Ataxia Cerebellar Diseases Congenital, Hereditary, and Neonatal Diseases and Abnormalities Dyskinesias Genetic Diseases, Inborn Heredodegenerative Disorders, Nervous System Insulin-Like Growth Factor I Medicine, Chinese Traditional Nervous System Diseases Neurodegenerative Diseases Neurologic Manifestations Spinal Cord Diseases Spinocerebellar Ataxias Spinocerebellar Degenerations

Age range

20 year–80 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Changhua Christian Hospital

Changhua, 500, Taiwan

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

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.

Primary outcomes

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

    Time frame: 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.

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

    Time frame: 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 outcomes

  1. Change from baseline in scale for the assessment and rating of ataxia(SARA) at week 12, or from week 16 to week 28

    Time frame: baseline and 12 weeks, or 16 weeks and 28 weeks

    scores of SARA are from 0 to 40, with higher scores means worse outcome

  2. Change from baseline in Modified Fatigue Impact Scale (MFIS) at week 12, or from week 16 to week 28

    Time frame: baseline and 12 weeks, or 16 weeks and 28 weeks

    scores of MFIS are from 0 to 84, with higher scores means worse outcome

  3. Change from baseline in Epworth Sleepiness Scale (ESS) at week 12, or from week16 to week 28

    Time frame: baseline and 12 weeks, or 16 weeks and 28 weeks

    scores of ESS are from 0 to 24, with higher scores means worse outcome

  4. Change from baseline in Hand-grip strength test (HST) at week 12, or from week16 to week 28

    Time frame: 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

  5. Change from baseline in mitochondria DNA (mtDNA) at week 12, or from week16 to week 28

    Time frame: 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

  6. Change from baseline in 8_OHdG at week 12, or from week16 to week 28

    Time frame: 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

  7. Change from baseline in △Ct at week 12, or from week16 to week 28

    Time frame: 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

  8. Change from baseline in serum metabolites at week 12, or from week 16 to week 28

    Time frame: 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.

Sponsors and collaborators

Lead sponsor

Changhua Christian Hospital

Other

Registry information

Official study title

Chinese Medicine WT for Elevating IGF-1 of Patients With Spinocerebellar Ataxia Type 3 - Pilot Study

Important dates

Study start
2021
Primary completion
2022
Study completion
2022
First posted
Sep 9, 2021
Registry last updated
Jan 31, 2023

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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