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Chinese Medicine WT for Spinocerebellar Ataxia Type 3

Primary Purpose

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

Status

Completed

Phase

Phase 2

Locations

Taiwan

Study Type

Interventional

Intervention

Chinese medicine WT

About this trial

This is an interventional treatment trial for Ataxia, Spinocerebellar

Eligibility Criteria

20 Years - 80 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

Changhua Christian Hospital

Arms of the Study

Arm 1

Arm 2

Arm Type

Active Comparator

Placebo Comparator

Arm Label

Chinese Medicine

Placebo

Arm Description

Outcomes

Primary Outcome Measures

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.

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

Secondary Outcome Measures

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

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

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

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

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

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

mitochondrial DNA HST is appropriate to observe the change for a period instead of the absolute value

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

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

△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

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.

Full Information

NCT ID

NCT05038306

First Posted

May 27, 2021

Last Updated

January 28, 2023

Sponsor

Changhua Christian Hospital

1. Study Identification

Unique Protocol Identification Number

NCT05038306

Brief Title

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

2. Study Status

Record Verification Date

January 2023

Overall Recruitment Status

Completed

Study Start Date

May 31, 2021 (Actual)

Primary Completion Date

September 26, 2022 (Actual)

Study Completion Date

September 26, 2022 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Principal Investigator

Name of the Sponsor

Changhua Christian Hospital

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Studies a U.S. FDA-regulated Device Product

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

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

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 2

Interventional Study Model

Crossover Assignment

Masking

ParticipantCare ProviderInvestigatorOutcomes Assessor

Allocation

Randomized

Enrollment

33 (Actual)

8. Arms, Groups, and Interventions

Arm Title

Chinese Medicine

Arm Type

Active Comparator

Arm Title

Placebo

Arm Type

Placebo Comparator

Intervention Type

Drug

Intervention Name(s)

Chinese medicine WT

Intervention Description

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 Outcome Measure Information:

Title

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

Description

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

Time Frame

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

Title

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

Description

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

Time Frame

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

Secondary Outcome Measure Information:

Title

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

Description

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

Time Frame