Ataxia, SpinocerebellarPhase 2NCT05038306
Chinese Medicine WT for Spinocerebellar Ataxia Type 3
A Phase 2 study testing Chinese medicine WT in people with Ataxia, Spinocerebellar and Insulin-Like Growth Factor I, run by Changhua Christian Hospital at 1 site in Taiwan.
Trial 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. Read more about this study
- Who is it for?
- Ages 20 to 80 years
- Conditions the study is for
- Ataxia, Spinocerebellar, Insulin-Like Growth Factor I, Medicine, Chinese Traditional
- What is being tested?
- Chinese medicine WT
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 33 participants
- Where
- 1 siteTaiwan
- Study dates
- 31 May 2021to 26 Sep 2022
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 20 to 80 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- diagnosed as Spinocerebellar Ataxia type 3
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Chinese Medicine WT for Elevating IGF-1 of Patients With Spinocerebellar Ataxia Type 3 - Pilot Study
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.
- Being tested
- Chinese medicine WT
- Medical subject headings
- Spinocerebellar Ataxias
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Chinese medicine WTDrug
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.
| Study group | Role | What they receive |
|---|---|---|
| Chinese Medicine | Active comparator | Not described Receives: Drug: Chinese medicine WT |
| Placebo | Placebo comparator | Not described Receives: Drug: Chinese medicine WT |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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
Measured: 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
scores of SARA are from 0 to 40, with higher scores means worse outcome
Measured: 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
scores of MFIS are from 0 to 84, with higher scores means worse outcome
Measured: 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
scores of ESS are from 0 to 24, with higher scores means worse outcome
Measured: 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
HST is appropriate to observe the change for a period instead of the absolute value
Measured: 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
mitochondrial DNA HST is appropriate to observe the change for a period instead of the absolute value
Measured: baseline and 12 weeks, or 16 weeks and 28 weeks
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
Measured: baseline and 12 weeks, or 16 weeks and 28 weeks
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
Measured: baseline and 12 weeks, or 16 weeks and 28 weeks
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.
Measured: baseline and 12 weeks, or 16 weeks and 28 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Sep 2021
- Study start31 May 2021
- Primary completion26 Sep 2022
- Study completion26 Sep 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Ataxia, SpinocerebellarStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 33 participants (actual)
Sponsor and investigators
- Lead sponsor
- Changhua Christian Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Chiu Chung Min, Changhua Christian Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05038306
- Sponsor's protocol ID
- 200703
- Status last verified
- Jan 2023
- First submitted
- 27 May 2021
- Last updated
- 31 Jan 2023
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 31 Jan 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Chinese Medicine WT for Elevating IGF-1 of Patients With Spinocerebellar Ataxia Type 3 - Pilot Study
- ClinicalTrials.gov ID
- NCT05038306
- Acronym / sponsor ID
- 200703
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 33 participants (actual)
- Lead sponsor
- Changhua Christian HospitalUniversity, hospital or other
- Study start
- 31 May 2021 (actual)
- Primary completion
- 26 Sep 2022 (actual)
- Study completion
- 26 Sep 2022 (actual)
- First posted
- 9 Sep 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Chinese Medicine | Active comparator | Drug: Chinese medicine WT |
| Placebo | Placebo comparator | Drug: Chinese medicine WT |
Who may qualify
- Ages
- 20 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (1 listed)
- diagnosed as Spinocerebellar Ataxia type 3
Key exclusion criteria (1 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Chinese medicine WTThe 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. | Drug | Chinese Medicine, Placebo |
Primary outcome
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.
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
8 secondary outcomes
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
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
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
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
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
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
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
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
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 31 Jan 2023.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Chinese medicine WT in people with Ataxia, Spinocerebellar and Insulin-Like Growth Factor I, run by Changhua Christian Hospital at 1 site in Taiwan.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 1 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 31 Jan 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Taiwan. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 31 Jan 2023.