Cerebellar AtaxiaPhase 2NCT02540655
Efficacy and Safety Study of Stemchymal® in Polyglutamine Spinocerebellar Ataxia
A Phase 2 study testing Stemchymal® and Excipients in people with Cerebellar Ataxia, run by Steminent Biotherapeutics Inc. at 1 site in Taiwan.
Trial summary
The purpose of the clinical trial is to study the therapeutic efficacy and safety of Stemchymal® infusions for polyglutamine spinocerebellar ataxia treatment by a randomized, double-blind, placebo-controlled study design. Eligible subjects will receive Stemchymal® through intravenous infusion. Read more about this study
- Who is it for?
- Ages 20 to 70 years
- Conditions the study is for
- Cerebellar Ataxia
- What is being tested?
- Stemchymal® and Excipients
- Type of treatment
- Injection or infusion, Surgery or procedure
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 56 participants
- Where
- 1 siteTaiwan
- Study dates
- Sep 2015to 26 Feb 2021
- 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 70 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Subjects are with genotypically confirmed spinocerebellar ataxia type 2 or spinocerebellar ataxia type 3.
- Subjects' baseline SARA score are in the range of 8 to15.
- Subjects are between 20 and 70 years of age.
- Subjects who had signed informed consent.
Who cannot join
- Subjects had been enrolled in any kind of cell therapy within six months prior to screening visit.
- Females with a positive pregnancy test result.
- Subjects who had had severe vital organ disease as diagnosed, including but not limited to cardiac (ex. heart failure), liver (ex. acute hepatic failure or chronic liver cirrhosis), lung (ex. respiratory failure) and renal (ex. hemodialysis or peritoneal dialysis) insufficiency, within six months prior to screening visit.
- Subjects with immunological disorders (ex. Systemic lupus erythematosus), within six months prior to screening visit.
- Subjects with other neurological disorders (ex. Alzheimer's disease), within six months prior to screening visit.
- Subjects who had received chemotherapy/radiotherapy within five years prior to screening visit.
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- Subjects with any history of malignancy tumors.
- Subjects with a history of hypersensitivity/allergy to penicillin.
- Subjects with dementia or other psychiatric illnesses, including but not limited to disabling depression, bipolar disorder, and schizophrenia.
- Subjects with Beck Depression Inventory Second Edition (BDI-II) score over 20 points.
- Subjects with unstable illnesses or contraindication for this clinical trial according to PI's judgment.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: A Phase II, Randomized, Double-Blind, Placebo-Controlled, Single-Center Study to Evaluate the Safety and Efficacy of Stemchymal® Infusion for the Treatment of Polyglutamine Spinocerebellar Ataxia
The purpose of the clinical trial is to study the therapeutic efficacy and safety of Stemchymal® infusions for polyglutamine spinocerebellar ataxia treatment by a randomized, double-blind, placebo-controlled study design. Eligible subjects will receive Stemchymal® through intravenous infusion.
- Conditions
- Cerebellar Ataxia
- Being tested
- Stemchymal®, Excipients
- Keywords
- Stem Cells, Adult Stem Cells, Spinocerebellar Ataxia, Steminent
- Medical subject headings
- Cerebellar Ataxia, Spinocerebellar Ataxias
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Stemchymal®Biological
Patients will receive Stemchymal® through intravenous infusion
ExcipientsProcedure or surgery
Patients will receive excipients through intravenous infusion
| Study group | Role | What they receive |
|---|---|---|
| Stemchymal® | Experimental | Infusion of Stemchymal® Receives: Biological: Stemchymal® |
| Vehicle | Placebo comparator | Infusion of excipients Receives: Procedure: Excipients |
What is being measured
The primary outcome is the main question the study is designed to answer.
To assess the changes from the baseline to all visits on Scale for the Assessment and Rating of Ataxia (SARA) score.
Measured: 14 months
Secondary outcomes (6)
To assess the safety by incidence of adverse events, changes of vital signs, blood biochemistry tests, complete blood count, immunoactivity assay, urinalysis and magnetic resonance imaging (MRI).
Measured: 14 months
To assess the changes from the baseline to all visits on SCA functional index (SCAFI)
Measured: 14 months
To assess the changes from the baseline to all visits on sensory organization test (SOT)
Balance test
Measured: 14 months
To assess the changes from the baseline to all visits on inventory of non-ataxia signs (INAS)
Measured: 14 months
To assess the changes from the baseline to all visits on clinical global impression (CGI)
Measured: 14 months
To assess the changes from the baseline to all visits on patient global impression of improvement (PGI-I)
Measured: 14 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Sep 2015 (estimated)
- Study startSep 2015
- Primary completion26 Feb 2021
- Study completion26 Feb 2021
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Cerebellar AtaxiaStudies 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
- Parallel groups
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 56 participants (actual)
Sponsor and investigators
- Lead sponsor
- Steminent Biotherapeutics Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02540655
- Sponsor's protocol ID
- IB02
- Status last verified
- Oct 2022
- First submitted
- 26 Aug 2015
- Last updated
- 27 Oct 2022
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 27 Oct 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase II, Randomized, Double-Blind, Placebo-Controlled, Single-Center Study to Evaluate the Safety and Efficacy of Stemchymal® Infusion for the Treatment of Polyglutamine Spinocerebellar Ataxia
- ClinicalTrials.gov ID
- NCT02540655
- Acronym / sponsor ID
- IB02
- Conditions
- Cerebellar Ataxia
- Keywords
- Stem Cells, Adult Stem Cells, Spinocerebellar Ataxia, Steminent
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 56 participants (actual)
- Lead sponsor
- Steminent Biotherapeutics Inc.Industry (pharma, biotech, devices)
- Study start
- Sep 2015
- Primary completion
- 26 Feb 2021 (actual)
- Study completion
- 26 Feb 2021 (actual)
- First posted
- 4 Sep 2015 (estimated)
- 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.
The purpose of the clinical trial is to study the therapeutic efficacy and safety of Stemchymal® infusions for polyglutamine spinocerebellar ataxia treatment by a randomized, double-blind, placebo-controlled study design. Eligible subjects will receive Stemchymal® through intravenous infusion.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Stemchymal® | Experimental | Biological: Stemchymal®Infusion of Stemchymal® |
| Vehicle | Placebo comparator | Procedure: ExcipientsInfusion of excipients |
Who may qualify
- Ages
- 20 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- Subjects are with genotypically confirmed spinocerebellar ataxia type 2 or spinocerebellar ataxia type 3.
- Subjects' baseline SARA score are in the range of 8 to15.
- Subjects are between 20 and 70 years of age.
- Subjects who had signed informed consent.
Key exclusion criteria (11 listed)
- Subjects had been enrolled in any kind of cell therapy within six months prior to screening visit.
- Females with a positive pregnancy test result.
- Subjects who had had severe vital organ disease as diagnosed, including but not limited to cardiac (ex. heart failure), liver (ex. acute hepatic failure or chronic liver cirrhosis), lung (ex. respiratory failure) and renal (ex. hemodialysis or peritoneal dialysis) insufficiency, within six months prior to screening visit.
- Subjects with immunological disorders (ex. Systemic lupus erythematosus), within six months prior to screening visit.
- Subjects with other neurological disorders (ex. Alzheimer's disease), within six months prior to screening visit.
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 |
|---|---|---|
| Stemchymal®Patients will receive Stemchymal® through intravenous infusion | Biological | Stemchymal® |
| ExcipientsPatients will receive excipients through intravenous infusion | Procedure or surgery | Vehicle |
Primary outcome
To assess the changes from the baseline to all visits on Scale for the Assessment and Rating of Ataxia (SARA) score.
Time frame: 14 months
6 secondary outcomes
To assess the safety by incidence of adverse events, changes of vital signs, blood biochemistry tests, complete blood count, immunoactivity assay, urinalysis and magnetic resonance imaging (MRI).
Time frame: 14 months
To assess the changes from the baseline to all visits on SCA functional index (SCAFI)
Time frame: 14 months
To assess the changes from the baseline to all visits on sensory organization test (SOT)
Time frame: 14 months
To assess the changes from the baseline to all visits on inventory of non-ataxia signs (INAS)
Time frame: 14 months
To assess the changes from the baseline to all visits on clinical global impression (CGI)
Time frame: 14 months
To assess the changes from the baseline to all visits on patient global impression of improvement (PGI-I)
Time frame: 14 months
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 27 Oct 2022.
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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 Stemchymal® and Excipients in people with Cerebellar Ataxia, run by Steminent Biotherapeutics Inc. 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 4 inclusion and 11 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 27 Oct 2022. 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?
To assess the changes from the baseline to all visits on Scale for the Assessment and Rating of Ataxia (SARA) score. (time frame: 14 months). 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 27 Oct 2022.