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Efficacy and Safety Study of Stemchymal® in Polyglutamine Spinocerebellar Ataxia

Primary Purpose

Cerebellar Ataxia

Status

Completed

Phase

Phase 2

Locations

Taiwan

Study Type

Interventional

Intervention

Stemchymal®

Excipients

About this trial

This is an interventional treatment trial for Cerebellar Ataxia focused on measuring Stem Cells, Adult Stem Cells, Spinocerebellar Ataxia, Steminent

Eligibility Criteria

20 Years - 70 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

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.

Exclusion Criteria:

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.
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.

Sites / Locations

Taipei Veterans General Hospital

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Placebo Comparator

Arm Label

Stemchymal®

Vehicle

Arm Description

Infusion of Stemchymal®

Infusion of excipients

Outcomes

Primary Outcome Measures

To assess the changes from the baseline to all visits on Scale for the Assessment and Rating of Ataxia (SARA) score.

Secondary Outcome Measures

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).

To assess the changes from the baseline to all visits on SCA functional index (SCAFI)

To assess the changes from the baseline to all visits on sensory organization test (SOT)

Balance test

To assess the changes from the baseline to all visits on inventory of non-ataxia signs (INAS)

To assess the changes from the baseline to all visits on clinical global impression (CGI)

To assess the changes from the baseline to all visits on patient global impression of improvement (PGI-I)

Full Information

NCT ID

NCT02540655

First Posted

August 26, 2015

Last Updated

October 26, 2022

Sponsor

Steminent Biotherapeutics Inc.

1. Study Identification

Unique Protocol Identification Number

NCT02540655

Brief Title

Efficacy and Safety Study of Stemchymal® in Polyglutamine Spinocerebellar Ataxia

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

Study Type

Interventional

2. Study Status

Record Verification Date

October 2022

Overall Recruitment Status

Completed

Study Start Date

September 2015 (undefined)

Primary Completion Date

February 26, 2021 (Actual)

Study Completion Date

February 26, 2021 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Steminent Biotherapeutics Inc.

4. Oversight

Data Monitoring Committee

Yes

5. Study Description

Brief 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.

6. Conditions and Keywords

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

Cerebellar Ataxia

Keywords

Stem Cells, Adult Stem Cells, Spinocerebellar Ataxia, Steminent

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 2

Interventional Study Model

Parallel Assignment

Masking

ParticipantCare ProviderInvestigator

Allocation

Randomized

Enrollment

56 (Actual)

8. Arms, Groups, and Interventions

Arm Title

Stemchymal®

Arm Type

Experimental

Arm Description

Infusion of Stemchymal®

Arm Title

Vehicle

Arm Type

Placebo Comparator

Arm Description

Infusion of excipients

Intervention Type

Biological

Intervention Name(s)

Stemchymal®

Intervention Description

Patients will receive Stemchymal® through intravenous infusion

Intervention Type

Procedure

Intervention Name(s)

Excipients

Intervention Description

Patients will receive excipients through intravenous infusion

Primary Outcome Measure Information:

Title

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

Secondary Outcome Measure Information:

Title

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

Title

To assess the changes from the baseline to all visits on SCA functional index (SCAFI)

Time Frame

14 months

Title

To assess the changes from the baseline to all visits on sensory organization test (SOT)

Description

Balance test

Time Frame

14 months

Title

To assess the changes from the baseline to all visits on inventory of non-ataxia signs (INAS)

Time Frame

14 months

Title

To assess the changes from the baseline to all visits on clinical global impression (CGI)

Time Frame

14 months

Title

To assess the changes from the baseline to all visits on patient global impression of improvement (PGI-I)

Time Frame

14 months

10. Eligibility

Sex

All

Minimum Age & Unit of Time

20 Years

Maximum Age & Unit of Time

70 Years

Accepts Healthy Volunteers

Eligibility Criteria

Inclusion Criteria: 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. Exclusion Criteria: 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. 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.

Facility Information:

Facility Name

Taipei Veterans General Hospital

City

Taipei

Country

Taiwan

12. IPD Sharing Statement

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Efficacy and Safety Study of Stemchymal® in Polyglutamine Spinocerebellar Ataxia

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