Efficacy and Safety Study of Stemchymal® in Polyglutamine Spinocerebellar Ataxia

• ClinicalTrials.gov Identifier: NCT02540655
• Recruitment Status: Completed
• First Posted: September 4, 2015
• Last Update Posted: October 27, 2022
• Sponsor: Steminent Biotherapeutics Inc.
• Information provided by (Responsible Party): Steminent Biotherapeutics Inc.

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.

Condition or disease	Intervention/treatment	Phase
Cerebellar Ataxia	Biological: Stemchymal®; Procedure: Excipients	Phase 2

Study Design

• Study Type: Interventional (Clinical Trial)
• Actual Enrollment: 56 participants
• Allocation: Randomized
• Intervention Model: Parallel Assignment
• Masking: Triple (Participant, Care Provider, Investigator)
• Primary Purpose: Treatment
• 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 Start Date: September 2015
• Actual Primary Completion Date: February 26, 2021
• Actual Study Completion Date: February 26, 2021

Arms and Interventions

Arm	Intervention/treatment
Experimental: Stemchymal®; Infusion of Stemchymal®	Biological: Stemchymal®; Patients will receive Stemchymal® through intravenous infusion
Placebo Comparator: Vehicle; Infusion of excipients	Procedure: Excipients; Patients will receive excipients through intravenous infusion

Outcome Measures

Primary Outcome Measures :

1. 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 Measures :

1. 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 ]
2. To assess the changes from the baseline to all visits on SCA functional index (SCAFI) [ Time Frame: 14 months ]
3. To assess the changes from the baseline to all visits on sensory organization test (SOT) [ Time Frame: 14 months ]
   Balance test
4. To assess the changes from the baseline to all visits on inventory of non-ataxia signs (INAS) [ Time Frame: 14 months ]
5. To assess the changes from the baseline to all visits on clinical global impression (CGI) [ Time Frame: 14 months ]
6. To assess the changes from the baseline to all visits on patient global impression of improvement (PGI-I) [ Time Frame: 14 months ]

Eligibility Criteria

• Ages Eligible for Study: 20 Years to 70 Years (Adult, Older Adult)
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Criteria

Inclusion Criteria:

1. Subjects are with genotypically confirmed spinocerebellar ataxia type 2 or spinocerebellar ataxia type 3.
2. Subjects' baseline SARA score are in the range of 8 to15.
3. Subjects are between 20 and 70 years of age.
4. Subjects who had signed informed consent.

Exclusion Criteria:

1. Subjects had been enrolled in any kind of cell therapy within six months prior to screening visit.
2. Females with a positive pregnancy test result.
3. 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.
4. Subjects with immunological disorders (ex. Systemic lupus erythematosus), within six months prior to screening visit.
5. Subjects with other neurological disorders (ex. Alzheimer's disease), within six months prior to screening visit.
6. Subjects who had received chemotherapy/radiotherapy within five years prior to screening visit.
7. Subjects with any history of malignancy tumors.
8. Subjects with a history of hypersensitivity/allergy to penicillin.
9. Subjects with dementia or other psychiatric illnesses, including but not limited to disabling depression, bipolar disorder, and schizophrenia.
10. Subjects with Beck Depression Inventory Second Edition (BDI-II) score over 20 points.
11. Subjects with unstable illnesses or contraindication for this clinical trial according to PI's judgment.

Contacts and Locations

Locations

Taiwan

Taipei Veterans General Hospital

Taipei, Taiwan

Sponsors and Collaborators

Steminent Biotherapeutics Inc.

More Information

• Responsible Party: Steminent Biotherapeutics Inc.
• ClinicalTrials.gov Identifier: NCT02540655
• Other Study ID Numbers: IB02
• First Posted: September 4, 2015
• Last Update Posted: October 27, 2022
• Last Verified: October 2022

Keywords provided by Steminent Biotherapeutics Inc.:

Stem Cells; Adult Stem Cells; Spinocerebellar Ataxia; Steminent

Additional relevant MeSH terms:

Ataxia; Cerebellar Ataxia; Spinocerebellar Ataxias; Spinocerebellar Degenerations; Dyskinesias; Neurologic Manifestations; Nervous System Diseases; Cerebellar Diseases; Brain Diseases; Central Nervous System Diseases; Spinal Cord Diseases; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Genetic Diseases, Inborn