Efficacy and Safety Study of Stemchymal® in Polyglutamine Spinocerebellar Ataxia

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.

Study Overview

• Status: Completed
• Conditions: Cerebellar Ataxia
• Intervention / Treatment: Biological: Stemchymal®; Procedure: Excipients

• Study Type: Interventional
• Enrollment (Actual): 56
• Phase: Phase 2

Contacts and Locations

Study Locations

• Taiwan
  • Taipei, Taiwan
    • Taipei Veterans General Hospital

Participation Criteria

Eligibility Criteria

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

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Triple

Number of Arms

2

Arms and Interventions

Participant Group / 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

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
To assess the changes from the baseline to all visits on Scale for the Assessment and Rating of Ataxia (SARA) score.	14 months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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).		14 months
To assess the changes from the baseline to all visits on SCA functional index (SCAFI)		14 months
To assess the changes from the baseline to all visits on sensory organization test (SOT)	Balance test	14 months
To assess the changes from the baseline to all visits on inventory of non-ataxia signs (INAS)		14 months
To assess the changes from the baseline to all visits on clinical global impression (CGI)		14 months
To assess the changes from the baseline to all visits on patient global impression of improvement (PGI-I)		14 months

Collaborators and Investigators

• Sponsor: Steminent Biotherapeutics Inc.

Study record dates

Study Major Dates

• Study Start: September 1, 2015
• Primary Completion (Actual): February 26, 2021
• Study Completion (Actual): February 26, 2021

Study Registration Dates

• First Submitted: August 26, 2015
• First Submitted That Met QC Criteria: September 1, 2015
• First Posted (Estimate): September 4, 2015

Study Record Updates

• Last Update Posted (Actual): October 27, 2022
• Last Update Submitted That Met QC Criteria: October 26, 2022
• Last Verified: October 1, 2022

More Information

Terms related to this study

• Keywords: Stem Cells; Spinocerebellar Ataxia; Adult Stem Cells; Steminent
• Additional Relevant MeSH Terms: Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Neurologic Manifestations; Genetic Diseases, Inborn; Neurodegenerative Diseases; Dyskinesias; Spinal Cord Diseases; Heredodegenerative Disorders, Nervous System; Cerebellar Diseases; Ataxia; Cerebellar Ataxia; Spinocerebellar Ataxias; Spinocerebellar Degenerations
• Other Study ID Numbers: IB02