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Efficacy and Safety Study of Stemchymal® in Polyglutamine Spinocerebellar Ataxia

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ClinicalTrials.gov Identifier: NCT02540655
Recruitment Status : Recruiting
First Posted : September 4, 2015
Last Update Posted : November 1, 2016
Sponsor:
Information provided by (Responsible Party):
Steminent Biotherapeutics Inc.

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 Type : Interventional  (Clinical Trial)
Estimated Enrollment : 60 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
Estimated Primary Completion Date : November 2017
Estimated Study Completion Date : February 2018


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




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 ]


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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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02540655


Contacts
Contact: Chih-Yuan Ho, Ph.D. +886-2-26279216 ext 670 kevinho@steminent.com

Locations
Taiwan
Taipei Veterans General Hospital Recruiting
Taipei, Taiwan
Sponsors and Collaborators
Steminent Biotherapeutics Inc.

Responsible Party: Steminent Biotherapeutics Inc.
ClinicalTrials.gov Identifier: NCT02540655     History of Changes
Other Study ID Numbers: IB02
First Posted: September 4, 2015    Key Record Dates
Last Update Posted: November 1, 2016
Last Verified: October 2016

Keywords provided by Steminent Biotherapeutics Inc.:
Stem Cells
Adult Stem Cells
Spinocerebellar Ataxia
Steminent

Additional relevant MeSH terms:
Spinocerebellar Ataxias
Spinocerebellar Degenerations
Ataxia
Cerebellar Ataxia
Dyskinesias
Neurologic Manifestations
Nervous System Diseases
Signs and Symptoms
Cerebellar Diseases
Brain Diseases
Central Nervous System Diseases
Spinal Cord Diseases
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Genetic Diseases, Inborn