NCT02540655

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.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
56

participants targeted

Target at P25-P50 for phase_2

Timeline
Completed

Started Sep 2015

Longer than P75 for phase_2

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

August 26, 2015

Completed
6 days until next milestone

Study Start

First participant enrolled

September 1, 2015

Completed
3 days until next milestone

First Posted

Study publicly available on registry

September 4, 2015

Completed
5.5 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

February 26, 2021

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

February 26, 2021

Completed
Last Updated

October 27, 2022

Status Verified

October 1, 2022

Enrollment Period

5.5 years

First QC Date

August 26, 2015

Last Update Submit

October 26, 2022

Conditions

Cerebellar Ataxia

Keywords

Stem CellsAdult Stem CellsSpinocerebellar AtaxiaSteminent

Outcome Measures

Primary Outcomes (1)

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

    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).

    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)

    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

  • +1 more secondary outcomes

Study Arms (2)

Stemchymal®

EXPERIMENTAL

Infusion of Stemchymal®

Biological: Stemchymal®

Vehicle

PLACEBO COMPARATOR

Infusion of excipients

Procedure: Excipients

Interventions

Stemchymal®BIOLOGICAL

Patients will receive Stemchymal® through intravenous infusion

Stemchymal®
ExcipientsPROCEDURE

Patients will receive excipients through intravenous infusion

Vehicle

Eligibility Criteria

Age20 Years - 70 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • 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.

You may not qualify if:

  • 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.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Taipei Veterans General Hospital

Taipei, Taiwan

Location

MeSH Terms

Conditions

Cerebellar AtaxiaSpinocerebellar Ataxias

Interventions

Excipients

Condition Hierarchy (Ancestors)

Cerebellar DiseasesBrain DiseasesCentral Nervous System DiseasesNervous System DiseasesAtaxiaDyskinesiasNeurologic ManifestationsSigns and SymptomsPathological Conditions, Signs and SymptomsSpinocerebellar DegenerationsSpinal Cord DiseasesHeredodegenerative Disorders, Nervous SystemNeurodegenerative DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Pharmaceutical VehiclesPharmaceutic AidsPharmaceutical PreparationsSpecialty Uses of ChemicalsChemical Actions and Uses

Study Design

Study Type
interventional
Phase
phase 2
Allocation
RANDOMIZED
Masking
TRIPLE
Who Masked
PARTICIPANT, CARE PROVIDER, INVESTIGATOR
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

August 26, 2015

First Posted

September 4, 2015

Study Start

September 1, 2015

Primary Completion

February 26, 2021

Study Completion

February 26, 2021

Last Updated

October 27, 2022

Record last verified: 2022-10

Locations

TW(1)