NCT01360164

Brief Summary

The Hereditary Ataxias are a group of genetic disorders characterized by slowly progressive incoordination of gait and often associated with poor coordination of hands, speech, and eye movements. Current treatments for Hereditary Ataxias are mainly pharmacological, rehabilitative, or psychological treatments，while no effective treatment available. Stem Cell therapy is a novel and promising therapeutic strategy for Hereditary Ataxias treatment. In this study, the safety and efficacy of Human Umbilical Cord Mesenchymal Stem Cells transplantation will be evaluated in patients with Hereditary Ataxias.

Trial Health

43
At Risk

Trial Health Score

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

Trial has exceeded expected completion date
Enrollment
20

participants targeted

Target at P25-P50 for phase_1

Timeline
Completed

Started Jan 2010

Longer than P75 for phase_1

Geographic Reach
1 country

1 active site

Status
unknown

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

Study Start

First participant enrolled

January 1, 2010

Completed
1.4 years until next milestone

First Submitted

Initial submission to the registry

May 23, 2011

Completed
2 days until next milestone

First Posted

Study publicly available on registry

May 25, 2011

Completed
1.5 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 1, 2012

Completed
1 year until next milestone

Study Completion

Last participant's last visit for all outcomes

December 1, 2013

Completed
Last Updated

November 28, 2012

Status Verified

November 1, 2012

Enrollment Period

2.9 years

First QC Date

May 23, 2011

Last Update Submit

November 26, 2012

Conditions

Hereditary Ataxia

Keywords

Human Umbilical Cord Mesenchymal Stem Cells

Outcome Measures

Primary Outcomes (2)

  • scores of International Cooperative Ataxia Rating Scale （ICARS） scale and Berg Scale

    1 year after treatment

  • the volume of Cerebellum of Brain Magnetic Resonance Imaging （MRI） Scan

    1 year after treatment

Secondary Outcomes (2)

  • Number of Participants with Adverse Events as a Measure of Safety and Tolerability

    1 year after treatment

  • Number of Participants with Serious Adverse Events as a Measure of Safety and Tolerability

    1 year after treatment

Study Arms (1)

Human umbilical cord mesenchymal stem cells transplantation

EXPERIMENTAL

Participants will be given umbilical cord mesenchymal stem cells transplantation with a 1 year follow-up.

Biological: human umbilical cord mesenchymal stem cells

Interventions

human umbilical cord mesenchymal stem cellsBIOLOGICAL

Participants will be given hUC-MSCs transplantation.

Also known as: Intervention Group
Human umbilical cord mesenchymal stem cells transplantation

Eligibility Criteria

Age16 Years - 65 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • Aged 16-65 years.
  • Harding Diagnosis of SCAs, gene type confirmed.
  • Candidates who did not receive any stem cell therapy in past 6months.
  • sign the consent form and follow the clinic trail procedure.

You may not qualify if:

  • Cardiac insufficiency; Renal insufficiency; hepatic insufficiency; Total bilirubin higher than 1.5 times of upper limit of normal value; AST /ALT higher than 2.5 times of upper limit of normal value；
  • Routine Blood Test: WBC count \<3.0×109/ L; PLT count \<5×109/L ; or Hemoglobin \<100g/L；
  • Combined Pneumonia or other Severe systemic bacteria infection；
  • Severe drug allergic history or anaphylaxis to 2 or more food or medicine；
  • Other brain organic disease (eg. Brain cancer)；
  • HIV+, Tumor Markers + ；
  • Severe psychotic patients, cognitive dysfunction， or can not understand or sign the Consent Form；
  • Other severe systemic or organic disease；
  • Uncontrolled hypertension，blood pressure≥180mmHg/110 mmHg after treatment；
  • Pregnancy；
  • Enrollment in other trials in the last 3 months；

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Nanjing University Medical College Affiliated Drum Tower Hospital

Nanjing, Jiangsu, China

RECRUITING

Related Publications (1)

  • Jin JL, Liu Z, Lu ZJ, Guan DN, Wang C, Chen ZB, Zhang J, Zhang WY, Wu JY, Xu Y. Safety and efficacy of umbilical cord mesenchymal stem cell therapy in hereditary spinocerebellar ataxia. Curr Neurovasc Res. 2013 Feb;10(1):11-20. doi: 10.2174/156720213804805936.

    PMID: 23151076DERIVED

MeSH Terms

Conditions

Spinocerebellar Degenerations

Condition Hierarchy (Ancestors)

Cerebellar DiseasesBrain DiseasesCentral Nervous System DiseasesNervous System DiseasesSpinal Cord DiseasesHeredodegenerative Disorders, Nervous SystemNeurodegenerative DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Central Study Contacts

Yun Xu

CONTACT

xuyun20042001@yahoo.com.cn

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NON RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

May 23, 2011

First Posted

May 25, 2011

Study Start

January 1, 2010

Primary Completion

December 1, 2012

Study Completion

December 1, 2013

Last Updated

November 28, 2012

Record last verified: 2012-11

Locations

CN(1)