Safety and Efficacy of Umbilical Cord Mesenchymal Stem Cell Therapy for Patients With Hereditary Ataxia

Phase I/II Study of Umbilical Cord Mesenchymal Stem Cell Therapy for Patients With Hereditary Ataxia

Status

UNKNOWN

Phases

Phase 1Phase 2

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT01360164

Enrollment

Registered

2011-05-25

Start date

2010-01-31

Completion date

2013-12-31

Last updated

2012-11-28

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Hereditary Ataxia

Keywords

Human Umbilical Cord Mesenchymal Stem Cells

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.

Detailed description

This Study is designed to evaluate the the safety and efficacy of Human Umbilical Cord Mesenchymal Stem Cells transplantation in patients with Hereditary Ataxias.

Interventions

BIOLOGICALhuman umbilical cord mesenchymal stem cells

Participants will be given hUC-MSCs transplantation.

Study design

Allocation

NON_RANDOMIZED

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Masking

NONE

Eligibility

Sex/Gender

ALL

Age

16 Years to 65 Years

Healthy volunteers

Inclusion criteria

* 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.

Exclusion criteria

* 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； * Other criteria the investigator consider improper for inclusion.

Design outcomes

Primary

Measure	Time frame
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

Measure	Time frame
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

Countries

China

Contacts

Primary ContactYun Xu

xuyun20042001@yahoo.com.cn

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026