A New Method to Treat Hereditary Cerebellar Ataxia - Umbilical Cord Mesenchymal Stem Cells Transplantation

NCT ID: NCT01489267

Last Updated: 2012-06-20

Basic Information

• Recruitment Status: UNKNOWN
• Clinical Phase: PHASE2
• Total Enrollment: 20 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2011-12-31
• Study Completion Date: 2014-07-31

Brief Summary

Hereditary cerebellar ataxia is a type of autosomal dominant genetic disease, lesions mainly involving the cerebellum, but the spinal cord and cranial nerves may also be some involvement. A total of 20 molecularly diagnosed SCA1 patients divided in two groups. One group accepted for the treatment of stem cell transplantation,the other group will be the control. Purpose of this project to prove that allogeneic umbilical cord mesenchymal stem cells are applied to clinical safely, and in the treatment of hereditary cerebellar ataxia is valid.

Detailed Description

After admission the patients accepted transplantation would receive physical examination, blood and urine tests, electrocardiogram, electrophysiologic study;evaluation of SCA1 symptoms (balance, walking, dexterity, tremor, memory, mood and concentration),then stem cell therapy：after stem cell prepared, the patients accepted 4 times stem cell transplantations through lumbar puncture, the time is 3-5days between two treatments. The control only receive evaluation of SCA1 symptoms .

Conditions

Hereditary Cerebellar Ataxia.

Study Design

• Allocation Method: NON_RANDOMIZED
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Control group

Ten patients in the group only receive nerve functional evaluation and electrophysiology examination before and 1,3,6,12 months after recruit. They will not accept cell therapy.

Group Type: NO_INTERVENTION

No interventions assigned to this group

Stem cell transplantation

10 patients in the group accept stem cell transplantation.

Group Type: EXPERIMENTAL

stem cell transplantation

Intervention Type: OTHER

The patients accepted 4 times stem cell transplantations through lumbar puncture, the time is 3-5days between two treatments and the dose is about 2 ml(including 1×10'7 cells).

Interventions

stem cell transplantation

The patients accepted 4 times stem cell transplantations through lumbar puncture, the time is 3-5days between two treatments and the dose is about 2 ml(including 1×10'7 cells).

Intervention Type: OTHER

Other Intervention Names

Cell therapy.

Eligibility Criteria

Inclusion Criteria

1. 18-65 years of age

2. Molecularly diagnosed SCA1

Exclusion Criteria

1. Cognitively impaired individuals

2. Schizophrenics

3. Patients with severe kidney and liver disease, epilepsy, heart disease, pulmonary disease, cardiac arrhythmia, diabetes insipidus, leukemia and some other central neural disease (such as Parkinson disease, accidence of brain vascular et al)

4. Age less than 18 years, age greater than 65 years

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 65 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

General Hospital of Chinese Armed Police Forces

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Yihua An, doctor

Role: STUDY_DIRECTOR

director of neural stem cell transplantation department in general hospital of chinese armed police forces

Locations

Yihua An

Beijing, , China

Countries

China

Other Identifiers

20111031limin

Identifier Type: OTHER

Identifier Source: secondary_id

20111031SCA

Identifier Type: -

Identifier Source: org_study_id