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

General Hospital of Chinese Armed Police Forces

Status and phase: Unknown; Phase 2

Conditions: Hereditary Cerebellar Ataxia.

Treatments: Other: stem cell transplantation

Study type: Interventional

Funder types: Other

Identifiers

NCT01489267

20111031SCA

20111031limin (Other Identifier)

Details and patient eligibility

About

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.

Full 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 .

Enrollment: 20 estimated patients

Sex: All

Ages: 18 to 65 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Single Group Assignment

Masking: None (Open label)

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20 participants in 2 patient groups

Control group

No Intervention group

Description:

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.

Stem cell transplantation

Experimental group

Description:

10 patients in the group accept stem cell transplantation.

Treatment:

Other: stem cell transplantation