Clinical trial NCT01489267: A New Method to Treat Hereditary Cerebellar Ataxia - Umbilical Cord Mesenchymal Stem Cells Transplantation
Official title: The Safety and Efficacy of Umbilical Cord Mesenchymal Stem Cells Transplantation in Hereditary Cerebellar Ataxia Patients
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE2
Conditions: Hereditary Cerebellar Ataxia.
Keywords: cerebellar ataxia
hereditary disease
stem cell
Lead sponsor: General Hospital of Chinese Armed Police Forces
Sponsor class: OTHER
First posted: 2011-12-09
Enrollment: 20
Sex: ALL
Minimum age (years): 18
Maximum age (years): 65
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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 .

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

Primary outcomes:
measure: blood test
timeFrame: within one week before transplantation
measure: Nerve functional evaluation
timeFrame: within one week before transplantation
measure: blood test
timeFrame: 1 month after transplantation
measure: blood test
timeFrame: 3 months after transplantation
measure: blood test
timeFrame: 6 months after transplantation
measure: blood test
timeFrame: 12 months after transplantation
measure: Nerve functional evaluation
timeFrame: 1 month after transplantation
measure: Nerve functional evaluation
timeFrame: 3 months after transplantation
measure: Nerve functional evaluation
timeFrame: 6 months after transplantation
measure: Nerve functional evaluation
timeFrame: 12 months after transplantation
Secondary outcomes:
measure: Urinal test
timeFrame: within one week before transplantation
measure: Electrophysiology examination
timeFrame: within one week before transplantation
measure: Urinal test
timeFrame: 1 month after transplantation
measure: Urinal test
timeFrame: 3 months after transplantation
measure: Urinal test
timeFrame: 6 months after transplantation
measure: Urinal test
timeFrame: 12 months after transplantation
measure: Electrophysiology examination
timeFrame: 1 month after transplantation
measure: Electrophysiology examination
timeFrame: 3 months after transplantation
measure: Electrophysiology examination
timeFrame: 6 months after transplantation
measure: Electrophysiology examination
timeFrame: 12 months after transplantation
Other outcomes:
Locations:
city: Beijing
country: China
facility: Yihua An
Study officials:
affiliation: director of neural stem cell transplantation department in general hospital of chinese armed police forces
name: Yihua An, doctor
role: STUDY_DIRECTOR
References: