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.
This Study is designed to evaluate the the safety and efficacy of Human Umbilical Cord Mesenchymal Stem Cells transplantation in patients with Hereditary Ataxias.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
20
Participants will be given hUC-MSCs transplantation.
Nanjing University Medical College Affiliated Drum Tower Hospital
Nanjing, Jiangsu, China
RECRUITINGscores of International Cooperative Ataxia Rating Scale (ICARS) scale and Berg Scale
Time frame: 1 year after treatment
the volume of Cerebellum of Brain Magnetic Resonance Imaging (MRI) Scan
Time frame: 1 year after treatment
Number of Participants with Adverse Events as a Measure of Safety and Tolerability
Time frame: 1 year after treatment
Number of Participants with Serious Adverse Events as a Measure of Safety and Tolerability
Time frame: 1 year after treatment
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