Clinical trial NCT01360164: Safety and Efficacy of Umbilical Cord Mesenchymal Stem Cell Therapy for Patients With Hereditary Ataxia
Official title: Phase I/II Study of Umbilical Cord Mesenchymal Stem Cell Therapy for Patients With Hereditary Ataxia
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE1
PHASE2
Conditions: Hereditary Ataxia
Keywords: Human Umbilical Cord Mesenchymal Stem Cells
Lead sponsor: Shenzhen Beike Bio-Technology Co., Ltd.
Sponsor class: INDUSTRY
Collaborators: name: The Affiliated Nanjing Drum Tower Hospital of Nanjing University Medical School
name: Nanjing Medical University
name: Nanjing University Medical College Affiliated Wuxi Second Hospital
name: Xuzhou Medical University
name: The Second Hospital of Nanjing Medical University
First posted: 2011-05-25
Enrollment: 20
Sex: ALL
Minimum age (years): 16
Maximum age (years): 65
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.

Eligibility criteria: 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.

Primary outcomes:
measure: scores of International Cooperative Ataxia Rating Scale （ICARS） scale and Berg Scale
timeFrame: 1 year after treatment
measure: the volume of Cerebellum of Brain Magnetic Resonance Imaging （MRI） Scan
timeFrame: 1 year after treatment
Secondary outcomes:
measure: Number of Participants with Adverse Events as a Measure of Safety and Tolerability
timeFrame: 1 year after treatment
measure: Number of Participants with Serious Adverse Events as a Measure of Safety and Tolerability
timeFrame: 1 year after treatment
Other outcomes:
Locations:
city: Nanjing
country: China
facility: Nanjing University Medical College Affiliated Drum Tower Hospital
state: Jiangsu
Study officials:
References:
citation: Jin JL, Liu Z, Lu ZJ, Guan DN, Wang C, Chen ZB, Zhang J, Zhang WY, Wu JY, Xu Y. Safety and efficacy of umbilical cord mesenchymal stem cell therapy in hereditary spinocerebellar ataxia. Curr Neurovasc Res. 2013 Feb;10(1):11-20. doi: 10.2174/156720213804805936.