Safety and Efficacy of Umbilical Cord Mesenchymal Stem Cell Therapy for Patients With Hereditary Ataxia

Beike Biotech

Status and phase: Unknown; Phase 2; Phase 1

Conditions: Hereditary Ataxia

Treatments: Biological: human umbilical cord mesenchymal stem cells

Study type: Interventional

Funder types: Other; Industry

Identifiers

NCT01360164

BKCR-HA-1.0(2010)

Details and patient eligibility

About

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.

Full 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.

Enrollment: 20 estimated patients

Sex: All

Ages: 16 to 65 years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Single Group Assignment

Masking: None (Open label)

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20 participants in 1 patient group

Human umbilical cord mesenchymal stem cells transplantation

Experimental group

Description:

Participants will be given umbilical cord mesenchymal stem cells transplantation with a 1 year follow-up.

Treatment:

Biological: human umbilical cord mesenchymal stem cells