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

Hereditary Ataxia Clinical Trial

Official title:

Phase I/II Study of Umbilical Cord Mesenchymal Stem Cell Therapy for Patients With Hereditary Ataxia

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT01360164
• Other study ID #: BKCR-HA-1.0(2010)
• Status: Recruiting
• Phase: Phase 1/Phase 2
• First received: May 23, 2011
• Last updated: November 26, 2012
• Start date: January 2010
• Est. completion date: December 2013

Study information

• Verified date: November 2012
• Source: Shenzhen Beike Bio-Technology Co., Ltd.
• Contact: Yun Xu
• Email: xuyun20042001[@]yahoo.com.cn
• Is FDA regulated: No
• Health authority: China: Ministry of Health
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 20
• Est. completion date: December 2013
• Est. primary completion date: December 2012
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 16 Years to 65 Years

Eligibility

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.

Study Design

Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Ataxia
• Cerebellar Ataxia
• Hereditary Ataxia
• Spinocerebellar Degenerations

Intervention

Biological:
• human umbilical cord mesenchymal stem cells
Participants will be given hUC-MSCs transplantation.

Locations

Country	Name	City	State
China	Nanjing University Medical College Affiliated Drum Tower Hospital	Nanjing	Jiangsu

Sponsors (6)

Lead Sponsor	Collaborator
Shenzhen Beike Bio-Technology Co., Ltd.	Nanjing University Medical College Affiliated Wuxi Second Hospital, The Affiliated Nanjing Brain Hospital of Nanjing University Medical School, The Affiliated Nanjing Drum Tower Hospital of Nanjing University Medical School, The Second Hospital of Nanjing Medical University, Xuzhou Medical College

Country where clinical trial is conducted

China, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	scores of International Cooperative Ataxia Rating Scale (ICARS) scale and Berg Scale		1 year after treatment	No
Primary	the volume of Cerebellum of Brain Magnetic Resonance Imaging (MRI) Scan		1 year after treatment	No
Secondary	Number of Participants with Adverse Events as a Measure of Safety and Tolerability		1 year after treatment	Yes
Secondary	Number of Participants with Serious Adverse Events as a Measure of Safety and Tolerability		1 year after treatment	Yes