Efficacy of Stem Cell Transplantation Compared to Rehabilitation Treatment of Patients With Cerebral Paralysis (CP)

Efficacy of Stem Cell Transplantation Compared to Rehabilitation Treatment of Children With Cerebral Paralysis

Cerebral palsy (CP) is described as a group of permanent disorders affecting motor development and posture, resulting in activity limitation attributed to nonprogressive disturbances of the fetal or infant brain. The prevalence of cerebral palsy has increased among the children with low birth-weight, jaundice, respiratory distress and intrauterine infection and so on. The incidence of cerebral palsy is increasing gradually with increased neonatal survival rate. Although there are many kinds of functional therapy programs especially the rehabilitation treatment for cerebral palsy, their effects are limited. Increasing cerebral palsy patients become a heavy burden to the family and society. Stem cell based therapy, a new prospective therapy for central nervous system disorders, has the potential to repair the damaged brain tissue in patients with cerebral palsy.

In this study, 300 patients with cerebral palsy will be divided into three groups and the investigators will use mesenchymal stem cells derived from umbilical cord to treat 100 CP patients of them randomly. We will also follow up the other 100 patients who only receive rehabilitation treatment and another 100 patients who accept neither stem cell therapy nor rehabilitation treatment. On this basis, as the investigators we can compare the efficacy of cell therapy and rehabilitation treatments for cerebral palsy patients.

Multiple sources of assessment were used to ascertain and classify all cases of cerebral palsy. Particularly the Gross Motor Function Measure (GMFM) as an important valid and reliable outcome measure, has made it possible to evaluate the severity of movement disability，change over time and the effects of clinical interventions. It also will be the primary outcome measure in follow-up analysis of this study.

Study Overview

• Status: Completed
• Conditions: Cerebral Palsy
• Intervention / Treatment: Other: rehabilitation; Biological: stem cell injection

Detailed Description

Patients enrolled in this study need to finish our whole follow-up survey for 12 months, which is carried out by clinical doctors, rehabilitators and epidemiologist.

• Study Type: Interventional
• Enrollment (Actual): 300
• Phase: Phase 3

Contacts and Locations

Study Locations

• China
  • Beijing
    • Beijing, Beijing, China, 100050
      • General Hospital of Chinese People's Armed Police Forces

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 1 year to 14 years (Child)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• Patients with diagnosis of cerebral palsy.
• Patients' curator must be able to give voluntary consent.

Exclusion Criteria:

• Intracranial infection.
• Severe respiratory and circulatory system diseases.
• Hematologic malignancies.
• Positive serological tests such as AIDS, hepatitis B virus, hepatitis C virus and syphilis （antigen or antibody）.
• Tumors.
• Genetic and metabolic diseases.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Single

Number of Arms

3

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Active Comparator: rehabilitation; Patients in the group accept rehabilitation for three weeks in hospital and other eleven months in their home under the guidance of physical therapist.	Other: rehabilitation; Patients only receive rehabilitation of physical therapy and occupational therapy.; Other Names: phsical exercise rehabilitation
No Intervention: control; Patients receive no professional treatment in hospital or rehabilitation centre.
Experimental: stem cell injection; Patients in the group accept cell therapy including four times stem cells transplant via intrathecal injection.	Biological: stem cell injection; Mesenchymal stem cells derived from umbilical cord are transplanted directly into subarachnoid by Lumbar puncture.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Gross Motor Function Measure Score	Gross Motor Function Measure-88 and Gross Motor Function Measure-66	Change from baseline at 12 monthes after enrollment or transplantation

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Routine Blood Test and Biochemical Test	red blood cell; white blood cell; platelet count; glutamic pyruvic transaminase; glutamic oxaloacetic transaminase	Change from baseline at 12 monthes after enrollment or transplantation

Other Outcome Measures

Outcome Measure	Measure Description	Time Frame
Magnetic resonance imaging （MRI）	The MRI repots would describe brain tissue especially white matter and malacia.	Change from baseline at 12 monthes after enrollment or transplantation

Collaborators and Investigators

• Sponsor: General Hospital of Chinese Armed Police Forces
• Investigators: Study Director: Yihua An, Doctor, the General Hospital of Chinese People's Armed Police Forces

Publications and helpful links

General Publications

• Rethlefsen SA, Ryan DD, Kay RM. Classification systems in cerebral palsy. Orthop Clin North Am. 2010 Oct;41(4):457-67. doi: 10.1016/j.ocl.2010.06.005.
• Pharoah PO, Platt MJ, Cooke T. The changing epidemiology of cerebral palsy. Arch Dis Child Fetal Neonatal Ed. 1996 Nov;75(3):F169-73. doi: 10.1136/fn.75.3.f169.
• Richards CL, Malouin F. Cerebral palsy: definition, assessment and rehabilitation. Handb Clin Neurol. 2013;111:183-95. doi: 10.1016/B978-0-444-52891-9.00018-X.
• Himmelmann K. Epidemiology of cerebral palsy. Handb Clin Neurol. 2013;111:163-7. doi: 10.1016/B978-0-444-52891-9.00015-4. No abstract available.
• Reddihough DS, Collins KJ. The epidemiology and causes of cerebral palsy. Aust J Physiother. 2003;49(1):7-12. doi: 10.1016/s0004-9514(14)60183-5.
• Lubis MU, Tjipta GD, Marbun MD, Saing B. Cerebral palsy. Paediatr Indones. 1990 Mar-Apr;30(3-4):65-70.

Study record dates

Study Major Dates

• Study Start: October 1, 2013
• Primary Completion (Actual): December 1, 2015
• Study Completion (Actual): December 1, 2016

Study Registration Dates

• First Submitted: August 20, 2013
• First Submitted That Met QC Criteria: August 21, 2013
• First Posted (Estimate): August 28, 2013

Study Record Updates

• Last Update Posted (Actual): December 27, 2017
• Last Update Submitted That Met QC Criteria: December 23, 2017
• Last Verified: July 1, 2015

More Information

Terms related to this study

• Keywords: rehabilitation; cerebral palsy; cell transplantation
• Additional Relevant MeSH Terms: Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Neurologic Manifestations; Brain Damage, Chronic; Cerebral Palsy; Paralysis
• Other Study ID Numbers: 2013-05-13 CP III