Transplantation of Umbilical Cord-derived Mesenchymal Stem Cells Via Different Routes

January 26, 2018 updated by: Liu Jing, The First Affiliated Hospital of Dalian Medical University

Transplantation of Umbilical Cord-derived Mesenchymal Stem Cells Via Different Routes for the Treatment of Cerebral Palsy in Children

To preliminarily evaluate the efficacy and safety of umbilical cord-derived mesenchymal stem cells (UC-MSCs), and compare the efficacy of UC-MSCs administered through the intravenous, intrathecal, and intranasal routes, in the treatment of cerebral palsy in children.

Study Overview

Status

Unknown

Conditions

Intervention / Treatment

Detailed Description

Cerebral palsy refers to a neurological disorder caused by a non-progressive brain injury or malformation that occurs in early childhood. It can result in central motor deficits, developmental retardation, abnormal posture, abnormal muscular strength, abnormal muscle tone and/or dysreflexia. It has a high disability rate. There is currently no effective treatment for cerebral palsy. Conventional treatments for cerebral palsy are often tiresome and expensive and have a slow onset of action. Stem cells have been recently used in the treatment of cerebral palsy. This provides a novel method for the treatment of cerebral palsy. According to the existing clinical studies, neural stem cells, bone marrow-derived mesenchymal stem cells, and umbilical cord-derived mesenchymal stem cells (UC-MSCs) are mainly used as the seed cells for the treatment of cerebral palsy. UC-MSCs are the most promising seed cells for the treatment of cerebral palsy because of the advantages including rich sources, ease of harvesting, short doubling time, low immunogenicity, long-time survival post-transplantation, and no ethical issues. UC-MSCs have been widely used to treat Parkinson's disease, rheumatoid arthritis, traumatic brain injury, aplastic anemia, and decompensated liver disease. However,only two studies, and performed only in China, are reported on the treatment of cerebral palsy with UC-MSCs. One from the Hospital 463 of PLA reported 51 patients with cerebral palsy whose symptoms had not been obviously improved after 1 year of rehabilitative treatment. These patients received intrathecal injection of UC-MSCs for three times, once a week, followed by one injection of UC-MSCs via the peripheral vein in the fourth week. Four UC-MSCs injections, once per week, were considered as one course of treatment. Activities of daily living scale score was compared between before and after treatment to evaluate efficacy. Another study is a case report from China. In this report, a combined intravenous and intrathecal injection of UC-MSCs was used to treat cerebral palsy in a 5-year-old child. 28-month follow-up results revealed that the child's gross motor function, immune function, muscle strength, and language ability improved and adverse reactions were not obvious.

Study Type

Interventional

Enrollment (Anticipated)

44

Phase

  • Not Applicable

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • China
    • Liaoning
      • Dalian, Liaoning, China, 116011
        • The First Affiliated Hospital of Dalian Medical University

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

2 years to 18 years (Child, Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

Inclusion Criteria:

  • Patients diagnosed with hypoxic/ischemic cerebral palsy, primarily including birth asphyxia and premature children
  • Patients with spastic quadriplegia
  • Patients with moderate to severe cerebral palsy, GMFM scores of 2-3
  • Age at 2-18 years
  • Provision of signed informed consent by legal representatives of the child prior to start of the study

Exclusion Criteria:

  • Those with systemic diseases that likely interfere with the treatment or child's compliance
  • Those complicated by life-threatening diseases of any organ
  • Those with brain deformity
  • Those with uncontrolled epilepsy
  • Those with abnormal behavior or mood disorders
  • Those with allergies especially those who are allergic to blood products
  • Those are infected with infectious diseases
  • Those who had underwent a craniocerebral surgery in other clinical trials

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

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

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Other: Control group
Routine rehabilitation treatments
Other: Control group
The control group received routine rehabilitation treatments including acupuncture, massage, Bobath therapy, and Vojat therapy.
Experimental: Intravenous UC-MSCs group
Injection of UC-MSCs via the peripheral vein.
Procedure: UC-MSCs
Injection of umbilical cord-derived mesenchymal stem cells (UC-MSCs) (at least 1×10^7), once every 2 weeks, one course of treatment including two injections within 4 weeks, a total of two courses of treatment.
Experimental: Intrathecal UC-MSCs group
Injection of UC-MSCs via the intrathecal route.
Procedure: UC-MSCs
Injection of umbilical cord-derived mesenchymal stem cells (UC-MSCs) (at least 1×10^7), once every 2 weeks, one course of treatment including two injections within 4 weeks, a total of two courses of treatment.
Experimental: Intranasal UC-MSCs group
Injection of UC-MSCs via the nasal route.
Procedure: UC-MSCs
Injection of umbilical cord-derived mesenchymal stem cells (UC-MSCs) (at least 1×10^7), once every 2 weeks, one course of treatment including two injections within 4 weeks, a total of two courses of treatment.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
The Gross Motor Function Measure (GMFM)-88 item score
Time Frame: 12 months after 2 courses of treatment with UC-MSCs.
a 4-point scale that is divided into five categories including lying and rolling, sitting, crawling and kneeling, standing, walking, running, and jumping. A score of 0 indicates that the child does not initiate the task; 1 indicates that the child initiates the task (completes < 10% of the activity); 2 indicates that the child partially completes the task (completes from 10 to 99% of the activity); 3 indicates that the child completes the task (100%). The sum of score of the five categories is used as the evaluation outcome.It is used to evaluate gross motor function.
12 months after 2 courses of treatment with UC-MSCs.

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Fine Motor Function Measure scale (FMFM) score
Time Frame: 1, 3, 6, 9 and 12 months after 2 courses of treatment with UC-MSCs.
To evaluate fine motor function.The scale is divided into 45 items covering visual tracing (7 items), upper extremity range of motion (8 items), grab ability (8 items), operational ability (10 items), and hand-eye coordination (12 items). The FMFM is a 4-point scale with the score range of 0-3. The original total score is 135. The summed score for evaluating fine motor function measure is 0-100.
1, 3, 6, 9 and 12 months after 2 courses of treatment with UC-MSCs.
The Modified Ashworth scale (MAS) score
Time Frame: 1, 3, 6, 9 and 12 months after 2 courses of treatment with UC-MSCs.
To evaluate muscle tone. The MAS score is divided into six grades: 0, 1, 1+, 2, 3 and 4. Passive full ranges of motion exercises are performed by moving the knee and ankle until the patient feel a slight resistance from hamstrings and plantar flexor muscles (soleus muscle and gastrocnemius muscle). The average score is calculated across three repeated measurements.
1, 3, 6, 9 and 12 months after 2 courses of treatment with UC-MSCs.
Gesell Development Schedule score
Time Frame: 1, 3, 6, 9 and 12 months after 2 courses of treatment with UC-MSCs.
To evaluate neurobehavioral development.
1, 3, 6, 9 and 12 months after 2 courses of treatment with UC-MSCs.
Incidence of adverse reactions
Time Frame: 1, 3, 6, 9 and 12 months after 2 courses of treatment with UC-MSCs.
Liver and kidney dysfunction, fever, infection, vomiting.
1, 3, 6, 9 and 12 months after 2 courses of treatment with UC-MSCs.

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

The First Affiliated Hospital of Dalian Medical University

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Anticipated)

April 1, 2018

Primary Completion (Anticipated)

September 1, 2020

Study Completion (Anticipated)

December 1, 2020

Study Registration Dates

First Submitted

January 22, 2018

First Submitted That Met QC Criteria

January 26, 2018

First Posted (Actual)

January 30, 2018

Study Record Updates

Last Update Posted (Actual)

January 30, 2018

Last Update Submitted That Met QC Criteria

January 26, 2018

Last Verified

January 1, 2018

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • FirstHospitalDalianMU004

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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