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Clinical Effect and Safety of Autologous Umbilical Cord Blood Transfusion in the Treatment of Cerebral Palsy

Primary Purpose

Safety Issues, Effect of Drugs

Status

Unknown status

Phase

Phase 1

Locations

China

Study Type

Interventional

Intervention

0.9% Sodium Chloride

Autologous Umbilical Cord Blood

About this trial

This is an interventional treatment trial for Safety Issues focused on measuring Cerebral palsy, Autologous Umbilical Cord Blood, safety, effect

Eligibility Criteria

6 Months - 10 Years (Child)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

A patient who was diagnosed with a mild case, and severity case of cerebral palsy or developmental disability through developmental assessment from more than two specialists including a specialist in pediatrics and rehabilitation medicine..
In case there is a sign of the general decrease in white matter, or periventricular leukomalacia near cerebral ventricles in MRI DTI test.
A subject who did a written consent to participation in this clinical trial through the very person and a legal representative.

Exclusion Criteria:

In case a patient underwent a surgical procedure, or was administered autologous cord blood within one year before participating in a clinical trial.
Accompanied by a serious disease, such as chromosome abnormality, etc.
In case where a patient's medical condition is judged to be maladapted by a researcher.
In case a patient or his or her legal representative doesn't agree to participation in a clinical trial.
A patient having a predisposition to allergies.
A patient having serious disorders in the liver, kidney, and cardiopulmonary function.

Sites / Locations

Guangdong Women and Children's Hospital and Health InstituteRecruiting

Arms of the Study

Arm 1

Arm 2

Arm Type

Placebo Comparator

Experimental

Arm Label

Placebo

Cord Blood Mononuclear Cells(CBMNC)

Arm Description

0.9% sodium chloride infusion any time after entering the Placebo Group, doses is 20-30ml. The infusion speed is 1ml/min.

Autologous Umbilical Cord Blood Mononuclear Cells Therapy any time after the diagnosis of cerebral palsy, doses is 20-30ml (total Mononuclear cells>1*10^7/kg). The infusion speed is 1ml/min.

Outcomes

Primary Outcome Measures

The Change of Gross Motor Function Classification System(GMFCS) Score

GMFCS is used to assess the gross motor function of the children who were diagnosised as cerebral palsy.The results of GMFCS are divided into five levels. Grade I represents the best outcome while grade Ⅴ represents the worst.

The Change of Gesell Developmental Scale(GDS) Score

The GDS is used to measure the gross motor,fine action,adaptability，language and individual-society function of the children whose age ranges from 1-month to 6-year-old. The score less than 75 for each subscale represent the worse outcome and more than 75 represent the better.

Secondary Outcome Measures

Full Information

NCT ID

NCT03791372

First Posted

December 18, 2018

Last Updated

January 3, 2019

Sponsor

Guangdong Women and Children Hospital

1. Study Identification

Unique Protocol Identification Number

NCT03791372

Brief Title

Clinical Effect and Safety of Autologous Umbilical Cord Blood Transfusion in the Treatment of Cerebral Palsy

Official Title

Clinical Effect and Safety of Autologous Umbilical Cord Blood Transfusion in the Treatment of Cerebral Palsy

Study Type

Interventional

2. Study Status

Record Verification Date

January 2019

Overall Recruitment Status

Unknown status

Study Start Date

October 10, 2017 (Actual)

Primary Completion Date

October 10, 2020 (Anticipated)

Study Completion Date

April 10, 2021 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Principal Investigator

Name of the Sponsor

Guangdong Women and Children Hospital

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Studies a U.S. FDA-regulated Device Product

Data Monitoring Committee

Yes

5. Study Description

Brief Summary

To study the clinical efficacy and safety of autologous umbilical cord blood transfusion in the treatment of cerebral palsy.

Detailed Description

This is a Phase 1 clinical trial that constitutes two time points cohorts with participants who will receive intravenous autologous umbilical cord blood and 0.9% sodium chloride respectively. The timing of treatment is any time after the diagnosis of cerebral palsy. The investigator will proceed the groups during the same period. Demographic Data and Baseline Characteristics of the Studied Group were collected: Basic patient's information survey Medical history Physical examination Basic blood test result Children's developmental disorders evaluation before the treatment Brain Magnetic Resonance Imaging-Diffusion Tensor Imaging (MRI-DTI) before the treatment Neurocognitive function test before the treatment Assessment of clinical condition in the course by measurement of blood pressure, heart and respiratory rates, temperature and adverse events was recorded. Autologous cord blood doses is 20-30ml (total Mononuclear cells>1*10^7/kg)，the infusion speed is 1ml/min, and with same volume of 0.9% sodium chloride as placebo. The follow-up: clinical test until 30th month in 3 month gaps.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Safety Issues, Effect of Drugs

Keywords

Cerebral palsy, Autologous Umbilical Cord Blood, safety, effect

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 1

Interventional Study Model

Parallel Assignment

Model Description

Parallel assignment

Masking

Outcomes Assessor

Allocation

Non-Randomized

Enrollment

25 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title

Placebo

Arm Type

Placebo Comparator

Arm Description

0.9% sodium chloride infusion any time after entering the Placebo Group, doses is 20-30ml. The infusion speed is 1ml/min.

Arm Title

Cord Blood Mononuclear Cells(CBMNC)

Arm Type

Experimental

Arm Description

Autologous Umbilical Cord Blood Mononuclear Cells Therapy any time after the diagnosis of cerebral palsy, doses is 20-30ml (total Mononuclear cells>1*10^7/kg). The infusion speed is 1ml/min.

Intervention Type

Drug

Intervention Name(s)

0.9% Sodium Chloride

Intervention Description

0.9% Sodium Chloride in control group

Intervention Type

Biological

Intervention Name(s)

Autologous Umbilical Cord Blood

Other Intervention Name(s)

Autologous Umbilical Cord Blood Mononuclear Cells Therapy

Intervention Description

Autologous Umbilical Cord Blood Mononuclear Cells Therapy in Cerebral Palsy for safety and effect evaluation.

Primary Outcome Measure Information:

Title

The Change of Gross Motor Function Classification System(GMFCS) Score

Description

Time Frame

up to 30 months after therapy at a 3-month interval

Title

The Change of Gesell Developmental Scale(GDS) Score

Description

Time Frame

every 3-month after therapy until 30 months

10. Eligibility

Sex

All

Minimum Age & Unit of Time

6 Months

Maximum Age & Unit of Time

10 Years

Accepts Healthy Volunteers

Eligibility Criteria

Inclusion Criteria: A patient who was diagnosed with a mild case, and severity case of cerebral palsy or developmental disability through developmental assessment from more than two specialists including a specialist in pediatrics and rehabilitation medicine.. In case there is a sign of the general decrease in white matter, or periventricular leukomalacia near cerebral ventricles in MRI DTI test. A subject who did a written consent to participation in this clinical trial through the very person and a legal representative. Exclusion Criteria: In case a patient underwent a surgical procedure, or was administered autologous cord blood within one year before participating in a clinical trial. Accompanied by a serious disease, such as chromosome abnormality, etc. In case where a patient's medical condition is judged to be maladapted by a researcher. In case a patient or his or her legal representative doesn't agree to participation in a clinical trial. A patient having a predisposition to allergies. A patient having serious disorders in the liver, kidney, and cardiopulmonary function.

Central Contact Person:

First Name & Middle Initial & Last Name or Official Title & Degree

Xiao Huimei, MD

Phone

+862039151772

Ext

+86

xiaowu2010727@163.com

First Name & Middle Initial & Last Name or Official Title & Degree

Chang Yanqun, Phd

Phone

+862039151772

Ext

+86

sfycyq@sina.com

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Chang Yanqun, Phd

Organizational Affiliation

Guangdong Women and Children Hospital

Official's Role

Study Director

Facility Information:

Facility Name

Guangdong Women and Children's Hospital and Health Institute

City

Guangzhou

State/Province

Guangdong

ZIP/Postal Code

510010

Country

China

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Chang Yanqun, Phd

Phone

+862039151772

sfycyq@sina.com

12. IPD Sharing Statement

Plan to Share IPD

Undecided

Citations:

PubMed Identifier

29080265

Citation

Sun JM, Song AW, Case LE, Mikati MA, Gustafson KE, Simmons R, Goldstein R, Petry J, McLaughlin C, Waters-Pick B, Chen LW, Wease S, Blackwell B, Worley G, Troy J, Kurtzberg J. Effect of Autologous Cord Blood Infusion on Motor Function and Brain Connectivity in Young Children with Cerebral Palsy: A Randomized, Placebo-Controlled Trial. Stem Cells Transl Med. 2017 Dec;6(12):2071-2078. doi: 10.1002/sctm.17-0102. Epub 2017 Oct 28.

Results Reference

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Clinical Effect and Safety of Autologous Umbilical Cord Blood Transfusion in the Treatment of Cerebral Palsy

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