Clinical trial NCT03791372: 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
Overall status: UNKNOWN
Phase: PHASE1
Conditions: Safety Issues
Effect of Drugs
Keywords: Cerebral palsy
Autologous Umbilical Cord Blood
safety
effect
Lead sponsor: Guangdong Women and Children Hospital
Sponsor class: OTHER
First posted: 2019-01-02
Enrollment: 25
Sex: ALL
Minimum age (years): 6
Maximum age (years): 10
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: PARALLEL
Intervention model description: Parallel assignment
Primary purpose: TREATMENT

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.

1. 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
2. Assessment of clinical condition in the course by measurement of blood pressure, heart and respiratory rates, temperature and adverse events was recorded.
3. 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.
4. The follow-up: clinical test until 30th month in 3 month gaps.

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.

Primary outcomes:
measure: The Change of Gross Motor Function Classification System(GMFCS) Score
timeFrame: up to 30 months after therapy at a 3-month interval
measure: The Change of Gesell Developmental Scale(GDS) Score
timeFrame: every 3-month after therapy until 30 months
Secondary outcomes:
Other outcomes:
Locations:
city: Guangzhou
country: China
facility: Guangdong Women and Children's Hospital and Health Institute
state: Guangdong
Study officials:
affiliation: Guangdong Women and Children Hospital
name: Chang Yanqun, Phd
role: STUDY_DIRECTOR
References:
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.