Phase II, Randomized, Double-blind Clinical Trial for Efficacy of G-CSF and Autologous Cord Blood Infusion in Children With Cerebral Palsy
Trial Snapshot
- Phase
- Phase 2
- Sponsor
- Enrollment
- 88
- Locations
- 1
- Primary Endpoint
- Confirm the safety of autologous cord blood infusion and repeated injection of G-CSF in children with cerebral palsy by repeated follow-up every 3 months over 18 months with clinical and laboratory evaluations
Study Overview
Brief Summary
This study is performed to reveal the safety and feasibility of combination therapy with autologous cord blood mononuclear cells (CB) and G-CSF as well as repeated administration of G-CSF for children with cerebral palsy. The evaluation tools are as follows: (1) Developmental tests (DDST, PEDI, GMFM, GMFCS, MACS, QUEST), (2) Neurocognitive function test (WPPSI-IV), (3) Brain MRI-DTI, (4) Peripheral blood CD34+ cell counts, (5) Neurotrophic factors/anti-inflammatory cytokines.
Study Design
- Study Type
- Interventional
- Allocation
- Randomized
- Intervention Model
- Parallel
- Primary Purpose
- Treatment
- Masking
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Eligibility Criteria
- Ages
- 2 Years to 10 Years (Child)
- Sex
- All
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- •Ages from 2 years to 10 years at the time of enrollment
- •Non-severe type of cerebral palsy
- •Willing to comply with all study procedures
Exclusion Criteria
- •Previous participation within 1 year in a clinical study with stem cell therapy including cord blood, G-CSF, and erythropoietin
- •Presence of chromosomal abnormalities
- •Unwillingness to participate clinical trial
- •Presence of hypersensitivity reaction to G-CSF
- •Evidence of hepatic, renal, cardiac dysfunctions
Arms & Interventions
CB + G-CSF
Intervention: G-CSF (Leucostim) (Drug)
CB + G-CSF
Intervention: CB (autologous cord blood) (Biological)
CB + placebo
Intervention: CB (autologous cord blood) (Biological)
CB + placebo
Intervention: Placebo (Normal saline) (Drug)
G-CSF
Intervention: G-CSF (Leucostim) (Drug)
Placebo
Intervention: Placebo (Normal saline) (Drug)
Outcomes
Primary Outcomes
Confirm the safety of autologous cord blood infusion and repeated injection of G-CSF in children with cerebral palsy by repeated follow-up every 3 months over 18 months with clinical and laboratory evaluations
Time Frame: For 18 months from date of randomization
Secondary Outcomes
- Confirm the efficacy of autologous cord blood infusion and/or G-CSF in children with cerebral palsy using standardized Gross Motor Function Measure evaluation.(Every 6 months from date of randomization up to 18 months)
Investigators
Young-Ho Lee
Professor, Department of Pediatrics
Hanyang University Seoul Hospital