Phase II, Randomized, Double-blind Clinical Trial for Efficacy of G-CSF and Autologous Cord Blood Infusion in Children With Cerebral Palsy
Overview
- Phase
- Phase 2
- Intervention
- G-CSF (Leucostim)
- Conditions
- Cerebral Palsy
- Sponsor
- Hanyang University Seoul Hospital
- 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
- Last Updated
- 9 years ago
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.
Investigators
Young-Ho Lee
Professor, Department of Pediatrics
Hanyang University Seoul Hospital
Eligibility Criteria
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)
CB + G-CSF
Intervention: CB (autologous cord blood)
CB + placebo
Intervention: CB (autologous cord blood)
CB + placebo
Intervention: Placebo (Normal saline)
G-CSF
Intervention: G-CSF (Leucostim)
Placebo
Intervention: Placebo (Normal saline)
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)