Umbilical Cord Blood Therapy for Children With Global Developmental Delay
Trial Snapshot
- Phase
- Not Applicable
- Status
- Completed
- Sponsor
- Enrollment
- 14
- Locations
- 2
- Primary Endpoint
- Changes in Cognition
Study Overview
Brief Summary
This open-label study aims to evaluate the safety and efficacy of autologous or allogeneic umbilical cord blood therapy for children with global developmental delay.
Detailed Description
Global developmental delay is defined as significant delay in two or more of the following developmental domains: gross/fine motor, speech/language, cognition, social/personal, and activities of daily living.
Umbilical cord blood has been used for inherited metabolic diseases that feature global developmental delay and many experimental animal studies have revealed umbilical cord blood is useful to repair neurological impairments in brain.
Study Design
- Study Type
- Interventional
- Allocation
- Na
- Intervention Model
- Single Group
- Primary Purpose
- Treatment
- Masking
- None
Eligibility Criteria
- Ages
- 6 Months to 15 Years (Child)
- Sex
- All
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- •Global developmental delay
- •Willing to comply with all study procedure
Exclusion Criteria
- •Medical instability including pneumonia or renal function at enrollment
- •Poor cooperation of guardian,including inactive attitude for rehabilitation and visits for follow-up
- •Uncontrolled persistent epilepsy
- •Not eligible according to the principal investigator
Arms & Interventions
Umbilical cord blood transplantation
Allogeneic umbilical cord blood will be administered intravenously or intraarterially under non-myeloablative immunosuppression. In case of autologous umbilical cord blood, immunosuppression is not required.
Intervention: Umbilical cord blood transplantation (Procedure)
Outcomes
Primary Outcomes
Changes in Cognition
Time Frame: Baseline - 6 months - 12 months
Korean Wechsler Preschool and Primary Scale of Intelligence (K-WPPSI) (Higher value means better cognitive function (K-WPPSI: below 70 - worst, over 130 - best)
Changes in Cognitive Neurodevelopmental Outcome
Time Frame: Baseline - 1 month - 3 months - 6 months - 12 months
Korean version of Bayley Scale of Infant Development-II Mental Scales (Higher value means better cognitive function: raw score 0 - worst, 178 - best.)
Secondary Outcomes
- Changes in Standardized Gross Motor Function(Baseline - 1 month - 3 months - 6 months - 12 months)
- Changes in Motor Performance(Baseline - 1 month - 3 months - 6 months - 12 months)
- Changes in Functional Performance in Daily Activities(Baseline - 1 month - 3 months - 6 months - 12 months)
- Changes in Motor Neurodevelopmental Outcome(Baseline - 1 month - 3 months - 6 months - 12 months)
- Changes in Functional Independence in Daily Activities(Baseline - 1 month - 3 months - 6 months - 12 months)
- Changes in Muscle Strength(Baseline - 1 month - 3 months - 6 months - 12 months)
- Changes in Language Evaluation(Baseline - 6 months - 12 months)
- Changes in Brain glucose metabolism(Baseline - 12 months)
- Changes in Visual Perception Test(Baseline - 1 month - 3 months - 6 months - 12 months)
Investigators
MinYoung Kim, M.D.
Associate Professor
Bundang CHA Hospital