Clinical trial NCT01991145: Allogeneic UCB Therapy With EPO in Children With CP
Official title: Safety and Efficacy of Allogeneic Umbilical Cord Blood Therapy Combined With Erythropoietin in Children With Cerebral Palsy: a Double-blind, Randomized, Placebo-controlled Clinical Trial
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: NA
Conditions: Cerebral Palsy
Keywords: Cerebral Palsy
Umbilical Cord Blood
Erythropoietin
Rehabilitation
Lead sponsor: Bundang CHA Hospital
Sponsor class: OTHER
Collaborators: name: Ministry of Health & Welfare, Korea
name: LG Life Sciences
name: Chong Kun Dang Pharmaceutical
name: CHA University
First posted: 2013-11-25
Enrollment: 92
Sex: ALL
Minimum age (years): 10
Maximum age (years): 6
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: This randomized controlled study aims to evaluate the efficacy and safety of allogeneic umbilical cord blood therapy combined with erythropoietin for children with cerebral palsy.

Detailed description: Cerebral palsy (CP) is a group of neurodevelopmental conditions with abnormal movement and posture resulted from a non-progressive cerebral disturbance. It is the most common cause of motor disability in childhood. Most therapies are palliative rather than restorative. Umbilical cord blood (UCB) and erythropoetin (EPO) may be used as restorative approach for children with CP.

Many experimental animal studies have revealed that UCB is beneficial to improve and repair neurological injuries. EPO is also known to have neuroprotective effects.

Based on animal studies and some clinical trials, UCB is suggested as a potential therapy for children with CP. EPO is combined to add synergistic effects to UCB therapy.

Eligibility criteria: Inclusion Criteria:

* Diagnosed with cerebral palsy
* Age of ≥10 months and ≤6 years
* Mismatch in HLA-A, B, and DR ≤2, and total nucleated cell count ≥3x107/kg. If the cell count is less than given values, more than 1 unit could be used.
* Hemoglobin ≤13.6 g/dL
* Decision of participation in the study by and acquisition of informed consent from the subject's representative
* Willingness and ability to be hospitalized according to the schedule specified in the protocol and continue the study for 12 months after study entry

Exclusion Criteria:

* Current aspiration pneumonia
* Known genetic disease
* History of hypersensitivity reaction to any study drugs pertinent to the study
* History of participation in any other study with stem cell
* Prior treatment with EPO within 3 months prior to study entry
* Known coagulopathy with family history of thrombosis or medical history of recurrent thrombosis
* Patient with severe seizure disease who has clinical convulsion despite combination therapy with 3 or more agents
* Uncontrolled hypertension defined as systolic blood pressure \>115 mmHg and/or diastolic blood pressure \>70 mmHg
* Hepatic impairment defined as asparate aminotransferase (AST) \>55 IU/L and/or alanine aminotransferase (ALT) \>45 IU/L
* Renal impairment defined as creatinine (Cr) ≥1.2 mg/dL
* Absolute neutrophil count ≤500/dL
* Presence of diagnosed or suspected malignant tumor and/or hematologic malignancy
* Non-compliance with study visits specified in the protocol or unwillingness of care-giver due to lack of understanding of the patient

Primary outcomes:
measure: Changes in Standardized Gross Motor Function
timeFrame: Baseline - 1 month - 3 months - 6 months - 12months
measure: Changes in Motor Performance
timeFrame: Baseline - 1 month - 3 months - 6 months - 12 months
measure: Changes in Cognitive Neurodevelopmental Outcome
timeFrame: Baseline - 1 month - 3 months - 6 months - 12 months
measure: Changes in Motor Neurodevelopmental Outcome
timeFrame: Baseline - 1 month - 3 months - 6 months - 12 months
Secondary outcomes:
measure: Changes in Gross Motor Function Classification System
timeFrame: Baseline - 1 month - 3 months - 6 months - 12 months
measure: Changes in Functional Independence in Daily Activities
timeFrame: Baseline - 1 month - 3 months - 6 months - 12 months
measure: Changes in Functional Performance in Daily Activities
timeFrame: Baseline - 1 month - 3 months - 6 months - 12 months
measure: Changes in Upper Extremity Function
timeFrame: Baseline - 1 month - 3 months - 6 months - 12 months
measure: Changes in Visual Perception Test
timeFrame: Baseline - 1 month - 3 months - 6 months - 12 months
measure: Changes in Selective Movement of Lower Extremity
timeFrame: Baseline - 1 month - 3 months - 6 months - 12 months
measure: Changes in Spasticity
timeFrame: Baseline - 1 month - 3 months - 6 months - 12 months
measure: Changes in Dynamic Component of Spasticity
timeFrame: Baseline - 1 month - 3 months - 6 months - 12 months
measure: Changes in Muscle Strength
timeFrame: Baseline - 1 month - 3 months - 6 months - 12 months
measure: Changes in Brain MRI
timeFrame: Baseline - 12 months
measure: Changes in Brain 18F-FDG PET
timeFrame: Baseline - 12 months
measure: Changes in EEG
timeFrame: Baseline - 12 months
measure: Changes in EP
timeFrame: Baseline - 12 months
measure: Number of adverse events and participants with those adverse events
timeFrame: 12 months
Other outcomes:
Locations:
city: Seongnam-si
country: Korea, Republic of
facility: CHA Bundang Medical Center, CHA University
state: Gyeonggi-do
Study officials:
affiliation: CHA University
name: MinYoung Kim, M.D., Ph.D.
role: PRINCIPAL_INVESTIGATOR
References:
citation: Min K, Suh MR, Cho KH, Park W, Kang MS, Jang SJ, Kim SH, Rhie S, Choi JI, Kim HJ, Cha KY, Kim M. Potentiation of cord blood cell therapy with erythropoietin for children with CP: a 2 x 2 factorial randomized placebo-controlled trial. Stem Cell Res Ther. 2020 Nov 27;11(1):509. doi: 10.1186/s13287-020-02020-y.