Allogeneic UCB Therapy With EPO in Children With CP

MinYoung Kim, MD, PhD

Status: Completed

Conditions: Cerebral Palsy

Treatments: Procedure: Placebo UCB; Other: Rehabilitation; Biological: Erythropoietin alfa; Biological: Placebo EPO; Procedure: Umbilical Cord Blood therapy

Study type: Interventional

Funder types: Other; Industry

Identifiers

NCT01991145

UCBnEPOinCP

Details and patient eligibility

About

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.

Full 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.

Enrollment: 92 patients

Sex: All

Ages: 10 months to 6 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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92 participants in 4 patient groups, including a placebo group

UCB and EPO

Experimental group

Description:

UCB + EPO + Rehabilitation

Treatment:

Other: Rehabilitation

Procedure: Umbilical Cord Blood therapy

Biological: Erythropoietin alfa

UCB and placebo EPO

Active Comparator group

Description:

UCB + placebo EPO + Rehabilitation

Treatment:

Other: Rehabilitation

Procedure: Umbilical Cord Blood therapy

Biological: Placebo EPO

placebo UCB and EPO

Active Comparator group

Description:

placebo UCB + EPO + Rehabilitation

Treatment:

Other: Rehabilitation

Procedure: Placebo UCB

Biological: Erythropoietin alfa

placebo UCB and placebo EPO

Placebo Comparator group

Description:

placebo UCB + placebo EPO + Rehabilitation

Treatment:

Other: Rehabilitation

Procedure: Placebo UCB

Biological: Placebo EPO