Erythropoietin Therapy for Children With Cerebral Palsy: Phase 1

MinYoung Kim, M.D.

Status and phase: Completed; Phase 1

Conditions: Cerebral Palsy

Treatments: Drug: Erythropoietin

Study type: Interventional

Funder types: Other; Industry

Identifiers

NCT01586052

CPEPOPhase1

Details and patient eligibility

About

This purpose of this phase 1 study is to investigate the safety and efficacy of erythropoetin for children with cerebral palsy.

Full description

Cerebral palsy is a disorder of movement and posture resulted from a nonprogressive lesion or injury of the immature brain. It is a leading cause of childhood onset disability.

Many experimental animal studies have revealed that erythropoietin is useful to repair neurological injury in brain. The main mechanism of erythropoietin is supposed as follows; neuroprotection effect, angiogenesis, and anti-inflammation.

On the basis of many experimental studies, erythropoietin is suggested as a potential therapy for cerebral palsy.

Enrollment: 11 patients

Sex: All

Ages: 6 months to 3 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Cerebral Palsy
• Abnormal Muscle Tone
• GMFCS (Gross Motor Functional Classification System): II to IV
• Age: 6 months ~ 3 years
• Abnormal Brain MRI compatible to clinical features and non-progressive
• Willing to Comply with All Study Procedure

Exclusion criteria

• Known Genetic Disorder
• Baseline Erythropoietin level > 45 mU/mL
• Presence of Drug Hypersensitivity Related to the Study Remedy
• Previous Erythropoietin Treatment before 3 months
• Coagulopathy:

Family History, Unknown Cerebral Infarction, Thromboembolic Events History

• Intractable Seizure Disorder
• Poor Cooperation of Guardian including Inactive Attitude for Rehabilitation
• Uncontrolled Hypertension
• Liver Dysfunction
• Renal Dysfunction
• Absolute Neutrophil Count < 500/dL
• Intracerebral or Intraventricular Hemorrhage
• Malignancy

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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11 participants in 1 patient group

Erythropoietin and Rehabilitation

Experimental group

Description:

recombinant human erythropoietin injection and active rehabilitation

Treatment:

Drug: Erythropoietin