Combination Therapy of Cord Blood and G-CSF for Patients With Brain Injury or Neurodegenerative Disorders

Not Applicable

Completed

• Conditions: Brain Injury; Amyotrophic Lateral Sclerosis; Parkinson's Disease; Cerebral Palsy
• Interventions: Procedure: Umbilical cord blood therapy; Biological: Filgrastim

• Registration Number: NCT02236065
• Lead Sponsor: MinYoung Kim, M.D.

Brief Summary

This open label trial is conducted to investigate the efficacy and safety of the combination therapy of allogeneic umbilical cord blood (UCB) and granulocyte-colony stimulating factor (G-CSF) for patients with brain injury or neurodegenerative disorders.

Detailed Description

Current treatments for brain injury or neurodegenerative disorders are palliative rather than curative. Preclinical and some clinical studies suggest that UCB and G-CSF can be used as restorative approach for such disorders.

Study Timeline

• Study Start: 2014-08
• Study First Submitted: 2014-09-08
• Study First Submitted QC: 2014-09-08
• Study First Posted: 2014-09-10
• Primary Completion: 2016-07
• Study Completion: 2016-07
• Status Verified: 2017-10
• Last Update Submitted: 2017-10-10
• Last Update Posted: 2017-10-12

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 10

Inclusion Criteria

should be included one of the 4 disorders

• Brain injury: onset duration over 12 months, Age: 19 years or over
• Cerebral palsy: spastic, dyskinetic or ataxic, Age: 19 years or over
• Parkinson's disease: modified Hoehn and Yahr stage 2.5, 3, 4, Age: 30 to 75 years
• ALS: definite of possible ALS, progression during the past 6 months, Age: 19 to 65

Exclusion Criteria

• Uncontrolled pulmonary, renal dysfunction at enrollment
• Uncontrolled seizure
• Malignant cancer
• Possibility of hypersensitivity to drugs used in this study
• Contraindication to the study intervention or assessment
• Pregnant or breast feeding women
• Non-compliance with the study visits specified in the protocol

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
UCB + G-CSF	Umbilical cord blood therapy	UCB + G-CSF
UCB + G-CSF	Filgrastim	UCB + G-CSF

Primary Outcome Measures

Name	Time	Method
Changes in Berg Balance Scale	Baseline - 1 month - 3 months - 6 months	Berg Balance Scale for brain injury and parkinson's disease (range: 0 to 56, Higher scores indicates better balance function.)
Changes in the Level of Disability	Baseline - 1 month - 3 months - 6 months	FIM (Functional Independence Measure) for brain injury and cerebral palsy (range: 18 to 126, Higher scores indicate more independence in activities of daily living.)
Changes in Standardized Gross Motor Function	Baseline - 1 month - 3 months - 6 months	GMFM (Gross Motor Function Measure) for cerebral palsy (range: 0 to 100, Higher scores indicate better gross motor function.)
Changes in Motor Performance	Baseline - 1 month - 3 months - 6 months	GMPM (Gross Motor Performance Measure) for cerebral palsy (range: 0 to 100, Higher scores indicate better motor quality.)
Changes in ALSFRS-R	Baseline - 1 month - 3 months - 6 months	ALSFRS-R (Amyotrophic Lateral Sclerosis Functional Rating Scale-revised) for ALS (range: 0 to 48, Higher scores indicate better physical function.)
Changes in UPDRS	Baseline - 1 month - 3 months - 6 months	UPDRS (Unified Parkinson's Disease Rating Scale) for parkinson's disease (Part 1: mentation, behavior and mood; Part 2: activities of daily living; Part 3: motor examination; Part 4: complications of therapy; Part 5: Schwab and England activities of daily living scale)

Secondary Outcome Measures

Name	Time	Method
Changes in Brain MRI	Baseline - 6 months	Analysis of Diffusion Tensor Image (DTI) for brain injury, cerebral palsy, ALS
Changes in Brain PET	Baseline - 6 months	for parkinson's disease
Number of adverse events and participants with those adverse events	6 months	The numbers of adverse events and subjects with those serious adverse events within each group; A serious adverse event is any untoward medical occurrence that at any dose: results in death or is life-threatening, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, or causes a congenital anomaly/birth defect.

Trial Locations

Locations (1)

CHA Bundang Medical Center, CHA University; 🇰🇷 Seongnam-si, Gyeonggi-do, Korea, Republic of