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

A Pilot Study of Combination Therapy of Allogeneic Umbilical Cord Blood and Granulocyte-colony Stimulating Factor for Patients With Brain Injury or Neurodegenerative Disorders

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.

Study Overview

• Status: Completed
• Conditions: Cerebral Palsy; Parkinson's Disease; Brain Injury; Amyotrophic Lateral Sclerosis
• Intervention / Treatment: Biological: Filgrastim; Procedure: Umbilical cord blood therapy

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 Type: Interventional
• Enrollment (Actual): 10
• Phase: Not Applicable

Contacts and Locations

Study Locations

• Korea, Republic of
  • Gyeonggi-do
    • Seongnam-si, Gyeonggi-do, Korea, Republic of, 463-712
      • CHA Bundang Medical Center, CHA University

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 19 years to 75 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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 Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: UCB + G-CSF	Biological: Filgrastim; Procedure: Umbilical cord blood therapy

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Changes in Berg Balance Scale	Berg Balance Scale for brain injury and parkinson's disease (range: 0 to 56, Higher scores indicates better balance function.)	Baseline - 1 month - 3 months - 6 months
Changes in the Level of Disability	FIM (Functional Independence Measure) for brain injury and cerebral palsy (range: 18 to 126, Higher scores indicate more independence in activities of daily living.)	Baseline - 1 month - 3 months - 6 months
Changes in Standardized Gross Motor Function	GMFM (Gross Motor Function Measure) for cerebral palsy (range: 0 to 100, Higher scores indicate better gross motor function.)	Baseline - 1 month - 3 months - 6 months
Changes in Motor Performance	GMPM (Gross Motor Performance Measure) for cerebral palsy (range: 0 to 100, Higher scores indicate better motor quality.)	Baseline - 1 month - 3 months - 6 months
Changes in ALSFRS-R	ALSFRS-R (Amyotrophic Lateral Sclerosis Functional Rating Scale-revised) for ALS (range: 0 to 48, Higher scores indicate better physical function.)	Baseline - 1 month - 3 months - 6 months
Changes in UPDRS	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)	Baseline - 1 month - 3 months - 6 months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Changes in Brain MRI	Analysis of Diffusion Tensor Image (DTI) for brain injury, cerebral palsy, ALS	Baseline - 6 months
Changes in Brain PET	for parkinson's disease	Baseline - 6 months
Number of adverse events and participants with those adverse events	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.	6 months

Collaborators and Investigators

• Sponsor: MinYoung Kim, M.D.

Study record dates

Study Major Dates

• Study Start: August 1, 2014
• Primary Completion (Actual): July 1, 2016
• Study Completion (Actual): July 1, 2016

Study Registration Dates

• First Submitted: September 8, 2014
• First Submitted That Met QC Criteria: September 8, 2014
• First Posted (Estimate): September 10, 2014

Study Record Updates

• Last Update Posted (Actual): October 12, 2017
• Last Update Submitted That Met QC Criteria: October 10, 2017
• Last Verified: October 1, 2017

More Information

Terms related to this study

• Keywords: Safety; Efficacy; Neurodegenerative disorder; Brain injury; Umbilical cord blood; Granulocyte-colony stimulating factor
• Additional Relevant MeSH Terms: Metabolic Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Brain Damage, Chronic; Neuromuscular Diseases; Parkinsonian Disorders; Basal Ganglia Diseases; Movement Disorders; Synucleinopathies; Craniocerebral Trauma; Trauma, Nervous System; Spinal Cord Diseases; TDP-43 Proteinopathies; Proteostasis Deficiencies; Cerebral Palsy; Parkinson Disease; Brain Injuries; Wounds and Injuries; Motor Neuron Disease; Amyotrophic Lateral Sclerosis; Neurodegenerative Diseases; Physiological Effects of Drugs; Immunologic Factors; Adjuvants, Immunologic; Lenograstim
• Other Study ID Numbers: UCBnG-CSF