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Mechanism of Allogeneic UCB Therapy in Cerebral Palsy

This study is currently recruiting participants.
See Contacts and Locations
Verified April 2017 by MinYoung Kim, M.D., Bundang CHA Hospital
Sponsor:
Information provided by (Responsible Party):
MinYoung Kim, M.D., Bundang CHA Hospital
ClinicalTrials.gov Identifier:
NCT03130816
First received: April 20, 2017
Last updated: April 23, 2017
Last verified: April 2017
  Purpose

In our prior study on the therapeutic mechanism of UCB, changes in cytokine levels were observed but the results are inconclusive and further studies on animal models and changes of protein expression before and after UCB therapy in the clinical settings are required.

The changes in protein expression will be assessed by multiplex RT-PCR mRNA assay. Clinical efficacy of UCB therapy will be evaluated with various functional assessment tools. Factors regarding UCB therapy (number of transplanted cells, HLA matching status, serum level of immunosuppressant, etc.) and patient factors (age, functional status, etc.) will be analyzed for correlation with protein expression after UCB therapy. Several target proteins for analysis are available. Pentraxin and toll-like receptor (TLR) 4 are receptors modulating intrinsic immune reaction and was shown to have a significant correlation with clinical efficacy of stem cell therapy. Ubiquitine is a regulatory protein that combines with the target protein and affects its degradation, interaction, localization and activation. The ubiquitine system controls total protein quantity for homeostasis and can be found in all tissues. Deubiquitination (DUB) enzyme down-regulates this ubiquitine and is known to modulate all cellular changes


Condition Intervention Phase
Cerebral Palsy Child Development Biological: allogeneic cord blood transplantation Phase 1 Phase 2

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Intervention Model Description:
Patients diagnosed with cerebral palsy volunteered for participation in this study.
Masking: No masking
Primary Purpose: Treatment
Official Title: Mechanism of Allogeneic Umbilical Cord Blood Therapy in Cerebral Palsy

Resource links provided by NLM:


Further study details as provided by MinYoung Kim, M.D., Bundang CHA Hospital:

Primary Outcome Measures:
  • Change of GMFM [ Time Frame: Baseline before UCB administration, months 3, 6, and 12 after UCB treatment ]
    Gross motor function measure measured at baseline before UCB administration is compared to the score measured at months 3, 6, and 12 after UCB treatment.


Secondary Outcome Measures:
  • Change of mRNA assay [ Time Frame: Change between the baseline level before UCB therapy and levels after UCB administration at 2 days, 1 week, 5 weeks, and 12 months ]
    Separate peripheral blood mononuclear cell (PBMC) from the patients' blood sample and screen for changes in protein enzymes including those related to DUB at the mRNA level after UCB therapy.

  • Change of GMPM [ Time Frame: Baseline before UCB administration, months 3, 6, and 12 after UCB treatment ]
    Dissociated Movement, Coordination, Alignment, Weight shift, and Stability are rated with GMPM (Gross Motor Performance Measure). Each raw score (1-5 point) and converted percent scores are evaluated. Total score is 100(%), higher scores indicate better function. GMPM measured at baseline before UCB administration is compared to the score measured at months 3, 6, and 12 after UCB treatment.


Estimated Enrollment: 90
Actual Study Start Date: July 29, 2015
Estimated Study Completion Date: July 2019
Estimated Primary Completion Date: July 2019 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: allogeneic cord blood transplantation

Intravenous(IV) infusion will be done by the following method A. After 4 hours of fasting, subjects will be sedated with chloral hydrate (Pocral®) syrup B. Intravenous infusion will be conducted in stem cell center, CHA Bundang Medical Center and the therapy will be performed by the Principal Investigator or a physician delegated from the Principal Investigator. The physician conducting the infusion will not participate in the efficacy and result analysis of this study.

C. Oxygen saturation will be monitored during therapy.

Biological: allogeneic cord blood transplantation
UCB with total nucleated cell count ≤ 7x108/kg will be used for this clinical trial. Suitable UCB (i.e., containing total nucleated cell count ≥2x107/kg with three or less mismatch among HLA-A, -B, and -DR) will be selected. This criterion was selected upon the rationale that even though minimal HLA mismatch is preferred, prior studies indicate significant effects of UCB therapy for patients with 3 HLA mismatches.

  Eligibility

Ages Eligible for Study:   10 Months to 20 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Diagnosed with cerebral palsy
  2. Age of ≥10 months and ≤20 years
  3. Mismatch in HLA-A, B, and DR ≤3, and total nucleated cell count ≥2x107/kg. If the cell count is less than given values, more than 2 units may be used.
  4. Voluntary decision to participation in the study with informed consent agreed and obtained from the subject's representative.
  5. Patient and/or representatives are both willing and capable of being hospitalized according to the schedule specified in the protocol and continue the study for 12 months after study entry.
  6. If the patient has participated in another clinical trial, at least 3 months should have passed since end of the study.

Exclusion Criteria:

  1. Current aspiration pneumonia
  2. Known genetic disease
  3. History of hypersensitivity reaction to any study drugs pertinent to the study
  4. Patient with severe convulsion disease who has clinical convulsion despite combination therapy with 3 or more agents
  5. Uncontrolled hypertension defined as systolic blood pressure >115 mmHg and/or diastolic blood pressure >70 mmHg
  6. Hepatic impairment defined as asparate aminotransferase (AST) >55 IU/L and/or alanine aminotrasferase (ALT) >45 IU/L
  7. Renal impairment defined as creatinine (Cr) ≥1.3 mg/dL
  8. Presence of diagnosed or suspected malignant tumor and/or hematologic malignancy
  9. Non-compliance with study visits specified in the protocol or poor compliance of care-giver.
  10. Any factors not specified above that the principal investigator determines medically inadequate for participation in this study.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT03130816

Contacts
Contact: MinYoung Kim, MD, PhD kmin@cha.ac.kr

Locations
Korea, Republic of
CHA Bundang Medical Center Recruiting
Seongnam, Gyeonggido, Korea, Republic of, 13496
Contact: Sun Hee Lee       murkogi0@chamc.co.kr   
Sponsors and Collaborators
Bundang CHA Hospital
Investigators
Principal Investigator: MinYoung Kim, MD, PhD CHA University
  More Information

Responsible Party: MinYoung Kim, M.D., Professor, Bundang CHA Hospital
ClinicalTrials.gov Identifier: NCT03130816     History of Changes
Other Study ID Numbers: 2015-06-093
Study First Received: April 20, 2017
Last Updated: April 23, 2017
Individual Participant Data  
Plan to Share IPD: Undecided

Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by MinYoung Kim, M.D., Bundang CHA Hospital:
Umbilical cord blood transplantation
Cytokine changes

Additional relevant MeSH terms:
Paralysis
Cerebral Palsy
Neurologic Manifestations
Nervous System Diseases
Signs and Symptoms
Brain Damage, Chronic
Brain Diseases
Central Nervous System Diseases

ClinicalTrials.gov processed this record on June 27, 2017