Mechanism of Allogeneic UCB Therapy in Cerebral Palsy
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT03130816|
Recruitment Status : Recruiting
First Posted : April 27, 2017
Last Update Posted : April 27, 2017
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 or disease||Intervention/treatment||Phase|
|Cerebral Palsy Child Development||Biological: allogeneic cord blood transplantation||Phase 1 Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||90 participants|
|Intervention Model:||Single Group Assignment|
|Intervention Model Description:||Patients diagnosed with cerebral palsy volunteered for participation in this study.|
|Masking:||None (Open Label)|
|Official Title:||Mechanism of Allogeneic Umbilical Cord Blood Therapy in Cerebral Palsy|
|Actual Study Start Date :||July 29, 2015|
|Estimated Primary Completion Date :||July 2019|
|Estimated Study Completion Date :||July 2019|
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.
- 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.
- 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.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03130816
|Contact: MinYoung Kim, MD, PhDemail@example.com|
|Korea, Republic of|
|CHA Bundang Medical Center||Recruiting|
|Seongnam, Gyeonggido, Korea, Republic of, 13496|
|Contact: Sun Hee Lee firstname.lastname@example.org|
|Principal Investigator:||MinYoung Kim, MD, PhD||CHA University|