NCT03130816

Mechanism of Allogeneic UCB Therapy in Cerebral Palsy

Mechanism of Allogeneic Umbilical Cord Blood Therapy in Cerebral Palsy

• Status: Completed
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 90 (estimated)
• Sponsor: Bundang CHA Hospital · Academic / Other
• Sex: All
• Age: 10 Months – 20 Years
• Healthy volunteers: Not accepted

Summary

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

Conditions

• Cerebral Palsy
• Child Development

Interventions

Type	Name	Description
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.

Timeline

• Start date: 2015-07-29
• Primary completion: 2019-05-21
• Completion: 2019-05-21
• First posted: 2017-04-27
• Last updated: 2020-11-04

Locations

1 site across 1 country: South Korea