NCT03885947

VPA Expanded UCB Transplantation for Treatment of Patients With Hematological Malignancies

Phase I Study of Valproic Acid Expanded Cord Blood Stem Cells as an Allogeneic Donor Source for Adults With Hematological Malignancies

• Status: Completed
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 7 (actual)
• Sponsor: Alla Keyzner · Academic / Other
• Sex: All
• Age: 18 Years – 65 Years
• Healthy volunteers: Not accepted

Summary

In this Phase I study, the study team will evaluate the safety of Valproic Acid (VPA) expanded cord blood stem cells defined by the lack of serious infusion reactions or graft failure in patients with hematological malignancies undergoing umbilical cord blood transplantation. Moreover, the study team will also evaluate time to neutrophil and platelet engraftment as well as transplant related outcomes such as graft versus host disease (GVHD), treatment related mortality (TRM), and overall survival (OS).

Detailed description

This is a phase I trial for safety of VPA expanded cord blood stem cells in patients with hematological malignancies undergoing allogeneic stem cell transplantation. The primary endpoint of the study is safety as defined by the incidence of infusion reactions and graft failure, lack of neutrophil engraftment by day +42. The trial will consist of two cohorts. First cohort of 5-7 patients, will undergo double umbilical cord blood (UCB) transplantation. One UCB unit will undergo CD34 selection followed VPA based expansion. CD34 negative portion of that unit will be cryopreserved to be infused later following infusion of the expanded portion. Infusion of the second unmanipulated UCB will follow it. Preparative regimen is Fludarabine 150 mg/m2/Cytoxan 50 mg/m2/Thiotepa 10 mg/m2/TBI 400cGy. Following successful engraftment in the first cohort, second cohort (10 patients) will only receive single manipulated unit. Otherwise, patients will receive standard allogeneic stem cell transplantation care.

Conditions

• Hematological Malignancy
• Acute Leukemia in Remission
• Acute Lymphoblastic Leukemia in Remission
• Myelodysplastic Syndromes
• Non-Hodgkin Lymphoma
• Hodgkin Lymphoma

Interventions

Type	Name	Description
BIOLOGICAL	Cord blood stem cells	CD34 selected VPA expanded umbilical cord blood cells used in combination with or without unmanipulated umbilical cord blood .
DRUG	Valproic Acid	Valproic Acid (VPA) expanded cord blood stem cells
DRUG	Fludarabine	Fludarabine 150 mg/m2
DRUG	cytoxan	Cytoxan 50 mg/m2
DRUG	Thiotepa	Thiotepa 10 mg/m2
BIOLOGICAL	TBI	TBI 400cGy

Timeline

• Start date: 2019-02-21
• Primary completion: 2021-03-10
• Completion: 2021-03-10
• First posted: 2019-03-22
• Last updated: 2021-05-24

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study