Safety and Tolerability of HSC835 in Patients With Hematological Malignancies Undergoing Single Umbilical Cord Blood Transplant

Study Overview
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Brief Summary
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This study was designed to evaluate the safety and tolerability of HSC835 for clinical use as measured by the absence of graft failure at day 42 in excess of that currently observed with double umbilical cord blood (UCB) transplantation (DUCBT) with non-myeloablative (NMA) conditioning.

Official Title
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A Single-arm, Open-label Study to Evaluate the Safety and Tolerability of Infusing HSC835 in Patients With Hematological Malignancies Undergoing UCB Transplantation Using a Non-myeloablative Conditioning Regimen

Conditions
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Single Umbilical Cord Blood Transplantation, Non-myeloablative Conditioning, Acute Lymphocytic Leukemia, Myelodysplastic Syndrome, Non-Hodgkin's Lymphoma, Multiple Myeloma, Chronic Lymphocytic Leukemia

Intervention / Treatment
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* Drug: HSC835


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Patients with a diagnosis that qualifies them for UCBT Adequate organ function Availability of eligible donor material Exclusion Criteria: Pregnancy or breastfeeding women and women of child-bearing potential unless two acceptable forms of contraception are being used Human immunodeficiency virus (HIV) infection Active infection
Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Basic Science
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: HSC835<br>Patients with hematologic malignancies requiring UCB transplant with a NMA conditioning regimen. | Drug: HSC835<br>* HSC835 is a stem cell therapy product providing a source of Hematopoietic stem cell (HSC).<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Absence of Graft Failure at Day 42 | This endpoint was to study safety and tolerability of HSC835 as measured by the absence of graft failure at day 42 in excess of that currently observed with double umbilical cord blood (UCB) transplantation (DUCBT) with non-myeloablative (NMA) conditioning. | 42 days | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Incidence of Neutrophil Recovery Within 42 Days | Engraftment is defined as the first of three consecutive days with ANC > 0.5 x 109/L. | 42 days | 
| Incidence of Non-relapse Mortality (NRM) Within 100 Days and One Year | NRM includes all patients who died from any other cause except relapse of the underlying disease during the study duration. | 1 year | 
| Incidence of Overall Survival Within One Year | Overall survival is the proportion of patients who were alive at the end of the one year study period. | 1 year | 
| Incidence of Relapse-free Survival Within One Year | Patients are considered to have achieved relapse-free survival if they had not experienced either relapse or death (of any cause) at the end of the study. | 1 year | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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HSC835, single umbilical cord blood transplantation, non-myeloablative conditioning regimen, Umbilical cord blood, UCB transplantation