Safety and Tolerability of HSC835 in Patients With Hematological Malignancies

Study Overview
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Brief Summary
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This study evaluated the safety and tolerability of using HSC835 in patients with hematological malignancies.

Official Title
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A First-in-human, Single-arm, Single-center, Open-label, Proof-of-concept Study to Evaluate the Safety and Tolerability of Infusing HSC835 (LFU835-expanded Umbilical Cord Blood Hematopoietic Stem Cells) in Patients With Hematological Malignancies

Conditions
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Burkitt's Lymphoma, Large-cell Lymphoma, Acute Myelocytic Leukemia, Acute Lymphocytic Leukemia, Chronic Myelogenous Leukemia, Myelodysplastic Syndrome, Chronic Lymphocytic Leukemia, Marginal Zone Lymphoma, Follicular Lymphomas, Lymphoblastic Lymphoma, High Grade Lymphomas, Mantle-cell Lymphoma, Lymphoplasmacytic Lymphoma

Intervention / Treatment
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* Biological: HSC835


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Patients with a diagnosis that qualifies them for a DUCBT Absence of recent active mold infection 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 Extensive prior chemotherapy Prior myeloablative allotransplantation or autologous transplant.

Ages Eligible for Study
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Minimum Age: 10 Years
Maximum Age: 55 Years

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: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: HSC835<br>HSC835 infusion | Biological: HSC835<br> <br> |

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Safety and Tolerability of HSC835 for Clinical Use Were Measured by Infusional Toxicity (Within First 48 Hours After Transplant) and Absence of Graft Failure After 32 Days in Excess of That Currently Observed With UCBT. | The safety and tolerability of HSC835 for clinical use were measured by infusional toxicity and absence of graft failure in excess of that currently observed with UCBT. Infusional toxicity - AE from transplant until first 48 hours. Administration of the HSC835 expanded CD34-positive cell product, infused over a period of approximately 15 minutes may theoretically cause adverse reactions based on hemodynamic effects, the release of factors like cytokines through administration into the systemic circulation, or acute hypersensitivity, among others. | 32 days | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Incidence of Neutrophil Recovery Within 42 Days | Neutrophil recovery (engraftment) is defined as the first of three consecutive days with ANC > 0.5 x 109/L which occurred for all patients before 42 days post transplant. | 42 days | 
| Incidence of Platelet Recovery Within Six Months | Incidence of platelet recovery within six months. Number of participants recovering platelet to ≥50,000 × 109/L for at least one week without transfusion in the prior 7 days to the first measurement. | 6 months | 
| Frequency of Expanded Unit Predominance at Day 100 (DUCBT Recipients Only) | Frequency of expanded unit predominance at day 100 (DUCBT recipients only) unit predominance was assessed by differences in microsatellite patterns between the recipient, HSC835 and the unmanipulated cord blood unit. Evaluation of sorted CD15-positive/CD33-positive myeloid and CD3-positive T cells in the peripheral blood, revealed three patterns: predominance of HSC835, Mixed dominance an unique chimerism pattern was observed with the CD15/CD33 population predominantly derived from HSC835 and the CD3 population almost exclusively derived from the unmanipulated unit, and predominance of the unmanipulated unit | Day 100 | 
| Incidence of Transplant Related Mortality (TRM) Within 100 Days and One Year | Number of participants with incidence of transplant related mortality (TRM) within 100 days and one year | Day 100 and Month 12 | 
| Incidence of Acute Graft Versus Host Disease (aGVHD) Within 100 Days and Chronic Graft Versus Host Disease (cGVHD) Within 1 Year | Number of participants with incidence of Acute Graft Versus Host Disease (aGVHD) within 100 days and Chronic Graft Versus Host Disease (cGVHD) within 1 year | Day 100 and Monnth 12 | 
| Incidence of Relapse Within One Year | Number of participants with Incidence of relapse within one year | Month 12 | 
| Overall Survival (OS) Within One Year | Number of participants with Overall survival (OS) within one year | Month 12 | 
| Disease Free Survival (DFS) Within One Year | Number of participants with Disease Free Survival (DFS) within one year. Patients are considered to have achieved DFS or relapse-free survival if they had not experienced either relapse or death (of any cause) | Month 12 | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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leukemia, hematologic malignancies, lymphoma