Safety and Tolerability of HSC835 in Patients With Hematological Malignancies

Phase 1

Completed

• Conditions: Chronic Lymphocytic Leukemia; Lymphoblastic Lymphoma; High Grade Lymphomas; Acute Myelocytic Leukemia; Burkitt's Lymphoma; Lymphoplasmacytic Lymphoma; Acute Lymphocytic Leukemia; Chronic Myelogenous Leukemia; Follicular Lymphomas; Myelodysplastic Syndrome
• Interventions: Biological: HSC835

• Registration Number: NCT01474681
• Lead Sponsor: Novartis Pharmaceuticals

Brief Summary

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

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2011-11-15
• Study First Submitted QC: 2011-11-17
• Study First Posted: 2011-11-18
• Study Start: 2012-01-09
• Primary Completion: 2016-10-03
• Study Completion: 2016-10-03
• Results First Submitted: 2017-04-03
• Results First Submitted QC: 2017-04-03
• Results First Posted: 2017-05-12
• Status Verified: 2019-03
• Last Update Submitted: 2020-12-09
• Last Update Posted: 2020-12-30

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 27

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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
HSC835	HSC835	HSC835 infusion

Primary Outcome Measures

Name	Time	Method
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.	32 days	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.

Secondary Outcome Measures

Name	Time	Method
Incidence of Neutrophil Recovery Within 42 Days	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.
Incidence of Platelet Recovery Within Six Months	6 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.
Frequency of Expanded Unit Predominance at Day 100 (DUCBT Recipients Only)	Day 100	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
Incidence of Transplant Related Mortality (TRM) Within 100 Days and One Year	Day 100 and Month 12	Number of participants with incidence of transplant related mortality (TRM) within 100 days and one year
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	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
Incidence of Relapse Within One Year	Month 12	Number of participants with Incidence of relapse within one year
Overall Survival (OS) Within One Year	Month 12	Number of participants with Overall survival (OS) within one year
Disease Free Survival (DFS) Within One Year	Month 12	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)

Trial Locations

Locations (1)

Novartis Investigative Site; 🇺🇸 Minneapolis, Minnesota, United States