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Safety and Tolerability of HSC835 in Patients With Hematological Malignancies

Primary Purpose

Acute Myelocytic Leukemia, Acute Lymphocytic Leukemia, Chronic Myelogenous Leukemia

Status
Completed
Phase
Phase 1
Locations
United States
Study Type
Interventional
Intervention
HSC835
Sponsored by
Novartis Pharmaceuticals
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Acute Myelocytic Leukemia focused on measuring hematologic malignancies, leukemia, lymphoma

Eligibility Criteria

10 Years - 55 Years (Child, Adult)All SexesDoes not accept healthy volunteers

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.

Sites / Locations

  • Novartis Investigative Site

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

HSC835

Arm Description

HSC835 infusion

Outcomes

Primary Outcome Measures

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.

Secondary Outcome Measures

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.
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.
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
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
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
Incidence of Relapse Within One Year
Number of participants with Incidence of relapse within one year
Overall Survival (OS) Within One Year
Number of participants with Overall survival (OS) within one year
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)

Full Information

First Posted
November 15, 2011
Last Updated
December 9, 2020
Sponsor
Novartis Pharmaceuticals
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1. Study Identification

Unique Protocol Identification Number
NCT01474681
Brief Title
Safety and Tolerability of HSC835 in Patients With Hematological Malignancies
Official Title
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
Study Type
Interventional

2. Study Status

Record Verification Date
March 2019
Overall Recruitment Status
Completed
Study Start Date
January 9, 2012 (Actual)
Primary Completion Date
October 3, 2016 (Actual)
Study Completion Date
October 3, 2016 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Novartis Pharmaceuticals

4. Oversight

Data Monitoring Committee
Yes

5. Study Description

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

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Acute Myelocytic Leukemia, Acute Lymphocytic Leukemia, Chronic Myelogenous Leukemia, Myelodysplastic Syndrome, Chronic Lymphocytic Leukemia, Marginal Zone Lymphoma, Follicular Lymphomas, Large-cell Lymphoma, Lymphoblastic Lymphoma, Burkitt's Lymphoma, High Grade Lymphomas, Mantle-cell Lymphoma, Lymphoplasmacytic Lymphoma
Keywords
hematologic malignancies, leukemia, lymphoma

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 1, Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
27 (Actual)

8. Arms, Groups, and Interventions

Arm Title
HSC835
Arm Type
Experimental
Arm Description
HSC835 infusion
Intervention Type
Biological
Intervention Name(s)
HSC835
Primary Outcome Measure Information:
Title
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.
Description
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.
Time Frame
32 days
Secondary Outcome Measure Information:
Title
Incidence of Neutrophil Recovery Within 42 Days
Description
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.
Time Frame
42 days
Title
Incidence of Platelet Recovery Within Six Months
Description
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.
Time Frame
6 months
Title
Frequency of Expanded Unit Predominance at Day 100 (DUCBT Recipients Only)
Description
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
Time Frame
Day 100
Title
Incidence of Transplant Related Mortality (TRM) Within 100 Days and One Year
Description
Number of participants with incidence of transplant related mortality (TRM) within 100 days and one year
Time Frame
Day 100 and Month 12
Title
Incidence of Acute Graft Versus Host Disease (aGVHD) Within 100 Days and Chronic Graft Versus Host Disease (cGVHD) Within 1 Year
Description
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
Time Frame
Day 100 and Monnth 12
Title
Incidence of Relapse Within One Year
Description
Number of participants with Incidence of relapse within one year
Time Frame
Month 12
Title
Overall Survival (OS) Within One Year
Description
Number of participants with Overall survival (OS) within one year
Time Frame
Month 12
Title
Disease Free Survival (DFS) Within One Year
Description
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)
Time Frame
Month 12

10. Eligibility

Sex
All
Minimum Age & Unit of Time
10 Years
Maximum Age & Unit of Time
55 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Novartis Pharmaceuticals
Organizational Affiliation
Novartis Pharmaceuticals
Official's Role
Study Director
Facility Information:
Facility Name
Novartis Investigative Site
City
Minneapolis
State/Province
Minnesota
ZIP/Postal Code
55455
Country
United States

12. IPD Sharing Statement

Links:
URL
https://www.novctrd.com/ctrdweb/patientsummary/patientsummaries?patientSummaryId=185
Description
A Plain Language Trial Summary is available on novartisclinicaltrials.com

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Safety and Tolerability of HSC835 in Patients With Hematological Malignancies

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