Safety and Tolerability of HSC835 in Patients With Hematological Malignancies Undergoing Single Umbilical Cord Blood Transplant (UCBT)
Primary Purpose
Single Umbilical Cord Blood Transplantation, Non-myeloablative Conditioning, Acute Lymphocytic Leukemia
Status
Completed
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
HSC835
Sponsored by
About this trial
This is an interventional basic science trial for Single Umbilical Cord Blood Transplantation focused on measuring HSC835, single umbilical cord blood transplantation, non-myeloablative conditioning regimen, Umbilical cord blood, UCB transplantation
Eligibility Criteria
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
Sites / Locations
- Novartis Investigative Site
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
HSC835
Arm Description
Patients with hematologic malignancies requiring UCB transplant with a NMA conditioning regimen.
Outcomes
Primary Outcome Measures
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.
Secondary Outcome Measures
Incidence of Neutrophil Recovery Within 42 Days
Engraftment is defined as the first of three consecutive days with ANC > 0.5 x 109/L.
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.
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.
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.
Full Information
NCT ID
NCT01930162
First Posted
August 23, 2013
Last Updated
December 9, 2020
Sponsor
Novartis Pharmaceuticals
1. Study Identification
Unique Protocol Identification Number
NCT01930162
Brief Title
Safety and Tolerability of HSC835 in Patients With Hematological Malignancies Undergoing Single Umbilical Cord Blood Transplant
Acronym
UCBT
Official Title
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
Study Type
Interventional
2. Study Status
Record Verification Date
August 2017
Overall Recruitment Status
Completed
Study Start Date
October 7, 2014 (Actual)
Primary Completion Date
August 29, 2016 (Actual)
Study Completion Date
August 29, 2016 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Novartis Pharmaceuticals
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes
5. Study Description
Brief Summary
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Single Umbilical Cord Blood Transplantation, Non-myeloablative Conditioning, Acute Lymphocytic Leukemia, Myelodysplastic Syndrome, Non-Hodgkin's Lymphoma, Multiple Myeloma, Chronic Lymphocytic Leukemia
Keywords
HSC835, single umbilical cord blood transplantation, non-myeloablative conditioning regimen, Umbilical cord blood, UCB transplantation
7. Study Design
Primary Purpose
Basic Science
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
9 (Actual)
8. Arms, Groups, and Interventions
Arm Title
HSC835
Arm Type
Experimental
Arm Description
Patients with hematologic malignancies requiring UCB transplant with a NMA conditioning regimen.
Intervention Type
Drug
Intervention Name(s)
HSC835
Intervention Description
HSC835 is a stem cell therapy product providing a source of Hematopoietic stem cell (HSC).
Primary Outcome Measure Information:
Title
Absence of Graft Failure at Day 42
Description
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.
Time Frame
42 days
Secondary Outcome Measure Information:
Title
Incidence of Neutrophil Recovery Within 42 Days
Description
Engraftment is defined as the first of three consecutive days with ANC > 0.5 x 109/L.
Time Frame
42 days
Title
Incidence of Non-relapse Mortality (NRM) Within 100 Days and One Year
Description
NRM includes all patients who died from any other cause except relapse of the underlying disease during the study duration.
Time Frame
1 year
Title
Incidence of Overall Survival Within One Year
Description
Overall survival is the proportion of patients who were alive at the end of the one year study period.
Time Frame
1 year
Title
Incidence of Relapse-free Survival Within One Year
Description
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.
Time Frame
1 year
10. Eligibility
Sex
All
Accepts Healthy Volunteers
No
Eligibility Criteria
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
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
Plan to Share IPD
Yes
IPD Sharing Plan Description
Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations.
This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com
Links:
URL
https://www.novctrd.com/ctrdweb/patientsummary/patientsummaries?patientSummaryId=183
Description
A Plain Language Trial Summary is available on novartisclinicaltrials.com
Learn more about this trial
Safety and Tolerability of HSC835 in Patients With Hematological Malignancies Undergoing Single Umbilical Cord Blood Transplant
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