Safety and Tolerability of HSC835 in Patients With Hematological Malignancies

December 9, 2020 updated by: Novartis Pharmaceuticals

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

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

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

27

Phase

  • Phase 2
  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • Minnesota
      • Minneapolis, Minnesota, United States, 55455
        • Novartis Investigative Site

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

10 years to 55 years (Child, Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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 Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: HSC835
HSC835 infusion
Biological: HSC835

What is the study measuring?

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.
Time Frame: 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.
32 days

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Incidence of Neutrophil Recovery Within 42 Days
Time Frame: 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
Time Frame: 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.
6 months
Frequency of Expanded Unit Predominance at Day 100 (DUCBT Recipients Only)
Time Frame: 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
Day 100
Incidence of Transplant Related Mortality (TRM) Within 100 Days and One Year
Time Frame: Day 100 and Month 12
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
Time Frame: 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
Day 100 and Monnth 12
Incidence of Relapse Within One Year
Time Frame: Month 12
Number of participants with Incidence of relapse within one year
Month 12
Overall Survival (OS) Within One Year
Time Frame: Month 12
Number of participants with Overall survival (OS) within one year
Month 12
Disease Free Survival (DFS) Within One Year
Time Frame: 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)
Month 12

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Novartis Pharmaceuticals

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

January 9, 2012

Primary Completion (Actual)

October 3, 2016

Study Completion (Actual)

October 3, 2016

Study Registration Dates

First Submitted

November 15, 2011

First Submitted That Met QC Criteria

November 17, 2011

First Posted (Estimate)

November 18, 2011

Study Record Updates

Last Update Posted (Actual)

December 30, 2020

Last Update Submitted That Met QC Criteria

December 9, 2020

Last Verified

March 1, 2019

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • CHSC835X2201

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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