Safety and Tolerability of HSC835 in Patients With Hematological Malignancies

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT01474681
Recruitment Status : Completed
First Posted : November 18, 2011
Results First Posted : May 12, 2017
Last Update Posted : May 12, 2017
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )

November 15, 2011
November 18, 2011
April 3, 2017
May 12, 2017
May 12, 2017
January 9, 2012
October 3, 2016   (Final data collection date for primary outcome measure)
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.
Safety and Tolerability: Safety and tolerability will be measured by occurrence of infusional toxicity or occurrence of graft failure [ Time Frame: 30 days ]
Complete list of historical versions of study NCT01474681 on Archive Site
  • 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.
  • 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.
  • 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
  • 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
  • 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
  • Incidence of Relapse Within One Year [ Time Frame: Month 12 ]
    Number of participants with Incidence of relapse within one year
  • Overall Survival (OS) Within One Year [ Time Frame: Month 12 ]
    Number of participants with Overall survival (OS) within one year
  • 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)
  • Incidence of neutrophil recovery [ Time Frame: 42 days ]
  • Incidence of platelet recovery [ Time Frame: 6 months ]
  • Incidence of mortality, acute graft versus host disease, chronic graft versus host disease, relapse, overall survival, disease-free survival [ Time Frame: 100 days ]
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Safety and Tolerability of HSC835 in Patients With Hematological Malignancies
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.
Not Provided
Phase 1
Phase 2
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
  • 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
Biological: HSC835
Experimental: HSC835
HSC835 infusion
Intervention: Biological: HSC835
Not Provided

*   Includes publications given by the data provider as well as publications identified by Identifier (NCT Number) in Medline.
October 3, 2016
October 3, 2016   (Final data collection date for primary outcome measure)

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.
Sexes Eligible for Study: All
10 Years to 55 Years   (Child, Adult)
Contact information is only displayed when the study is recruiting subjects
United States
Not Provided
Not Provided
Novartis ( Novartis Pharmaceuticals )
Novartis Pharmaceuticals
Not Provided
Study Director: Novartis Pharmaceuticals Novartis Pharmaceuticals
March 2017

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP