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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 : December 30, 2020
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )

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

Condition or disease Intervention/treatment Phase
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 Phase 1 Phase 2

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 27 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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
Actual Study Start Date : January 9, 2012
Actual Primary Completion Date : October 3, 2016
Actual Study Completion Date : October 3, 2016

Arm Intervention/treatment
Experimental: HSC835
HSC835 infusion
Biological: HSC835

Primary Outcome Measures :
  1. 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.

Secondary Outcome Measures :
  1. 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.

  2. 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.

  3. 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

  4. 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

  5. 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

  6. Incidence of Relapse Within One Year [ Time Frame: Month 12 ]
    Number of participants with Incidence of relapse within one year

  7. Overall Survival (OS) Within One Year [ Time Frame: Month 12 ]
    Number of participants with Overall survival (OS) within one year

  8. 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)

Information from the National Library of Medicine

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Ages Eligible for Study:   10 Years to 55 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT01474681

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United States, Minnesota
Novartis Investigative Site
Minneapolis, Minnesota, United States, 55455
Sponsors and Collaborators
Novartis Pharmaceuticals
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Study Director: Novartis Pharmaceuticals Novartis Pharmaceuticals
Additional Information:
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Responsible Party: Novartis Pharmaceuticals Identifier: NCT01474681    
Other Study ID Numbers: CHSC835X2201
First Posted: November 18, 2011    Key Record Dates
Results First Posted: May 12, 2017
Last Update Posted: December 30, 2020
Last Verified: March 2019
Keywords provided by Novartis ( Novartis Pharmaceuticals ):
hematologic malignancies
Additional relevant MeSH terms:
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Burkitt Lymphoma
Leukemia, Lymphoid
Leukemia, Lymphocytic, Chronic, B-Cell
Leukemia, Myeloid
Lymphoma, Mantle-Cell
Leukemia, Myelogenous, Chronic, BCR-ABL Positive
Precursor Cell Lymphoblastic Leukemia-Lymphoma
Hematologic Neoplasms
Waldenstrom Macroglobulinemia
Leukemia, Myeloid, Acute
Lymphoma, Non-Hodgkin
Myelodysplastic Syndromes
Neoplasms by Histologic Type
Lymphoproliferative Disorders
Lymphatic Diseases
Immunoproliferative Disorders
Immune System Diseases
Bone Marrow Diseases
Hematologic Diseases
Leukemia, B-Cell
Myeloproliferative Disorders
Lymphoma, B-Cell
Epstein-Barr Virus Infections
Herpesviridae Infections
DNA Virus Infections
Virus Diseases
Tumor Virus Infections