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Safety and Tolerability of HSC835 in Patients With Hematological Malignancies

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )
ClinicalTrials.gov Identifier:
NCT01474681
First received: November 15, 2011
Last updated: April 3, 2017
Last verified: March 2017
  Purpose
This study evaluated the safety and tolerability of using HSC835 in patients with hematological malignancies.

Condition Intervention 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

Study Type: Interventional
Study Design: 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

Resource links provided by NLM:


Further study details as provided by Novartis ( Novartis Pharmaceuticals ):

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. [ 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:
  • 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)


Enrollment: 27
Actual Study Start Date: January 9, 2012
Study Completion Date: October 3, 2016
Primary Completion Date: October 3, 2016 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: HSC835
HSC835 infusion
Biological: HSC835

  Eligibility

Ages Eligible for Study:   10 Years to 55 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01474681

Locations
United States, Minnesota
Novartis Investigative Site
Minneapolis, Minnesota, United States, 55455
Sponsors and Collaborators
Novartis Pharmaceuticals
Investigators
Study Director: Novartis Pharmaceuticals Novartis Pharmaceuticals
  More Information

Responsible Party: Novartis Pharmaceuticals
ClinicalTrials.gov Identifier: NCT01474681     History of Changes
Other Study ID Numbers: CHSC835X2201
Study First Received: November 15, 2011
Results First Received: April 3, 2017
Last Updated: April 3, 2017

Keywords provided by Novartis ( Novartis Pharmaceuticals ):
hematologic malignancies
leukemia
lymphoma

Additional relevant MeSH terms:
Lymphoma
Leukemia
Lymphoma, Follicular
Myelodysplastic Syndromes
Preleukemia
Leukemia, Lymphoid
Leukemia, Lymphocytic, Chronic, B-Cell
Leukemia, Myeloid
Lymphoma, Mantle-Cell
Leukemia, Myelogenous, Chronic, BCR-ABL Positive
Lymphoma, B-Cell, Marginal Zone
Precursor Cell Lymphoblastic Leukemia-Lymphoma
Burkitt Lymphoma
Waldenstrom Macroglobulinemia
Leukemia, Myeloid, Acute
Lymphoma, Non-Hodgkin
Neoplasms by Histologic Type
Neoplasms
Lymphoproliferative Disorders
Lymphatic Diseases
Immunoproliferative Disorders
Immune System Diseases
Bone Marrow Diseases
Hematologic Diseases
Precancerous Conditions
Leukemia, B-Cell
Myeloproliferative Disorders
Lymphoma, B-Cell
Epstein-Barr Virus Infections
Herpesviridae Infections

ClinicalTrials.gov processed this record on September 21, 2017