Safety and Tolerability of HSC835 in Patients With Hematological Malignancies

Chronic Lymphocytic Leukemia Clinical Trial

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01474681
• Other study ID #: CHSC835X2201
• Status: Completed
• Phase: Phase 1/Phase 2
• Start date: January 9, 2012
• Est. completion date: October 3, 2016

Study information

• Verified date: March 2019
• Source: Novartis
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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

Recruitment information / eligibility

• Status: Completed
• Enrollment: 27
• Est. completion date: October 3, 2016
• Est. primary completion date: October 3, 2016
• Accepts healthy volunteers: No
• Gender: All
• Age group: 10 Years to 55 Years

Eligibility

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.

Related Conditions & MeSH terms

• Acute Lymphocytic Leukemia
• Acute Myelocytic Leukemia
• Burkitt Lymphoma
• Burkitt's Lymphoma
• Chronic Lymphocytic Leukemia
• Chronic Myelogenous Leukemia
• Follicular Lymphomas
• Hematologic Neoplasms
• High Grade Lymphomas
• Large-cell Lymphoma
• Leukemia
• Leukemia, Lymphocytic, Chronic, B-Cell
• Leukemia, Lymphoid
• Leukemia, Myelogenous, Chronic, BCR-ABL Positive
• Leukemia, Myeloid
• Leukemia, Myeloid, Acute
• Lymphoblastic Lymphoma
• Lymphoma
• Lymphoma, Mantle-Cell
• Lymphoma, Non-Hodgkin
• Lymphoplasmacytic Lymphoma
• Mantle-cell Lymphoma
• Marginal Zone Lymphoma
• Myelodysplastic Syndrome
• Myelodysplastic Syndromes
• Neoplasms
• Precursor Cell Lymphoblastic Leukemia-Lymphoma
• Waldenstrom Macroglobulinemia

Intervention

Biological:
• HSC835

Locations

Country	Name	City	State
United States	Novartis Investigative Site	Minneapolis	Minnesota

Sponsors (1)

Lead Sponsor	Collaborator
Novartis Pharmaceuticals

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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.	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	Incidence of Neutrophil Recovery Within 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
Secondary	Incidence of Platelet Recovery Within Six 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
Secondary	Frequency of Expanded Unit Predominance at Day 100 (DUCBT Recipients Only)	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
Secondary	Incidence of Transplant Related Mortality (TRM) Within 100 Days and One Year	Number of participants with incidence of transplant related mortality (TRM) within 100 days and one year	Day 100 and Month 12
Secondary	Incidence of Acute Graft Versus Host Disease (aGVHD) Within 100 Days and Chronic Graft Versus Host Disease (cGVHD) Within 1 Year	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
Secondary	Incidence of Relapse Within One Year	Number of participants with Incidence of relapse within one year	Month 12
Secondary	Overall Survival (OS) Within One Year	Number of participants with Overall survival (OS) within one year	Month 12
Secondary	Disease Free Survival (DFS) Within One Year	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

External Links

•   A Plain Language Trial Summary is available on novartisclinicaltrials.com