Trials / Completed

CompletedNCT00469729

Efficacy and Safety Study of StemEx®, to Treat Subjects With High Risk Hematologic Malignancies, Following Myeloablative Therapy

A Multi-Center, Multi-National, Historical Cohort Controlled Study to Evaluate Efficacy and Safety of Transplantation of StemEx®, Umbilical Cord Blood Stem and Progenitor Cells Expanded Ex Vivo, in Subjects With Hematologic Malignancies Following Myeloablative Therapy

Status: Completed
Phase: Phase 2 / Phase 3
Study type: Interventional
Enrollment: 101 (actual)

Sponsor: Gamida Cell -Teva Joint Venture Ltd. · Academic / Other
Sex: All
Age: 12 Years – 55 Years
Healthy volunteers: Not accepted

Summary

The purpose of this study is to determine the efficacy and safety of transplanting StemEx® in patients with certain hematological malignancies. For these patients, it is suggested that StemEx® can improve upon the outcome of transplanting a single, unmanipulated cord blood unit by significantly increasing the number of stem/progenitor cells available to the patient.

Detailed description

Allogeneic hematopoietic stem cell transplantation is a life-saving procedure for patients with hematologic malignancies; yet wide application of this procedure is limited by the availability of suitably Human Leukocyte Antigen (HLA) - matched donors. Only 30% of patients who could benefit from this procedure have an HLA-matched sibling. The lengthy search for a matched donor may critically delay transplantation. In addition, far fewer patients of racial minorities find suitable HLA-matched donors. Umbilical cord blood (UCB) has been increasingly used as an alternative source of stem cells; however, its use in adults and adolescent patients is limited due to insufficient cell dose required for satisfactory hematopoietic reconstitution. Gamida Cell - Teva Joint Venture Ltd. is engaged in the development of StemEx®, an expanded hematopoietic UCB stem cell graft, as a potential medicinal product for the treatment of cancer and hematological malignancies. The expansion technology enables preferential expansion of hematopoietic stem and early progenitor cells and is based on the findings that copper chelators can regulate the balance between self-renewal and differentiation of stem cells. The multi-national, multi-center Phase II/III clinical study designated to evaluate the safety and efficacy of StemEx® will enroll approximately 100 subjects with high-risk hematologic malignancies who are candidates for allogeneic stem cell transplantation (SCT). This study will evaluate the effect of StemEx® on overall survival as measured by overall 100-day mortality. The study consists of 4 phases: 1. Screening phase includes subjects' clinical assessment and screening tests 2. Conditioning phase includes the myeloablative treatment prior transplantation procedure 3. Transplantation and post-transplant follow-up phase to day 180 4. Observational phase: survival status follow-up to day 730 (18 months)

Conditions

Interventions

Type	Name	Description
DRUG	StemEx®	The stem/progenitor cell based product composed of ex vivo expanded allogeneic umbilical cord blood cells, which is infused to subject at a rate of 1-3 ml/min in combination with non-manipulated cells derived from the same cord blood unit.

Timeline

Start date: 2007-10-01
Primary completion: 2013-02-01
Completion: 2015-06-01
First posted: 2007-05-04
Last updated: 2015-07-10

Locations

30 sites across 5 countries: United States, Hungary, Israel, Italy, Spain

Source: ClinicalTrials.gov record NCT00469729. Inclusion in this directory is not an endorsement.