Efficacy and Safety Study of StemEx®, to Treat Subjects With High Risk Hematologic Malignancies, Following Myeloablative Therapy
- Conditions
- Hematologic MalignanciesAcute Myeloid LeukemiaLymphoid LeukemiaChronic Myeloid LeukemiaHodgkin's DiseaseNon-Hodgkin's LymphomaMyelodysplastic Syndromes
- Interventions
- Drug: StemEx®
- Registration Number
- NCT00469729
- Lead Sponsor
- Gamida Cell -Teva Joint Venture Ltd.
- Brief 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)
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 101
- Clinical diagnosis of AML or ALL: CR2 or subsequent complete remission (CR) or CR1 with high-risk features or relapse with < 10% blasts in BM and no circulating blasts.
- Clinical diagnosis of CML: in CP1 (Chronic Phase 1) and resistant or intolerant to Gleevec or in CP2 or subsequent CP or in accelerated phase.
- Clinical diagnosis of HD: induction failure or relapse and sensitive to last chemotherapy course.
- Clinical diagnosis of NHL induction failure or relapse and sensitive to last chemotherapy course.
- Clinical diagnosis of MDS with intermediate 2- or high-risk IPSS score.
- Less than twenty-one days have elapsed since the subject's last radiation or chemotherapy prior to conditioning (except Hydroxyurea).
- HIV positive.
- Pregnancy or lactation.
- Uncontrolled bacterial, fungal or viral infection.
- Subjects with signs and symptoms of active central nervous system (CNS) disease.
- Availability of appropriate related and willing stem cell donor, who is HLA-matched at 5 or 6/6 antigens.
- Prior allogeneic cell transplant.
- Allergy to bovine or to any product, which may interfere with the treatment.
- Enrolled in another clinical trial or received an investigational treatment during the last 30 days, unless approved by Sponsor.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description StemEx StemEx® -
- Primary Outcome Measures
Name Time Method Overall 100-day mortality 100 days
- Secondary Outcome Measures
Name Time Method 180 day mortality, acute Graft versus Host Disease (GvHD) grades III-IV, engraftment failure 180 days Safety and tolerability measures: The incidence and frequency of adverse experiences, acute toxicity, laboratory data and vital signs follow-up. 180 days Proportion of overall mortality at 1 year One year post transplant Proportion of overall mortality at 2 years Two years post transplant
Trial Locations
- Locations (30)
UCLA's Jonsson Comprehensive Cancer Center
🇺🇸Los Angeles, California, United States
Children's Hospital of Orange County
🇺🇸Orange, California, United States
The Children's Hospital, B115, University of Colorado Health Sciences Center
🇺🇸Aurora, Colorado, United States
Northwestern University School of Medicine, Stem Cell Transplant Program, Children's Memorial Hospital
🇺🇸Chicago, Illinois, United States
Cardinal Bernardin Cancer Center, Loyola University Stritch School of Medicine
🇺🇸Maywood, Illinois, United States
The Cancer Center at Hackensack University Medical Center
🇺🇸Hackensack, New Jersey, United States
Cornell University, Joan & Sanford I. Weill Medical College
🇺🇸New York, New York, United States
Steven and Alexandra Cohen Children's Medical Center of New York
🇺🇸New York, New York, United States
Mount Sinai Medical Center
🇺🇸One Gustave L Levy Place, BOX 1410, New York, New York, United States
Case Western Reserve University
🇺🇸Cleveland, Ohio, United States
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