Acute Myeloid Leukemia, Chronic Myeloid Leukemia, Hematologic Malignancies, Hodgkin's Disease, Lymphoid Leukemia, Myelodysplastic Syndromes, Non-Hodgkin's Lymphoma
Conditions
Keywords
Tetraethylenepentamine, Umbilical Cord Blood Stem Cell Transplantation, Hematological Malignancies, Acute Lymphoid Leukemia, Subjects with high-risk hematologic malignancies who are candidates for allogeneic stem cell transplantation
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)
Interventions
DRUGStemEx®
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.
Sponsors
Gamida Cell -Teva Joint Venture Ltd.
Study design
Allocation
NON_RANDOMIZED
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
12 Years to 55 Years
Healthy volunteers
No
Inclusion criteria
1. 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. 2. 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. 3. Clinical diagnosis of HD: induction failure or relapse and sensitive to last chemotherapy course. 4. Clinical diagnosis of NHL induction failure or relapse and sensitive to last chemotherapy course. 5. Clinical diagnosis of MDS with intermediate 2- or high-risk IPSS score.
Exclusion criteria
1. Less than twenty-one days have elapsed since the subject's last radiation or chemotherapy prior to conditioning (except Hydroxyurea). 2. HIV positive. 3. Pregnancy or lactation. 4. Uncontrolled bacterial, fungal or viral infection. 5. Subjects with signs and symptoms of active central nervous system (CNS) disease. 6. Availability of appropriate related and willing stem cell donor, who is HLA-matched at 5 or 6/6 antigens. 7. Prior allogeneic cell transplant. 8. Allergy to bovine or to any product, which may interfere with the treatment. 9. Enrolled in another clinical trial or received an investigational treatment during the last 30 days, unless approved by Sponsor.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Overall 100-day mortality | 100 days |
Secondary
| Measure | Time frame |
|---|---|
| 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 |
Countries
Hungary, Israel, Italy, Spain, United States
Outcome results
None listed