Efficacy and Safety Study of StemEx®, to Treat Subjects With High Risk Hematologic Malignancies, Following Myeloablative Therapy (ExCell)
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| ClinicalTrials.gov Identifier: NCT00469729 |
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Recruitment Status :
Completed
First Posted : May 4, 2007
Last Update Posted : July 10, 2015
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| Condition or disease | Intervention/treatment | Phase |
|---|---|---|
| Hematologic Malignancies Acute Myeloid Leukemia Lymphoid Leukemia Chronic Myeloid Leukemia Hodgkin's Disease Non-Hodgkin's Lymphoma Myelodysplastic Syndromes | Drug: StemEx® | Phase 2 Phase 3 |
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:
- Screening phase includes subjects' clinical assessment and screening tests
- Conditioning phase includes the myeloablative treatment prior transplantation procedure
- Transplantation and post-transplant follow-up phase to day 180
- Observational phase: survival status follow-up to day 730 (18 months)
| Study Type : | Interventional (Clinical Trial) |
| Actual Enrollment : | 101 participants |
| Allocation: | Non-Randomized |
| Intervention Model: | Single Group Assignment |
| Masking: | None (Open Label) |
| Primary Purpose: | Treatment |
| Official Title: | 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 |
| Study Start Date : | October 2007 |
| Actual Primary Completion Date : | February 2013 |
| Actual Study Completion Date : | June 2015 |
| Arm | Intervention/treatment |
|---|---|
| Experimental: StemEx |
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. |
- Overall 100-day mortality [ Time Frame: 100 days ]
- 180 day mortality, acute Graft versus Host Disease (GvHD) grades III-IV, engraftment failure [ Time Frame: 180 days ]
- Safety and tolerability measures: The incidence and frequency of adverse experiences, acute toxicity, laboratory data and vital signs follow-up. [ Time Frame: 180 days ]
- Proportion of overall mortality at 1 year [ Time Frame: One year post transplant ]
- Proportion of overall mortality at 2 years [ Time Frame: Two years post transplant ]
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, Learn About Clinical Studies.
| Ages Eligible for Study: | 12 Years to 55 Years (Child, Adult) |
| Sexes Eligible for Study: | All |
| Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- 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.
Exclusion Criteria:
- 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.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00469729
Show 30 study locations
| Principal Investigator: | Ka Wah Chan, MD | Texas Transplant Institute | |
| Principal Investigator: | Scott D Rowley, MD | The Cancer Center at Hackensack University Medical Center | |
| Principal Investigator: | Mary Territo, MD | UCLA Oncology Center | |
| Principal Investigator: | Patrick Stiff, MD | Loyola University Cardinal Bernardin Cancer Center | |
| Principal Investigator: | Agha Mounzer, MD | University of Pittsburgh Cancer Institute/UPMC Cancer Centers | |
| Principal Investigator: | Entezam Sahovic, MD | The Western Pennsylvania Hospital | |
| Principal Investigator: | Celia Grosskreutz, MD | Icahn School of Medicine at Mount Sinai | |
| Principal Investigator: | Roger Giller, MD | The Children's Hospital, B115, University of Colorado Health Sciences Center | |
| Principal Investigator: | Steven Neudorf, MD | Children's Hospital of Orange County | |
| Principal Investigator: | Ronit Yerushalmi, MD | Chaim Sheba Medical Center | |
| Principal Investigator: | Tsila Zuckerman, MD | Rambam Health Care Campus | |
| Principal Investigator: | Christelle Ferra, MD | Germans Trias i Pujol Hospital | |
| Principal Investigator: | Cristina Arbona, MD | Hospital Clínico Universitario de Valencia | |
| Principal Investigator: | Guillermo Sanz, MD | Hospital Universitario La Fe | |
| Principal Investigator: | William Arcese, MD | Universita di Roma Tor Vergata | |
| Principal Investigator: | Alberto Bosi, MD | Ospedale di Careggi BMT Unit Department of Haematology | |
| Principal Investigator: | Sonali Chaudhury, MD | Northwestern University School of Medicine, Stem Cell Transplant Program, Children's Memorial Hospital | |
| Principal Investigator: | Jorge Sierra, MD | Fundació Institut de Recerca de l'Hospital de la Santa Creu i Sant Pau | |
| Principal Investigator: | Igor B. Resnick, MD, PhD | Department of Bone Marrow Transplantation And Cancer Immunotherapy Hebrew University Hospital Ein-Karem, Jerusalem | |
| Principal Investigator: | Prof. Franco Locatelli, MD | Ospedale Pedriatrico Bambino Gesù | |
| Principal Investigator: | Dr. Mi Kwon, MD | Hospital General Universitario Gregorio Marañón | |
| Principal Investigator: | Dr. Pere Barba, MD | Hospital Universitario Vall d´Hebrón | |
| Principal Investigator: | Dr. Cristina Diaz de Heredia, MD | Hospital Universitario Vall d´Hebrón | |
| Principal Investigator: | Prof. Mary J Laughlin, MD | Hematopoietic Stem Cell Transplant Program, University of Virginia |
Publications:
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
| Responsible Party: | Gamida Cell -Teva Joint Venture Ltd. |
| ClinicalTrials.gov Identifier: | NCT00469729 |
| Obsolete Identifiers: | NCT00763334 |
| Other Study ID Numbers: |
GC P#02.01.001 |
| First Posted: | May 4, 2007 Key Record Dates |
| Last Update Posted: | July 10, 2015 |
| Last Verified: | March 2013 |
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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 |
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Leukemia Leukemia, Myeloid Neoplasms Hematologic Neoplasms Leukemia, Myelogenous, Chronic, BCR-ABL Positive Hodgkin Disease Leukemia, Lymphoid Myelodysplastic Syndromes Neoplasms by Histologic Type |
Bone Marrow Diseases Hematologic Diseases Lymphoma Lymphoproliferative Disorders Lymphatic Diseases Immunoproliferative Disorders Immune System Diseases Neoplasms by Site Myeloproliferative Disorders |