Symptoms and General Pathology

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Chronic Graft Versus Host Disease

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Chronic Myeloid Leukemia

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Acute Graft Versus Host Disease

Chronic Myeloproliferative Disorders

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.

StemEx®

Ka Wah Chan, MD

Scott D Rowley, MD

Mary Territo, MD

Patrick Stiff, MD

Agha Mounzer, MD

Entezam Sahovic, MD

Celia Grosskreutz, MD

Roger Giller, MD

Steven Neudorf, MD

Ronit Yerushalmi, MD

Tsila Zuckerman, MD

Christelle Ferra, MD

Cristina Arbona, MD

Guillermo Sanz, MD

William Arcese, MD

Alberto Bosi, MD

Sonali Chaudhury, MD

Jorge Sierra, MD

Igor B. Resnick, MD, PhD

Prof. Franco Locatelli, MD

Dr. Mi Kwon, MD

Dr. Pere Barba, MD

Dr. Cristina Diaz de Heredia, MD

Prof. Mary J Laughlin, MD

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.

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)

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

Gamida Cell -Teva Joint Venture Ltd.

Behaviors and Mental Disorders

Quality of Life

Umbilical cord blood transplantation after reduced intensity conditioning

Cord blood transplantation

Bernard RIO, MD, PhD

Multicentric evaluation of the reduction of unrelated cord blood transplantation (UCBT) toxicity by using reduced intensity conditioning (RIC) in patients with acute myeloid leukaemia.UCBT related mortality and morbidity were limiting factors for the development of this procedure in adults. Non myeloablative conditioning regimen showed promising results and prospective evaluation has to be developed to confirm these retrospective data.

Individual meta-analysis is planned to compare geno-identical transplantation with myeloid-ablative or non myeloid-ablative conditioning with UCBT after RIC.

Reduction of Cord Blood Transplantation Toxicity in Patients With Acute Myeloid Leukemia

Assessment of Reduction of Cord Blood Transplantation Toxicity by Using Reduced Intensity Conditioning in Patients With Acute Myeloid Leukemia.

Assistance Publique - Hôpitaux de Paris

Anemia

Anemia, Aplastic

Primary Myelofibrosis

Leukemia, Lymphocytic, Chronic, B-Cell

Leukemia, B-Cell

Bone Marrow Failure Disorders

Pancytopenia

Chronic Lymphocytic Leukemia

All Drugs and Chemicals

Bone Density Conservation Agents

Hormones

Teriparatide

Parathyroid Hormone

Hormone Antagonists

Calcium

Calcium, Dietary

Parathyroid hormone after double umbilical cord blood transplant.

Day +1: PTH 40 mcg, Day +2: PTH 60 mcg, Day +3: PTH 80 mcg, Day +4 to Day +29 or until ANC\>2000/microL: PTH 100 mcg

Parathyroid Hormone (teriparatide)

Karen K. Ballen, MD

Joseph Antin, MD

David Avigan, MD

Elizabeth J Shpall, MD

Colleen Delaney, MD

Ram Kamble, MD

Katarzyna Jamieson, M.D.

Philip McCarthy, M.D.

Edward Ball, M.D.

Richard Maziarz, M.D.

The purpose of this study is to determine whether the addition of parathyroid hormone after a sequential cord blood transplant will improve engraftment, which is the ability of the transplanted stem cells to grow and to successfully begin producing new blood cells.

In this phase II, single stage study, participants will include 40 adults who are candidates for a hematopoietic stem cell transplant. All participants will undergo a sequential cord blood transplant using a well-known myeloablative regimen of fludarabine, cyclophosphamide, and total body irradiation, which is appropriate for those individuals who are likely to benefit from an ablative regimen. Tacrolimus will be combined with mycophenolate mofetil (MMF) for the graft-versus-host disease (GVHD) prophylaxis regimen. Parathyroid hormone (PTH) will be added to this regimen in an attempt to improve engraftment. PTH is an approved drug with minimal side effects in individuals with osteoporosis; the dose of PTH has been determined from a phase I study in individuals with hematologic cancer.

Study of Parathyroid Hormone Following Sequential Cord Blood Transplantation From an Unrelated Donor

A Phase II Study of Parathyroid Hormone Following Myeloablative Sequential Unrelated Cord Blood Transplantation

Median time to neutrophil engraftment (defined as an absolute neutrophil count \[ANC\] greater than 500)

Cumulative Incidence of Acute GVHD Grades II-IV at day 100

Platelet engraftment (greater than 20,000)

National Heart, Lung, and Blood Institute (NHLBI)

The Emmes Company, LLC

Parathyroid Hormone (Teriparatide)

Age, Categorical

Age Continuous

Sex: Female, Male

Race (NIH/OMB)

United States

Region of Enrollment

The study was closed prematurely due to early mortality and lack of efficacy.

Adam Mendizabal, Statistician

Median Time to Neutrophil Engraftment (Defined as an Absolute Neutrophil Count [ANC] Greater Than 500)

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization
Efficacy and Safety Study of StemEx®, to Treat Subjects With High Risk Hematologic Malignancies, Following Myeloablative Therapy	NCT00469729	Hematologic Mal... Acute Myeloid L... Lymphoid Leukem... Chronic Myeloid... Hodgkin's Disea... Non-Hodgkin's L... Myelodysplastic...	StemEx®	12 Years - 55 Years	Gamida Cell -Teva Joint Venture Ltd.
Reduction of Cord Blood Transplantation Toxicity in Patients With Acute Myeloid Leukemia	NCT00797758	Acute Myeloid L...	Cord blood tran...	4 Years - 65 Years	Assistance Publique - Hôpitaux de Paris
Reduction of Cord Blood Transplantation Toxicity in Patients With Acute Myeloid Leukemia	NCT00797758	Acute Myeloid L...	Cord blood tran...	4 Years - 65 Years	Assistance Publique - Hôpitaux de Paris
Efficacy and Safety Study of StemEx®, to Treat Subjects With High Risk Hematologic Malignancies, Following Myeloablative Therapy	NCT00469729	Hematologic Mal... Acute Myeloid L... Lymphoid Leukem... Chronic Myeloid... Hodgkin's Disea... Non-Hodgkin's L... Myelodysplastic...	StemEx®	12 Years - 55 Years	Gamida Cell -Teva Joint Venture Ltd.
Study of Parathyroid Hormone Following Sequential Cord Blood Transplantation From an Unrelated Donor	NCT00393380	Leukemia, Myelo... Anemia, Aplasti... Myelofibrosis Lymphoma Hodgkin Disease Leukemia, Lymph... Leukemia, Myelo... Leukemia, Lymph...	Parathyroid Hor...	18 Years - 45 Years	The Emmes Company, LLC

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