Symptoms and General Pathology

All Conditions

Neoplasms

Blood and Lymph Conditions

Diseases and Abnormalities at or Before Birth

Immune System Diseases

Nutritional and Metabolic Diseases

Rare Diseases

Syndrome

Leukemia

Leukemia, Myeloid

Leukemia, Myeloid, Acute

Preleukemia

Myelodysplastic Syndromes

Anemia, Sickle Cell

Precursor Cell Lymphoblastic Leukemia-Lymphoma

Leukemia, Lymphoid

Thalassemia

beta-Thalassemia

Hematologic Neoplasms

Immunologic Deficiency Syndromes

Metabolism, Inborn Errors

Hematologic Diseases

Severe Combined Immunodeficiency

Myeloid Leukemia

Acute Myeloid Leukemia

Acute Non Lymphoblastic Leukemia

Sickle Cell Anemia

Acute Lymphoblastic Leukemia

Lymphoblastic Lymphoma

Beta-thalassemia

Acute Graft Versus Host Disease

all subjects will be treated with UCB and HPDSC

single dose of UCB followed by one unit of HPDSC

UCB and HPDSC

Lolie Yu, MD

To investigate the safety of partially matched related human placental-derived stem cells (HPDSC) administered in conjunction with umbilical cord blood (UCB) stem cells from the same donor in subjects with various malignant or nonmalignant disorders potentially curable with stem cell transplantation and to assess potential restoration of normal hematopoiesis and immune function in subjects with these disorders

A Single-arm Safety Study of Transplantation Using Umbilical Cord Blood and Human Placental-derived Stem Cells From Partially Matched Related Donors in Persons With Certain Malignant Blood Diseases and Non-malignant Disorders

A Single-Arm Study to Assess the Safety of Transplantation With Umbilical Cord Blood Augmented With Human Placental-Derived Stem Cells From Partially Matched Related Donors in Subjects With Certain Malignant Hematologic Diseases and Non-Malignant Disorders

Celgene Corporation

Bone Marrow Diseases

Precancerous Conditions

Anemia

Hemolysis

Anemia, Hemolytic

Anemia, Hemolytic, Congenital

Hemoglobinopathies

Genetic Diseases, Inborn

Hematinics

All Drugs and Chemicals

Luspatercept

BMS Study Connect Contact Center www.BMSStudyConnect.com

Ji-Hyun Kim

South Korea Generic Country, Site 0001

Bristol-Myers Squibb

The purpose of this observational study is to assess the real-world safety of luspatercept in Korean participants with myelodysplastic syndrome (MDS) or beta thalassemia. Investigators will enroll participants who will begin treatment with at least 1 dose of luspatercept.

A Post-Marketing Surveillance Study to Assess Safety of Luspatercept in Korean Patients With Myelodysplastic Syndrome or β-thalassemia

Reblozyl® (Luspatercept) Post-Marketing Surveillance in Korean Patients With Myelodysplastic Syndrome or Beta Thalassemia

Anti-Inflammatory Agents

Antineoplastic Agents

Antiemetics

Gastrointestinal Agents

Antirheumatic Agents

Anti-Infective Agents

Tacrolimus

Dexamethasone

Cyclophosphamide

Fludarabine

Fludarabine phosphate

Rituximab

Bortezomib

Mycophenolic Acid

Thymoglobulin

Dexamethasone acetate

Immunosuppressive Agents

Immunologic Factors

Antineoplastic Agents, Alkylating

Alkylating Agents

Antineoplastic Agents, Immunological

Glucocorticoids

Hormones

Hormone Antagonists

Antineoplastic Agents, Hormonal

Calcineurin Inhibitors

Enzyme Inhibitors

Anti-Bacterial Agents

Antibiotics, Antitubercular

Antitubercular Agents

Participants will have severe sickle cell disease or transfusion-dependent β-thalassemia.

PK-guided fludarabine dosing will be used for each of the 2 cycles, using the InsightRx DoseMeRx platform.

Cyclophosphamide will be administered Post-Transplant

Tacrolimus will be administered beginning on day +5

Mycophenolate mofetil (MMF) will be administered three times daily starting on day +5.

Mycophenolate Mofetil

The dose and schedule of ATG will be determined according to the nomogram in Appendix A

Rabbit ATG

Standard Regimen: Dexamethasone on days -68 to -64 and days -40 to -36.

Bortezomib on days -71, -68, -65, -61, -43, -40, -37, and -33

Rituximab on days -71, -58, -43, and -30.

Maria Cancio, MD

Jaap Jan Boelens, MD, PhD

Hematopoietic Cell Transplantation/HCT involves receiving healthy blood-forming cells (stem cells) from a donor to replace the diseased or damaged cells in participants' bone marrow. The researchers think giving participants treatment with fludarabine and dexamethasone, drugs that lower the activity of the body's immune system (immune suppression), before standard conditioning therapy and HCT may help prevent serious side effects, including graft failure and GvHD. In this study, depending on how participants' body responds to the fludarabine and dexamethasone, the study doctor may decide participants should receive another drug, called cyclophosphamide, instead of fludarabine. In addition, depending on the results of participants' routine blood tests, participants may receive the drugs bortezomib and rituximab, which also help with immune suppression.

A Study of Immune Suppression Treatment for People With Sickle Cell Disease or β-Thalassemia Who Are Going to Receive an Allogeneic Hematopoietic Cell Transplantation (HCT)

Pre-Transplant Immune Suppression With Hematopoietic Cell Transplantation From Haploidentical Donors for Adults and Children With Sickle Cell Disease or ß-Thalassemia (Haplo PTCy)

Memorial Sloan Kettering Cancer Center supports the international committee of medical journal editors (ICMJE) and the ethical obligation of responsible sharing of data from clinical trials. The protocol summary, a statistical summary, and informed consent form will be made available on clinicaltrials.gov when required as a condition of Federal awards, other agreements supporting the research and/or as otherwise required. Requests for deidentified individual participant data can be made beginning 12 months after publication and for up to 36 months post publication. Deidentified individual participant data reported in the manuscript will be shared under the terms of a Data Use Agreement and may only be used for approved proposals. Requests may be made to: crdatashare@mskcc.org.

The primary outcome is to estimate treatment-related mortality (TRM) or primary graft failure at 1 year post-HCT.

Memorial Sloan Kettering Cancer Center

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization
A Single-arm Safety Study of Transplantation Using Umbilical Cord Blood and Human Placental-derived Stem Cells From Partially Matched Related Donors in Persons With Certain Malignant Blood Diseases and Non-malignant Disorders	NCT00596999	Hematologic Mal...	UCB and HPDSC	- 55 Years	Celgene
A Single-arm Safety Study of Transplantation Using Umbilical Cord Blood and Human Placental-derived Stem Cells From Partially Matched Related Donors in Persons With Certain Malignant Blood Diseases and Non-malignant Disorders	NCT00596999	Hematologic Mal...	UCB and HPDSC	- 55 Years	Celgene
A Post-Marketing Surveillance Study to Assess Safety of Luspatercept in Korean Patients With Myelodysplastic Syndrome or β-thalassemia	NCT06073860	Myelodysplastic... Beta Thalassemi...	Luspatercept	19 Years -	Bristol-Myers Squibb
A Study of Immune Suppression Treatment for People With Sickle Cell Disease or β-Thalassemia Who Are Going to Receive an Allogeneic Hematopoietic Cell Transplantation (HCT)	NCT05736419	Sickle Cell Dis... Thalassemia, Be... Thalassemia	Fludarabine Cyclophosphamid... Tacrolimus Mycophenolate M... Rabbit ATG Dexamethasone Bortezomib Rituximab	2 Years - 50 Years	Memorial Sloan Kettering Cancer Center

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