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A Phase I/II Study of the JAK1/2 Inhibitor Ruxolitinib for Relapsed / Refractory Immune Bone Marrow Failure
Background: Immune bone marrow failure is a condition that occurs when a person s immune system attacks the cells of the bone marrow. This can lead to disease…
Clinical Genetics Branch (CGB) Eligibility Screening Survey
Background: Clinical Genetics Branch (CGB) researchers study individuals and populations at high genetic risk of cancer in order to improve our understanding …
Allogeneic Stem Cell Transplantation for Malignant and Non-malignant Hematologic Diseases Utilizing Alpha/Beta T Cell and CD19+ B Cell Depletion - NYMC 588
Children, adolescents, and young adults with malignant and non-malignant conditionsundergoing an allogeneic stem cell transplantation (AlloSCT) will have the s…
A Phase I/II, Non-Randomized Study of the Safety and Efficacy of In Utero Hematopoietic Stem Cell Transplantation for the Treatment of Fanconi Anemia in Affected Fetuses
The investigators aim to evaluate the safety and efficacy of in utero hematopoietic stem cell transplantation (IUHSCT) for the treatment of fetuses diagnosed w…
A Phase 1 Study to Evaluate the Safety and Tolerability of Escalating Doses of VAS-101 in Subjects With Stable Sickle Cell Disease
Background: Sickle cell disease (SCD) is an inherited blood disorder. The disease affects the ability of red blood cells to carry oxygen. Research has shown t…
Defining the Natural History of Squamous Cell Carcinoma in Fanconi Anemia
Background: Fanconi anemia (FA) is an inherited disorder. People with FA are more likely to get certain cancers, especially squamous cell carcinoma (SCC). The…
Efficacy of Luspatercept In Clonal Cytopenias of Uncertain Significance
The purpose of this clinical trial is to test how well the drug luspatercept works in improving low blood cell counts in people with clonal cytopenias of uncer…
Verifying Antibodies After Live Immunization Delivery (VALID): a Study of Measles Vaccine Immunogenicity in Children With Sickle Cell Disease
The goal of this study is to learn if infants with sickle cell disease (SCD) develop adequate protection after measles vaccines. (not looking at any prolonged …
Trial of Darbepoetin Plus Slow-release Intravenous Iron to Decrease Transfusions and Improve Iron Status and Neurodevelopment in Preterm Infants
In this phase II trial, the investigators overarching goal is to demonstrate the feasibility and potential benefit of darbepoetin (Darbe) plus slow-release int…
TCRαβ+ T-cell/CD19+ B-cell Depleted Hematopoietic Grafts and a Reduced Intensity Preparative Conditioning Regimen Containing JSP191 (Briquilimab) to Achieve Engraftment and Blood Reconstitution in Patients With Fanconi Anemia
The objective of this clinical trial is to develop a cell therapy for Fanconi Anemia which enables enhanced donor hematopoietic and immune reconstitution with …
Kids With Iron Deficiency and Scoliosis (KIDS) Study
This study is a randomized controlled trial of preoperative oral iron supplementation, to identify whether iron deficiency is a modifiable risk factor for adve…
Better Birth Outcomes and Experiences Through Technology, Education, and Reporting (BETTER)
This is a pragmatic randomized control trial to evaluate the BETTER intervention compared to standard obstetrical care (control) to determine whether it helps …