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The Role of Brain-Bone Marrow-Gut Interaction Following Major Trauma
Traumatic injury followed by critical illness provokes pathophysiologic changes in the bone marrow and the gut that contribute to persistent anemia and changes…
A Phase II Study of Reduced Intensity Conditioning in Pediatric Patients and Young Adults ≤55 Years of Age With Non-Malignant Disorders Undergoing Umbilical Cord Blood, Bone Marrow, or Peripheral Blood Stem Cell Transplantation
The objective of this study is to evaluate the efficacy of using a reduced-intensity condition (RIC) regimen with umbilical cord blood transplant (UCBT), doubl…
An Adaptive Design Basket Trial of Hematopoietic Stem Cell BCL11A Enhancer Gene Editing for Severe β-Hemoglobinopathies
A promising approach for the treatment of genetic diseases is called gene therapy. Gene therapy is a relatively new field of medicine that uses genetic materia…
Diamond Blackfan Anemia Registry (DBAR)
The purpose of this study is to maintain a comprehensive registry of patients with the rare inherited bone marrow failure syndrome Diamond Blackfan anemia (DBA…
A Phase 3, Randomized, Open-Label, Multicenter Study to Evaluate the Safety, Efficacy and Pharmacokinetics of Ferumoxytol for the Treatment of Iron Deficiency Anemia (IDA) in Pediatric Subjects
This is a Phase 3, randomized, open-label, multicenter, study in male and female pediatric subjects (2 years to \<18 years of age) with IDA, or felt by their c…
Prospective Study of the Clinical, Genomic, Pharmacological, Laboratory, and Dietary Determinates of Pyrimidine and Purine Metabolism Disorders
Background: Pyrimidine and purine metabolism disorders (DPPMs) affect how the body metabolizes chemicals called pyrimidines and purines. DPPMs can cause dysfu…
A Study of T-Cell Replete, HLA-Mismatched Haploidentical Bone Marrow Transplantation With Post-Transplant Cyclophosphamide for Patients With Severe Aplastic Anemia Lacking HLA-Matched Related Donor
Severe aplastic anemia is a rare and serious form of bone marrow failure related to an immune-mediated mechanism that results in severe pancytopenia and high r…
Effectiveness of Small Phlebotomy Tubes in Reducing Blood Transfusions in Adult Medical Intensive Care Unit and Intermediate Care Unit Patients With Anemia: A Randomized Controlled Trial
The purpose of this study is to evaluate the effectiveness of small phlebotomy tubes to reduce RBC transfusions in medical intensive care unit (ICU) and Interm…
Intravenous Ferric Derisomaltose for Moderate to Severe Anemia Due to Uterine Bleeding in the Emergency Department: a Randomized Trial
The primary aim of this randomized trial is to assess the efficacy of IV Ferric Derisomaltose vs Oral Iron in the management of women with severe Iron Deficien…
Dissecting the Role of Acetaldehyde in Oral Carcinogenesis
This is a minimal risk intervention study where healthy volunteers and individuals with Fanconi anemia will consume a single dose of alcohol and provide primar…
Role of Single-dose Intravenous Iron Therapy for the Treatment of Anemia in the Setting of Orthopaedic Trauma: a Pilot Study
Acute blood loss in orthopaedic trauma and operative fracture care contributes substantially to perioperative anemia, which places participants at increased ri…