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Regenerative Medicine to Restore Hematopoiesis and Immune Function in Immunodeficiencies and Inherited Bone Marrow Failures
Phase II prospective trial to assess the rates of donor engraftment using reduced intensity conditioning (RIC) hematopoietic stem cell transplant (HSCT) and po…
Etiologic Investigation of Cancer Susceptibility in Inherited Bone Marrow Failure Syndromes: A Natural History Study
Background: A prospective cohort of Inherited Bone Marrow Failure Syndrome (IBMFS) will provide new information regarding cancer rates and types in these diso…
MT2017-17:T Cell Receptor Alpha/Beta T Cell Depleted Hematopoietic Cell Transplantation in Patients With Inherited Bone Marrow Failure (BMF) Disorders
This is a phase II trial of T cell receptor alpha/beta depletion (α/β TCD) peripheral blood stem cell (PBSC) transplantation in patients with inherited bone ma…
Targeting the Pathophysiology of Sickle Cell-Related Kidney Disease Using the SGLT2 Inhibitors, Empagliflozin
Sickle cell anemia (SCA) is an inherited red blood disorder. The kidneys are among the most commonly affected organ systems in SCA. The Food and Drug Administr…
Depth of Maximal Insertion During Retrograde Enteroscopy Using Colonoscopy With Through-the-scope Balloon Enteroscopy (NaviAid) Compared With Using the Standard Colonoscope Alone
Diagnostic modalities for the evaluation of small bowel pathology include video capsule endoscopy (VCE), antegrade and retrograde device-assisted enteroscopy, …
The Congenital Dyserythropoietic Anemia Registry (CDAR)
The investigators have created and maintain a comprehensive registry for patients with the diagnosis of Congenital Dyserythropoietic Anemia (CDA) in North Amer…
The Role of Metabolic and Hemodynamic Reserve in Age-Related Brain Vulnerability in Pediatric Sickle Cell Anemia
The purpose of this research study is to better understand how blood flow and metabolism change can influence brain development in the early decades of life. S…
Intensive Tailored Exercise Training With NAD+ Precursor Supplementation to Improve Muscle Mass and Fitness in Adolescent and Young Adult Hematopoietic Cell Transplant Survivors
This will be a randomized, placebo-controlled trial with a 2x2 factorial design testing the effects of an NAD+ precursor (NR) and exercise on skeletal muscle q…
Randomized Trial of Sucrosomial Iron Vs Intravenous Iron for Preoperative Anemia (RSIVI)
50 patients with anemia scheduled for heart surgery or procedures will be randomized to receive either oral sucrosomial iron or standard-of-care intravenous ir…
A New Combination of Evidence-Based Interventions to Improve Primary Care Diagnostic Safety and Efficiency: a Stepped Wedge Cluster Randomized Control Trial (RCT)
The purpose of this study is to measure the evidence-based intervention's (EBIs) impact on patient safety and efficiency, to assess the EBIs implementation by …
Early Antenatal Support for Iron Deficiency Anemia: A Randomized Controlled Trial of Early Initiation of Intravenous Versus Oral Iron Therapy for Treatment of Iron Deficiency Anemia in Pregnancy
This is a randomized, controlled multi-site trial of iron therapy in pregnancy. The purpose of this research is to see if second trimester initiation of intrav…
A 2 Step Approach to Haploidentical Transplant Using Radiation-Based Reduced Intensity Conditioning
This phase II clinical trial evaluates whether a modified modality of conditioning reduces treatment-related mortality (TRM) in patients who undergo a hematopo…