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Conditions: Bone Marrow Failure Syndrome, Severe Aplastic Anemia, Severe Congenital Neutropenia, Amegakaryocytic Thrombocytopenia, Diamond-Blackfan Anemia, Schwachman Diamond Syndrome, Primary Immunodeficiency Syndromes, Acquired Immunodeficiency Syndromes, Histiocytic Syndrome, Familial Hemophagocytic Lymphocytosis, Lymphohistiocytosis, Macrophage Activation Syndrome, Langerhans Cell Histiocytosis (LCH), Hemoglobinopathies, Sickle Cell Disease, Sickle Cell-beta-thalassemia
Interventions: CD34 Stem Cell Selection Therapy; Biological
Lead sponsor: Diane George; Other
Eligibility: Up to 40 Years
Enrollment: 37 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2013 – 2030
U.S. locations: 1
States / cities: New York, New York

Conditions: Diamond Blackfan Anemia, Pure Red Cell Aplasia
Interventions: Trifluoperazine; Drug
Lead sponsor: Adrianna Vlachos, MD; Other
Eligibility: 18 Years to 65 Years
Enrollment: 2 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2019 – 2021
U.S. locations: 2
States / cities: Boston, Massachusetts • Manhasset, New York

Conditions: Myeloid Malignancy, Bone Marrow Failure Syndrome, Transfusion-dependent Red Blood Cell (RBC) Defect, Congenital Immunodeficiency, Metabolic Disease, Severe Immune Dysregulation
Interventions: Alemtuzumab, Busulfan, Fludarabine, Clofarabine; Drug
Lead sponsor: University of California, San Francisco; Other
Eligibility: 3 Months to 30 Years
Enrollment: 16 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2012 – 2019
U.S. locations: 1
States / cities: San Francisco, California

Conditions: Severe Aplastic Anemia, Acquired Amegakaryocytic Thrombocytopenia, Acquired Pure Red Cell Aplasia, Paroxysmal Nocturnal Hemoglobinuria
Interventions: Rituximab, Rabbit ATG, Cyclophosphamide, Fludarabine, Total Body Irradiation, Cell Infusion, Post-Transplant G-CSF, Tacrolimus, Mycophenolate Mofetil; Drug · Radiation · Biological
Lead sponsor: Masonic Cancer Center, University of Minnesota; Other
Eligibility: 0 Years to 75 Years
Enrollment: 60 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2024 – 2036
U.S. locations: 1
States / cities: Minneapolis, Minnesota

Conditions: Primary Myelofibrosis, Anemia
Interventions: Luspatercept; Drug
Lead sponsor: Celgene; Industry
Eligibility: 18 Years and older
Enrollment: 95 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2017 – 2022
U.S. locations: 20
States / cities: Phoenix, Arizona • Stanford, California • Jacksonville, Florida + 7 more

Conditions: Accelerated Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive, Acute Leukemia in Remission, Acute Lymphoblastic Leukemia, Acute Myeloid Leukemia, Acute Myeloid Leukemia With FLT3/ITD Mutation, Acute Myeloid Leukemia With Gene Mutations, Aplastic Anemia, B-Cell Non-Hodgkin Lymphoma, CD40 Ligand Deficiency, Chronic Granulomatous Disease, Chronic Leukemia in Remission, Chronic Lymphocytic Leukemia, Chronic Myelogenous Leukemia, BCR-ABL1 Positive, Chronic Myelomonocytic Leukemia, Chronic Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive, Congenital Amegakaryocytic Thrombocytopenia, Congenital Neutropenia, Congenital Pure Red Cell Aplasia, Glanzmann Thrombasthenia, Immunodeficiency Syndrome, Myelodysplastic Syndrome, Myelofibrosis, Myeloproliferative Neoplasm, Paroxysmal Nocturnal Hemoglobinuria, Plasma Cell Myeloma, Polycythemia Vera, Recurrent Non-Hodgkin Lymphoma, Refractory Non-Hodgkin Lymphoma, Secondary Acute Myeloid Leukemia, Secondary Myelodysplastic Syndrome, Severe Aplastic Anemia, Shwachman-Diamond Syndrome, Sickle Cell Disease, T-Cell Non-Hodgkin Lymphoma, Thalassemia, Waldenstrom Macroglobulinemia, Wiskott-Aldrich Syndrome
Interventions: Cyclophosphamide, Fludarabine Phosphate, Laboratory Biomarker Analysis, Peripheral Blood Stem Cell Transplantation, Total-Body Irradiation; Drug · Other · Procedure + 1 more
Lead sponsor: Roswell Park Cancer Institute; Other
Eligibility: 1 Year to 75 Years
Enrollment: 31 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2017 – 2023
U.S. locations: 1
States / cities: Buffalo, New York

Conditions: Hemoglobinopathies, Hemolysis, Iron Deficiency and Overload, Anemias
Interventions: Not listed
Lead sponsor: National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); NIH
Eligibility: 1 Year and older
Enrollment: 334 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2005 – 2017
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Pyruvate Kinase Deficiency
Interventions: Not listed
Lead sponsor: Agios Pharmaceuticals, Inc.; Industry
Eligibility: Not listed
Enrollment: 500 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2018 – 2027
U.S. locations: 15
States / cities: Phoenix, Arizona • Little Rock, Arkansas • Orange, California + 10 more

Conditions: Sickle Cell Disease, Hemolytic Anemia
Interventions: Clinical Evaluations, Laboratory Studies; Procedure · Other
Lead sponsor: St. Jude Children's Research Hospital; Other
Eligibility: 5 Years and older
Enrollment: 390 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2009 – 2016
U.S. locations: 1
States / cities: Memphis, Tennessee

Conditions: Sickle Cell Disease
Interventions: Crizanlizumab, Placebo; Biological · Drug
Lead sponsor: Novartis Pharmaceuticals; Industry
Eligibility: 12 Years to 100 Years
Enrollment: 354 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2024 – 2030
U.S. locations: 16
States / cities: Birmingham, Alabama • Orange, California • Washington D.C., District of Columbia + 13 more

Conditions: Sickle Cell Disease, Thalassemia, Severe Congenital Neutropenia, Diamond-Blackfan Anemia, Shwachman-Diamond Syndrome
Interventions: Busulfan, Fludarabine, ATG, TLI, Busulfan, Cyclophosphamide, ATG, GCSF, Campath, Fludarabine, Cyclophosphamide, Total Body Irradiation, Stem cell infusion; Drug · Radiation · Procedure
Lead sponsor: Masonic Cancer Center, University of Minnesota; Other
Eligibility: Up to 50 Years
Enrollment: 22 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2002 – 2020
U.S. locations: 1
States / cities: Minneapolis, Minnesota

Conditions: Prematurity
Interventions: Umbilical Cord Milking; Procedure
Lead sponsor: Eastern Virginia Medical School; Other
Eligibility: 18 Years and older · Female only
Enrollment: 113 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2009 – 2011
U.S. locations: 1
States / cities: Norfolk, Virginia

Conditions: Sickle Cell Disease
Interventions: AG-348; Drug
Lead sponsor: National Institutes of Health Clinical Center (CC); NIH
Eligibility: 18 Years to 70 Years
Enrollment: 17 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2019 – 2021
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Transfusions
Interventions: NPO, Control group: continue feedings; Other
Lead sponsor: Christiana Care Health Services; Other
Eligibility: 3 Days to 6 Months
Enrollment: 12 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2013 – 2016
U.S. locations: 1
States / cities: Newark, Delaware

Conditions: Sickle Cell Disease, Vaso-occlusive Pain Episode in Sickle Cell Disease, Vaso-occlusive Crisis
Interventions: Inclacumab, Placebo; Drug
Lead sponsor: Pfizer; Industry
Eligibility: 12 Years and older
Enrollment: 241 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2021 – 2024
U.S. locations: 22
States / cities: Mobile, Alabama • Little Rock, Arkansas • Orange, California + 7 more

Conditions: Functional Iron Deficiency, Trauma, Anemia
Interventions: Iron sucrose, Oxandrolone, IV iron placebo, Oxandrolone placebo; Drug
Lead sponsor: Denver Health and Hospital Authority; Other
Eligibility: 18 Years and older
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2015 – 2017
U.S. locations: 1
States / cities: Denver, Colorado

Conditions: Glaucoma
Interventions: Indocyanine Green Angiography, Isocapnic Oxygen, Ocular Imaging with Optical Coherence Tomography (OCT) and Adaptive Optics (AO); Diagnostic Test · Biological
Lead sponsor: University of Maryland, Baltimore; Other
Eligibility: 18 Years to 88 Years
Enrollment: 90 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2022 – 2027
U.S. locations: 4
States / cities: Baltimore, Maryland • Silver Spring, Maryland

Conditions: Pediatric Pyruvate Kinase Deficiency, Pediatric Hemolytic Anemia
Interventions: Mitapivat, Mitapivat-matching placebo; Drug
Lead sponsor: Agios Pharmaceuticals, Inc.; Industry
Eligibility: 1 Year to 17 Years
Enrollment: 49 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2022 – 2029
U.S. locations: 9
States / cities: Phoenix, Arizona • Palo Alto, California • Atlanta, Georgia + 6 more

Conditions: Malaria
Interventions: Chloroquine Phosphate, Pyrimethamine, Sanaria PfSPZ Challenge; Drug · Biological
Lead sponsor: National Institute of Allergy and Infectious Diseases (NIAID); NIH
Eligibility: 18 Years to 50 Years
Enrollment: 55 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2017 – 2019
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Diamond Blackfan Anemia, Blackfan Diamond Syndrome, DBA, Congenital Hypoplastic Anemia, Pure Red Cell Aplasia
Interventions: leucine; Drug
Lead sponsor: Northwell Health; Other
Eligibility: 2 Years and older
Enrollment: 55 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2013 – 2020
U.S. locations: 11
States / cities: Phoenix, Arizona • Palo Alto, California • Indianapolis, Indiana + 8 more

Conditions: Anemia, Chronic Kidney Disease, Chronic Renal Failure
Interventions: peginesatide; Drug
Lead sponsor: Affymax; Industry
Eligibility: 18 Years and older
Enrollment: 165 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2005 – 2007
U.S. locations: 14
States / cities: Birmingham, Alabama • Pine Bluff, Arkansas • Los Angeles, California + 11 more

Conditions: Heart Defects, Congenital
Interventions: Rejuvesol, Cardiac surgery; Drug · Procedure
Lead sponsor: Duke University; Other
Eligibility: Up to 18 Years
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2020 – 2021
U.S. locations: 1
States / cities: Durham, North Carolina

Conditions: Sickle Cell, PKLR Variants, Adenosine Triphosphate Activities
Interventions: Not listed
Lead sponsor: National Heart, Lung, and Blood Institute (NHLBI); NIH
Eligibility: 18 Years to 80 Years
Enrollment: 800 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2018 – 2027
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Primary Myelofibrosis, MPN-associated Myelofibrosis
Interventions: Pomalidomide 0.5 mg, Placebo, Pomalidomide; Drug
Lead sponsor: Celgene; Industry
Eligibility: 18 Years and older
Enrollment: 267 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2010 – 2018
U.S. locations: 17
States / cities: Scottsdale, Arizona • Los Angeles, California • Gainesville, Florida + 12 more

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CD34+ (Non-Malignant) Stem Cell Selection for Patients Receiving Allogeneic Stem Cell Transplantation

The Use of Trifluoperazine in Transfusion Dependent DBA

Pilot Trial of Clofarabine Added to Standard Busulfan and Fludarabine for Conditioning Prior to Allogeneic Hematopoietic Cell Transplantation

MT2023-20: Hematopoietic Cell Transplant With Reduced Intensity Conditioning and Post-transplant Cyclophosphamide for Severe Aplastic Anemia and Other Forms of Acquired Bone Marrow Failure.

A Safety and Efficacy Study to Evaluate Luspatercept in Subjects With Myeloproliferative Neoplasm-associated Myelofibrosis Who Have Anemia With and Without Red Blood Cell-transfusion Dependence

Fludarabine Phosphate, Cyclophosphamide, Total Body Irradiation, and Donor Stem Cell Transplant in Treating Patients With Blood Cancer

Clinical and Laboratory Investigation of Humans With Informative Iron or Erythroid Phenotypes

Pyruvate Kinase Deficiency Global Longitudinal Registry

Long Term Effects of Erythrocyte Lysis

A Study to Investigate the Efficacy and Safety of Crizanlizumab (5 mg/kg) Compared With Placebo in Adolescent and Adult Sickle Cell Disease Patients Who Experience Frequent Vaso-Occlusive Crises (SPARKLE)

Stem Cell Transplant for Hemoglobinopathy

Umbilical Cord Milking on the Reduction of Red Blood Cell Transfusion Rates in Infants

A Dose-Finding Study of AG-348 in Sickle Cell Disease

Impact of Feeding on Pro-Inflammatory Cytokine Response in Neonates Receiving a RBC Transfusion

A Study to Assess the Safety and Efficacy of Inclacumab in Participants With Sickle Cell Disease Experiencing Vaso-occlusive Crises

RCT of Goal-directed Iron Supplementation of Anemic, Critically Ill Trauma Patients, With and Without Oxandrolone

Retinal Blood Flow and Autoregulation

A Study to Evaluate the Efficacy and Safety of Mitapivat in Pediatric Participants With Pyruvate Kinase Deficiency (PKD) Who Are Regularly Transfused, Followed by a 5-Year Extension Period

Dose Escalation PfSPZ-CVac

Pilot Phase I/II Study of Amino Acid Leucine in Treatment of Patients With Transfusion-Dependent Diamond Blackfan Anemia

Peginesatide for Anemia in Chronic Hemodialysis Patients

Rejuvenated, Washed Packed Red Blood Cells in Pediatric Cardiac Surgery

Genotype -Phenotype Correlation of PKLR Variants With Pyruvate Kinase, 2,3-Diphosphglycerate and Adenosine Triphosphate Activities in Red Blood Cells of People With Sickle Cell Disease

Study of Pomalidomide in Persons With Myeloproliferative-Neoplasm-Associated Myelofibrosis and RBC-Transfusion-Dependence