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Conditions: Primary Immunodeficiency, Common Variable Hypogammaglobulinemia, X-linked Hypogammaglobulinemia, Hypogammaglobulinemia, Immunodeficiency With Hyper-IgM, Wiskott-Aldrich Syndrome
Interventions: Gammaplex (Intravenous immunoglobulin); Biological
Lead sponsor: Bio Products Laboratory; Other
Eligibility: 3 Years and older
Enrollment: 50 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2006 – 2007
U.S. locations: 7
States / cities: North Palm Beach, Florida • Atlanta, Georgia • Chicago, Illinois + 3 more

Conditions: Sickle Cell, Sickle Cell Disease, Sickle Cell Disease Without Crisis, Mediterranean Diet
Interventions: Mediterranean Diet, Usual Diet; Other
Lead sponsor: University of Illinois at Chicago; Other
Eligibility: 18 Years and older
Enrollment: 37 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2025 – 2026
U.S. locations: 1
States / cities: Chicago, Illinois

Conditions: Hemophilia A With Inhibitor, Hemophilia A
Interventions: Wilate; Drug
Lead sponsor: University of California, Davis; Other
Eligibility: Male only
Enrollment: 1 participant
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2017 – 2019
U.S. locations: 3
States / cities: Sacramento, California • San Diego, California • New Orleans, Louisiana

Conditions: Granuloma, Granulomatous Disease, Chronic, Leukocyte Disease, Genetic Disease, X-Linked, Genetic Disease, Inborn
Interventions: Not listed
Lead sponsor: National Institute of Allergy and Infectious Diseases (NIAID); NIH
Eligibility: 18 Years to 70 Years
Enrollment: 850 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: Started 1994
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Graft Versus Host Disease, Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm, Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Diseases
Interventions: graft versus host disease prophylaxis/therapy, allogeneic bone marrow transplantation, in vitro-treated bone marrow transplantation; Biological · Procedure
Lead sponsor: Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins; Other
Eligibility: Up to 65 Years
Enrollment: 72 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2002 – 2007
U.S. locations: 1
States / cities: Baltimore, Maryland

Conditions: Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities, Adult Acute Myeloid Leukemia With Del(5q), Adult Acute Myeloid Leukemia With Inv(16)(p13;q22), Adult Acute Myeloid Leukemia With t(15;17)(q22;q12), Adult Acute Myeloid Leukemia With t(16;16)(p13;q22), Adult Acute Myeloid Leukemia With t(8;21)(q22;q22), Adult Acute Promyelocytic Leukemia (M3), Recurrent Adult Acute Myeloid Leukemia
Interventions: romidepsin; Drug
Lead sponsor: National Cancer Institute (NCI); NIH
Eligibility: 18 Years and older
Enrollment: 47 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: Started 2003
U.S. locations: 2
States / cities: Chicago, Illinois • Nashville, Tennessee

Conditions: Hemophilia
Interventions: Fitusiran; Drug
Lead sponsor: Genzyme, a Sanofi Company; Industry
Eligibility: 12 Years and older · Male only
Enrollment: 281 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2019 – 2026
U.S. locations: 10
States / cities: Phoenix, Arizona • Los Angeles, California • Orange, California + 7 more

Conditions: Anemia, Sickle Cell
Interventions: Dexamethasone, Placebo; Drug
Lead sponsor: Children's Hospital Medical Center, Cincinnati; Other
Eligibility: 5 Years and older
Enrollment: 12 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2006 – 2008
U.S. locations: 6
States / cities: Sacramento, California • Louisville, Kentucky • Boston, Massachusetts + 3 more

Conditions: Transfusion Dependent Beta Thalassemia, Sickle Cell Disease
Interventions: Not listed
Lead sponsor: Georgetown University; Other
Eligibility: 18 Years and older
Enrollment: 100 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2024 – 2026
U.S. locations: 1
States / cities: Washington D.C., District of Columbia

Conditions: Hemophilia, Hemophilia A
Interventions: Not listed
Lead sponsor: Boston VA Research Institute, Inc.; Other
Eligibility: Male only
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2016 – 2021
U.S. locations: 1
States / cities: Boston, Massachusetts

Conditions: Hemophilia A
Interventions: BAY94-9027 + Recombinant Factor VIII (Kogenate FS, BAY14-2222)); Biological
Lead sponsor: Bayer; Industry
Eligibility: 18 Years to 65 Years · Male only
Enrollment: 14 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2010 – 2011
U.S. locations: 4
States / cities: Davis, California • Boston, Massachusetts • Minneapolis, Minnesota + 1 more

Conditions: Factor 10 Deficiency
Interventions: Coagadex; Drug
Lead sponsor: Bio Products Laboratory; Other
Eligibility: 12 Years and older
Enrollment: 3 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2018 – 2021
U.S. locations: 3
States / cities: Gainesville, Florida • New Orleans, Louisiana • Kalamazoo, Michigan

Conditions: Leukemia, Lymphoma, Lymphoproliferative Disorder, Multiple Myeloma and Plasma Cell Neoplasm, Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Neoplasms
Interventions: anti-thymocyte globulin, donor lymphocytes, filgrastim, therapeutic allogeneic lymphocytes, busulfan, fludarabine phosphate, methotrexate, mycophenolate mofetil, tacrolimus, reduced-intensity transplant conditioning procedure, allogeneic hematopoietic stem cell transplantation, peripheral blood stem cell transplantation; Biological · Drug · Other + 1 more
Lead sponsor: Alliance for Clinical Trials in Oncology; Other
Eligibility: Up to 69 Years
Enrollment: 6 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2010 – 2013
U.S. locations: 10
States / cities: Lewes, Delaware • Newark, Delaware • Orlando, Florida + 7 more

Conditions: Bone Marrow Failure Syndrome, Congenital Amegakaryocytic Thrombocytopenia, Diamond-Blackfan Anemia, Hereditary Sideroblastic Anemia, Paroxysmal Nocturnal Hemoglobinuria, Shwachman-Diamond Syndrome, Hematologic Neoplasm With Germline GATA2 Mutation, Hematologic Neoplasm With Germline SAMD9 Mutation, Hematologic Neoplasm With Germline SAMD9L Mutation
Interventions: Treosulfan, Fludarabine Phosphate, Tacrolimus, Methotrexate, Lapine T-Lymphocyte Immune Globulin, Peripheral Blood Stem Cell Transplantation, Allogeneic Bone Marrow Transplantation, Quality-of-Life Assessment, Echocardiography, Multigated Acquisition Scan, Bone Marrow Biopsy, Bone Marrow Aspiration, Biospecimen Collection, X-Ray Imaging, Computed Tomography; Drug · Biological · Procedure + 1 more
Lead sponsor: Fred Hutchinson Cancer Center; Other
Eligibility: 1 Year to 49 Years
Enrollment: 40 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2022 – 2026
U.S. locations: 24
States / cities: Los Angeles, California • San Diego, California • San Francisco, California + 20 more

Conditions: Sickle Cell Disease
Interventions: Demographic Questionnaire, Medical Adherence Measure Questionnaire, Acceptability Questionnaire; Behavioral
Lead sponsor: St. Jude Children's Research Hospital; Other
Eligibility: Up to 17 Years
Enrollment: 36 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2014 – 2015
U.S. locations: 1
States / cities: Memphis, Tennessee

Conditions: Pain, Sickle Cell Disease
Interventions: Morphine, Nubain; Drug
Lead sponsor: Children's Healthcare of Atlanta; Other
Eligibility: 6 Years to 19 Years
Enrollment: 40 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2010 – 2016
U.S. locations: 1
States / cities: Atlanta, Georgia

Conditions: Haemophilia A, Haemophilia B
Interventions: No treatment given; Other
Lead sponsor: Novo Nordisk A/S; Industry
Eligibility: 1 Year to 21 Years · Male only
Enrollment: 563 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2018 – 2019
U.S. locations: 22
States / cities: Los Angeles, California • Orange, California • San Diego, California + 19 more

Conditions: Hurler Syndrome, Fanconi Anemia, Glanzmann Thrombasthenia, Wiskott-Aldrich Syndrome, Chronic Granulomatous Disease, Severe Congenital Neutropenia, Leukocyte Adhesion Deficiency, Shwachman-Diamond Syndrome, Diamond-Blackfan Anemia, Dyskeratosis-congenita, Chediak-Higashi Syndrome, Severe Aplastic Anemia, Thalassemia Major, Hemophagocytic Lymphohistiocytosis, Sickle Cell Disease
Interventions: Abatacept; Drug
Lead sponsor: Emory University; Other
Eligibility: Up to 21 Years
Enrollment: 10 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2014 – 2019
U.S. locations: 1
States / cities: Atlanta, Georgia

Conditions: Severe Hemophilia A
Interventions: rFVIIIFc; Biological
Lead sponsor: Bioverativ Therapeutics Inc.; Industry
Eligibility: 12 Years and older · Male only
Enrollment: 24 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2015 – 2017
U.S. locations: 8
States / cities: Los Angeles, California • Chicago, Illinois • Indianapolis, Indiana + 5 more

Conditions: Priapism, Anemia, Sickle Cell
Interventions: Not listed
Lead sponsor: University of Texas Southwestern Medical Center; Other
Eligibility: 5 Years and older · Male only
Enrollment: 1,464 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2005 – 2008
U.S. locations: 27
States / cities: Mobile, Alabama • Davis, California • Oakland, California + 16 more

Conditions: Hemoglobinopathies, Sickle Cell Disease, Thalassemia
Interventions: Busulfex, Fludarabine, Alemtuzumab, Stem Cell Transfusion; Drug · Procedure
Lead sponsor: Dana-Farber Cancer Institute; Other
Eligibility: 18 Years and older
Enrollment: 2 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2004 – 2009
U.S. locations: 6
States / cities: Atlanta, Georgia • Shreveport, Louisiana • Boston, Massachusetts + 1 more

Conditions: To Assess the Safety and Tolerabiltiy of an Amino Acid Composition in Subjects With Sickle Cell Disease, Anemia, Sickle Cell, Sickle Cell Anemia, Sickle Cell Disorders, Sickle Cell Disease
Interventions: AXA4010; Dietary Supplement
Lead sponsor: Axcella Health, Inc; Industry
Eligibility: 12 Years and older
Enrollment: 9 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2019 – 2021
U.S. locations: 4
States / cities: Hollywood, Florida • Miami, Florida • Atlanta, Georgia + 1 more

Conditions: Thrombocytopenia, Neonatal Alloimmune
Interventions: Nipocalimab, Intravenous immunoglobulins (IVIG), Prednisone; Drug
Lead sponsor: Janssen Research & Development, LLC; Industry
Eligibility: 18 Years to 45 Years · Female only
Enrollment: 50 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2025 – 2029
U.S. locations: 13
States / cities: Birmingham, Alabama • Sacramento, California • Aurora, Colorado + 10 more

Conditions: Sickle Cell Disease
Interventions: acupuncture; Other
Lead sponsor: Deepika Darbari; Other
Eligibility: 8 Years to 22 Years
Enrollment: 29 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2018 – 2022
U.S. locations: 1
States / cities: Washington D.C., District of Columbia

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Efficacy, Safety and Pharmacokinetics of Gammaplex in Primary Immunodeficiency Diseases.

Sickle Cell, Pain and Mediterranean Diet

INdividualized ITI Based on Fviii(ATE) Protection by VWF

Recruitment and Apheresis Collection of Peripheral Blood Hematopoietic Stem Cells, Mononuclear Cells and Granulocytes

Laboratory-Treated Donor Bone Marrow in Treating Patients Who Are Undergoing a Donor Bone Marrow Transplant for Hematologic Cancer

Romidepsin in Treating Patients With Relapsed or Refractory Acute Myeloid Leukemia

Long-term Safety and Efficacy Study of Fitusiran in Patients With Hemophilia A or B, With or Without Inhibitory Antibodies to Factor VIII or IX

Dexamethasone to Treat Acute Chest Syndrome in People With Sickle Cell Disease

Decision-Making and Quality of Life Surrounding Hematologic Disease and Gene Therapy

Improved Factor VIII Inhibitor Evaluation

Trial to Evaluate the Pharmacokinetics and Safety Profile of BAY94-9027 Following Single and Multiple Dose Administration

Registry Study of COAGADEX® Patients With Moderate or Severe Hereditary Factor X Deficiency Undergoing Major Surgery

Donor Stem Cell Transplant in Treating Patients With Relapsed Hematologic Malignancies or Secondary Myelodysplasia Previously Treated With High-Dose Chemotherapy and Autologous Stem Cell Transplant

Treosulfan-Based Conditioning Regimen Before a Blood or Bone Marrow Transplant for the Treatment of Bone Marrow Failure Diseases (BMT CTN 1904)

Medication Adherence in Youth With Sickle Cell Disease (SCD)

Choosing Opioid Management for Pain and Analyzing Acute Chest Syndrome (ACS) Rates Equally

A Study of the Impact of Hemophilia and Its Treatment on Brain Development, Thinking and Behaviour in Children With Hemophilia

Bone Marrow Transplant With Abatacept for Non-Malignant Diseases

Pharmacokinetics and Safety of rFVIIIFc Manufactured at 15,000 L (15K) Scale

Priapism in Boys and Men With Sickle Cell Disease - Demographics, Characteristics and Prevalence

Allogeneic Stem Cell Transplantation Following Chemotherapy in Patients With Hemoglobinopathies

To Assess Safety, Tolerability and Physiological Effects on Structure and Function of AXA4010 in Subjects With Sickle Cell Disease

A Study of Nipocalimab or Intravenous Immunoglobulin (IVIG) in Pregnancies At Risk of Fetal and Neonatal Alloimmune Thrombocytopenia (FNAIT)

Acupuncture for Pain in Sickle Cell Disease