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Conditions: Sickle Cell Disease
Interventions: single infusion of autologous bone marrow derived CD34+ HSC cells transduced with the lentiviral vector containing a short-hairpin RNA targeting BCL11a; Biological
Lead sponsor: David Williams; Other
Eligibility: 3 Years to 40 Years
Enrollment: 10 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2018 – 2026
U.S. locations: 2
States / cities: Los Angeles, California • Boston, Massachusetts

Conditions: Sickle Cell Disease
Interventions: Dronabinol, Placebo; Drug
Lead sponsor: Icahn School of Medicine at Mount Sinai; Other
Eligibility: 18 Years and older
Enrollment: 60 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2025 – 2027
U.S. locations: 1
States / cities: New York, New York

Conditions: Congenital Amegakaryocytic Thrombocytopenia, Diamond-blackfan Anemia, Leukemia, Myelodysplastic Syndromes, Severe Congenital Neutropenia
Interventions: alemtuzumab, busulfan, cyclosporine, fludarabine phosphate, methotrexate, methylprednisolone, allogeneic bone marrow transplantation, allogeneic hematopoietic stem cell transplantation, peripheral blood stem cell transplantation, umbilical cord blood transplantation; Biological · Drug · Procedure
Lead sponsor: University of California, San Francisco; Other
Eligibility: Up to 21 Years
Enrollment: 35 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2005 – 2011
U.S. locations: 2
States / cities: San Francisco, California • Madison, Wisconsin

Conditions: Chronic Myeloproliferative Disorders, Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm, Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Neoplasms, Nonmalignant Neoplasm
Interventions: trivalent influenza vaccine, Immunoenzyme technique, Laboratory biomarker analysis; Biological · Other
Lead sponsor: City of Hope Medical Center; Other
Eligibility: 18 Years to 120 Years
Enrollment: 459 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2007 – 2013
U.S. locations: 2
States / cities: Duarte, California • Pasadena, California

Conditions: Sickle Cell Disease, Thalassemia
Interventions: Etavopivat tablets; Drug
Lead sponsor: Forma Therapeutics, Inc.; Industry
Eligibility: 12 Years to 65 Years
Enrollment: 53 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2022 – 2025
U.S. locations: 14
States / cities: Cerritos, California • Los Angeles, California • Oakland, California + 9 more

Conditions: Chronic Cutaneous Ulcer, Sickle Cell Disease
Interventions: Deferoxamine Product, Placebo; Drug · Other
Lead sponsor: TauTona Group; Industry
Eligibility: 18 Years and older
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2020 – 2021
U.S. locations: 4
States / cities: Birmingham, Alabama • Miami, Florida • Atlanta, Georgia + 1 more

Conditions: Anemia, Sickle Cell
Interventions: Monthly blood transfusion regimen, Usual care; Procedure · Behavioral
Lead sponsor: UCSF Benioff Children's Hospital Oakland; Other
Eligibility: 21 Years to 55 Years
Enrollment: 36 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2004 – 2010
U.S. locations: 14
States / cities: Oakland, California • Washington D.C., District of Columbia • Hollywood, Florida + 10 more

Conditions: Acute Myeloid Leukemia, Acute Lymphoblastic Leukemia, Myelodysplastic Syndromes, Non-hodgkin Lymphoma, Hodgkin Lymphoma, Acquired Aplastic Anemia, Sickle Cell Disease
Interventions: Donor Search Prognosis Score; Other
Lead sponsor: Center for International Blood and Marrow Transplant Research; Network
Eligibility: Not listed
Enrollment: 1,753 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2019 – 2025
U.S. locations: 52
States / cities: Duarte, California • La Jolla, California • Los Angeles, California + 40 more

Conditions: Sickle Cell Disease
Interventions: AB1; Drug
Lead sponsor: Nirmish Shah; Other
Eligibility: 18 Years to 45 Years
Enrollment: 16 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2022 – 2024
U.S. locations: 3
States / cities: Augusta, Georgia • Durham, North Carolina • Greenville, North Carolina

Conditions: Hemolytic Anemia
Interventions: Not listed
Lead sponsor: Children's Hospital Medical Center, Cincinnati; Other
Eligibility: Not listed
Enrollment: 400 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2011 – 2052
U.S. locations: 1
States / cities: Cincinnati, Ohio

Conditions: Sickle Cell Anemia
Interventions: Hydroxyurea, L-Arginine, Sildenafil; Drug
Lead sponsor: National Institutes of Health Clinical Center (CC); NIH
Eligibility: 16 Years and older
Enrollment: 39 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2003 – 2006
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Thalassemia
Interventions: Busulfan, Fludarabine, Campath 1H, Cyclophosphamide, MESNA; Drug
Lead sponsor: Baylor College of Medicine; Other
Eligibility: Up to 64 Years
Enrollment: 10 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2004 – 2016
U.S. locations: 1
States / cities: Houston, Texas

Conditions: Sickle Cell, Diabetes, Cancer, Cardiovascular Disease, Genetic Screening
Interventions: Not listed
Lead sponsor: National Human Genome Research Institute (NHGRI); NIH
Eligibility: 18 Years to 100 Years
Enrollment: 1,061 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: Started 2012
U.S. locations: 3
States / cities: Bethesda, Maryland • Cincinnati, Ohio • Lewisburg, Pennsylvania

Conditions: Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities, 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), Blastic Phase Chronic Myelogenous Leukemia, Chronic Myelomonocytic Leukemia, de Novo Myelodysplastic Syndromes, Previously Treated Myelodysplastic Syndromes, Recurrent Adult Acute Lymphoblastic Leukemia, Recurrent Adult Acute Myeloid Leukemia, Refractory Anemia With Excess Blasts, Refractory Anemia With Excess Blasts in Transformation, Relapsing Chronic Myelogenous Leukemia, Secondary Acute Myeloid Leukemia, Secondary Myelodysplastic Syndromes
Interventions: becatecarin, laboratory biomarker analysis; Drug · Other
Lead sponsor: National Cancer Institute (NCI); NIH
Eligibility: 18 Years and older
Enrollment: 40 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: Started 2004
U.S. locations: 1
States / cities: Houston, Texas

Conditions: Iron, Abnormal Blood Level, Other Abnormal Blood Chemistry
Interventions: Fresh transfusion, Old transfusion; Procedure
Lead sponsor: Columbia University; Other
Eligibility: 18 Years to 65 Years
Enrollment: 14 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2008 – 2010
U.S. locations: 1
States / cities: New York, New York

Conditions: Acute Leukemia, Adenomatous Polyposis, Adrenocortical Carcinoma, AML, BAP1 Tumor Predisposition Syndrome, Carney Complex, Choroid Plexus Carcinoma, Constitutional Mismatch Repair Deficiency Syndrome, Diamond-Blackfan Anemia, DICER1 Syndrome, Dyskeratosis Congenita, Emberger Syndrome, Familial Acute Myeloid Leukemia, Familial Adenomatous Polyposis, Fanconi Anemia, Familial Cancer, Familial Wilms Tumor, Familial Neuroblastoma, GIST, Hereditary Breast and Ovarian Cancer, Hereditary Paraganglioma-Pheochromocytoma Syndrome, Hodgkin Lymphoma, Juvenile Polyposis, Li-Fraumeni Syndrome, Lynch Syndrome, MDS, Melanoma Syndrome, Multiple Endocrine Neoplasia Type 1, Multiple Endocrine Neoplasia Type 2, Neuroblastoma, Neurofibromatosis Type 1, Neurofibromatosis Type II, Nevoid Basal Cell Carcinoma Syndrome, Non Hodgkin Lymphoma, Noonan Syndrome and Other Rasopathy, Overgrowth Syndromes, Pancreatic Cancer, Peutz-Jeghers Syndrome, Pheochromocytoma/Paraganglioma, PTEN Hamartoma Tumor Syndrome, Retinoblastoma, Rhabdoid Tumor Predisposition Syndrome, Rhabdomyosarcoma, Rothmund-Thomson Syndrome, Tuberous Sclerosis, Von Hippel-Lindau Disease
Interventions: Not listed
Lead sponsor: St. Jude Children's Research Hospital; Other
Eligibility: Not listed
Enrollment: 1,500 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2017 – 2037
U.S. locations: 1
States / cities: Memphis, Tennessee

Conditions: Anemia, Cooley's, Beta-Thalassemia, Hematologic Diseases, Thalassemia, Osteoporosis, Iron Overload, Hypertension, Pulmonary
Interventions: Deferoxamine, Deferiprone, Arginine, Sildenafil, Decitabine; Drug
Lead sponsor: Carelon Research; Other
Eligibility: 1 Year to 75 Years
Enrollment: 1,000 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2000 – 2006
U.S. locations: 4
States / cities: Oakland, California • Boston, Massachusetts • New York, New York + 1 more

Conditions: Sickle Cell Disease
Interventions: Mitapivat, Mitapivat-matching placebo; Drug · Other
Lead sponsor: Agios Pharmaceuticals, Inc.; Industry
Eligibility: 16 Years and older
Enrollment: 286 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2022 – 2030
U.S. locations: 28
States / cities: La Jolla, California • Los Angeles, California • Oakland, California + 20 more

Conditions: Anemia, Sickle Cell
Interventions: Hydroxyurea; Drug
Lead sponsor: National Heart, Lung, and Blood Institute (NHLBI); NIH
Eligibility: 2 Years to 7 Years
Enrollment: 163 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2008 – 2011
U.S. locations: 14
States / cities: Birmingham, Alabama • Washington D.C., District of Columbia • Miami, Florida + 10 more

Conditions: Sickle Cell Disease, Hemolytic Anemia
Interventions: Mitapivat; Drug
Lead sponsor: National Heart, Lung, and Blood Institute (NHLBI); NIH
Eligibility: 18 Years to 70 Years
Enrollment: 15 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2020 – 2028
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Sickle Cell Disease, Sickle Cell Anemia, Anemia, Sickle Cell
Interventions: Not listed
Lead sponsor: National Heart, Lung, and Blood Institute (NHLBI); NIH
Eligibility: 18 Years and older
Enrollment: 106 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2012 – 2015
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Fanconi's Anemia
Interventions: anti-thymocyte globulin, cyclophosphamide, cyclosporine, filgrastim, fludarabine, methylprednisolone; Drug
Lead sponsor: University of Minnesota; Other
Eligibility: 0 Years to 34 Years
Enrollment: 30 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: Started 2000
U.S. locations: 1
States / cities: Minneapolis, Minnesota

Conditions: Sickle Cell Anemia, Priapism
Interventions: tadalafil, placebo; Drug
Lead sponsor: Johns Hopkins University; Other
Eligibility: 18 Years to 45 Years · Male only
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2006 – 2009
U.S. locations: 1
States / cities: Baltimore, Maryland

Conditions: Sickle Cell Disease
Interventions: SuCCESs; Behavioral
Lead sponsor: University of Maryland, Baltimore; Other
Eligibility: 6 Years to 17 Years
Enrollment: 20 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2023 – 2026
U.S. locations: 2
States / cities: Baltimore, Maryland

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Gene Transfer for Sickle Cell Disease

Cannabinoids for the Reduction of Inflammation and Sickle Cell Related Pain

Alemtuzumab, Fludarabine, and Busulfan Followed By Donor Stem Cell Transplant in Treating Young Patients With Hematologic Disorders

Flu Vaccine in Preventing Influenza Infection in Healthy Volunteers and in Patients Who Have Undergone Stem Cell Transplant

A Study of Etavopivat in Patients With Thalassemia or Sickle Cell Disease

Deferoxamine for Sickle Cell Chronic Leg Ulcer Treatment

Examining Cognitive Function and Brain Abnormalities in Adults With Sickle Cell Disease - Pilot Intervention Study

Clinical Transplant-Related Long-term Outcomes of Alternative Donor Allogeneic Transplantation (BMT CTN 1702)

AB1 in Adult Patients with Sickle Cell Disease (SCD)

Congenital Hemolytic and Dyserythropoietic Anemias

Evaluation of Hydroxyurea Plus L-arginine or Sildenafil to Treat Sickle Cell Anemia

Allogeneic Stem Cell Transplantation, Severe Homzygous 0/+Thalassemia or Sever Variants of Beta 0/+ Thalassemia, THALLO

Developing the Family Map: Looking at Communal Coping

Rebeccamycin Analog in Treating Patients With Relapsed or Refractory Acute Myeloid Leukemia, Myelodysplastic Syndrome, Acute Lymphoblastic Leukemia, or Chronic Myelogenous Leukemia

Fresh Versus Old Red Blood Cells for Transfusion

Familial Investigations of Childhood Cancer Predisposition

Thalassemia (Cooley's Anemia) Clinical Research Network (TCRN)

A Study Evaluating the Efficacy and Safety of Mitapivat (AG-348) in Participants With Sickle Cell Disease (RISE UP)

Long-Term Effects of Hydroxyurea in Children With Sickle Cell Anemia (The BABY HUG Follow-up Study)

Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Long-term Mitapivat Dosing in Subjects With Stable Sickle Cell Disease: An Extension of a Phase I Pilot Study of Mitapivat

Carbon Monoxide Levels and Sickle Cell Disease Severity

Phase I/II Study of Total Body Irradiation, Cyclophosphamide, and Fludarabine Followed by Alternate Donor Hematopoietic Cell Transplantation in Patients With Fanconi's Anemia

Tadalafil for Treatment of Priapism in Men With Sickle Cell Anemia

Sickle Cell Children's Exercise Study (SuCCESs)