- Conditions
- Sickle Cell Anemia, Beta-thalassemia Major, Diamond-blackfan Anemia
- Interventions
- CD3/CD19 depleted leukocytes, CD45RA depleted leukocytes, Hydroxyurea, Rituximab, Alemtuzumab, Fludarabine, Thiotepa
- Biological · Drug
- Lead sponsor
- Paul Szabolcs
- Other
- Eligibility
- 5 Years to 40 Years
- Enrollment
- 5 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2027
- U.S. locations
- 1
- States / cities
- Pittsburgh, Pennsylvania
Search Results
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Showing 25–48
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- 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
- Transfusion-dependent Beta-Thalassemia
- Interventions
- Safety and efficacy assessments
- Other
- Lead sponsor
- Genetix Biotherapeutics Inc.
- Industry
- Eligibility
- 0 Years to 50 Years
- Enrollment
- 66 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2014 – 2035
- U.S. locations
- 6
- States / cities
- Oakland, California • Chicago, Illinois • Bethesda, Maryland + 3 more
- Conditions
- Acute Leukemia, Severe Aplastic Anemia, Non-hodgkin Lymphoma, Hodgkin Lymphoma, Kostmann, Diamond Blackfan Anemia, Amegakaryocytic Thrombocytopenia, Sickle Cell Disease, Beta-Thalassemia
- Interventions
- alpha beta depletion
- Drug
- Lead sponsor
- Mitchell Cairo
- Other
- Eligibility
- 1 Day to 30 Years
- Enrollment
- 20 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2027
- U.S. locations
- 1
- States / cities
- Valhalla, New York
- Conditions
- Sickle Cell Disease, Sickle B+ Thalassemia, Sickle Beta Zero Thalassemia, Sickle Cell Hemoglobin C
- Interventions
- HU-Go app
- Other
- Lead sponsor
- Ann & Robert H Lurie Children's Hospital of Chicago
- Other
- Eligibility
- 12 Years to 25 Years
- Enrollment
- 33 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2020
- U.S. locations
- 1
- States / cities
- Chicago, Illinois
- Conditions
- Sickle Cell Anemia, Sickle Cell Thalassemia, Sickle Cell Pain, Hbss, Hbsc, Sickle Beta Thalassemia, Sickle Beta Zero Thalassemia, Sickle Cell Syndrome Variant
- Interventions
- Not listed
- Lead sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
- NIH
- Eligibility
- 16 Years to 80 Years
- Enrollment
- 6 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024 – 2027
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Hematologic Diseases, Anemia, Sickle Cell, Beta-Thalassemia, Hematopoietic Stem Cell Transplantation
- Interventions
- Sangstat, Cyclophosphamide, Busulfan, Mycophenolate Mofetil, Cyclosporine, Cord Blood Transplantation
- Drug · Procedure
- Lead sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
- NIH
- Eligibility
- 3 Years to 14 Years
- Enrollment
- 30 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 1999 – 2006
- U.S. locations
- 14
- States / cities
- Oakland, California • Washington D.C., District of Columbia • Jacksonville, Florida + 10 more
- Conditions
- Anemia, Sickle Cell
- Interventions
- pomalidomide
- Drug
- Lead sponsor
- Celgene
- Industry
- Eligibility
- 18 Years to 60 Years
- Enrollment
- 12 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2007 – 2013
- U.S. locations
- 1
- States / cities
- Detroit, Michigan
- Conditions
- Beta Thalassemia
- Interventions
- 5-Azacytidine
- Drug
- Lead sponsor
- National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
- NIH
- Eligibility
- Not listed
- Enrollment
- 24 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2000 – 2003
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Beta Thalassemia Intermedia, Sickle Cell Disease
- Interventions
- Benserazide Only Product
- Drug
- Lead sponsor
- Phoenicia BioScience
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 36 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2020 – 2026
- U.S. locations
- 4
- States / cities
- Oakland, California • Boston, Massachusetts • Weston, Massachusetts + 1 more
- Conditions
- β-thalassemia, Ineffective Erythropoiesis, Chronic Anemia
- Interventions
- PTG-300
- Drug
- Lead sponsor
- Protagonist Therapeutics, Inc.
- Industry
- Eligibility
- 12 Years to 65 Years
- Enrollment
- 63 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2020
- U.S. locations
- 3
- States / cities
- Oakland, California • Boston, Massachusetts • Philadelphia, Pennsylvania
- Conditions
- Skin Ulcers, Sickle Cell Anemia
- Interventions
- Arginine Butyrate, Standard local care dressing
- Drug · Other
- Lead sponsor
- Susan P. Perrine
- Other
- Eligibility
- 16 Years to 60 Years
- Enrollment
- 23 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 1997 – 2005
- U.S. locations
- 4
- States / cities
- Chicago, Illinois • Boston, Massachusetts • New York, New York + 1 more
- Conditions
- Thalassemia Major
- Interventions
- Vitamin D3, Placebo
- Drug
- Lead sponsor
- Weill Medical College of Cornell University
- Other
- Eligibility
- 6 Years to 60 Years
- Enrollment
- 40 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2011 – 2014
- U.S. locations
- 1
- States / cities
- New York, New York
- Conditions
- Thalassemia Major With Severe Transfusional Iron Overload
- Interventions
- Deferasirox and deferiprone
- Drug
- Lead sponsor
- Children's Hospital of Philadelphia
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 9 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2012 – 2017
- U.S. locations
- 2
- States / cities
- Chicago, Illinois • Philadelphia, Pennsylvania
- 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
- Beta-Thalassemia, Thalassemia, Hematologic Diseases, Genetic Diseases, Inborn, Hemoglobinopathies, Sickle Cell Disease, Sickle Cell Anemia
- Interventions
- CTX001
- Biological
- Lead sponsor
- Vertex Pharmaceuticals Incorporated
- Industry
- Eligibility
- 12 Years to 35 Years
- Enrollment
- 26 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022 – 2027
- U.S. locations
- 3
- States / cities
- New York, New York • Charlotte, North Carolina • Nashville, Tennessee
- Conditions
- Blood and Lymphatic Diseases
- Interventions
- BIVV003, ST-400
- Drug
- Lead sponsor
- Sangamo Therapeutics
- Industry
- Eligibility
- 18 Years to 45 Years
- Enrollment
- 8 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2038
- U.S. locations
- 7
- States / cities
- Oakland, California • Sacramento, California • Atlanta, Georgia + 3 more
- Conditions
- Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes, Inherited Metabolic Disorders (IMD), Hereditary Anemias, Inflammatory Conditions, Systemic Juvenile Idiopathic Arthritis (sJIA), Juvenile Rheumatoid Arthritis (JRA)
- Interventions
- Hydroxyurea, Alemtuzumab, Fludarabine, Melphalan, Thiotepa
- Drug
- Lead sponsor
- Paul Szabolcs
- Other
- Eligibility
- 2 Months to 55 Years
- Enrollment
- 100 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2014 – 2027
- U.S. locations
- 1
- States / cities
- Pittsburgh, Pennsylvania
- Conditions
- Transfusion-dependent Alpha-Thalassemia, Transfusion-dependent Beta-Thalassemia
- Interventions
- Placebo Matching Mitapivat, Mitapivat
- Drug
- Lead sponsor
- Agios Pharmaceuticals, Inc.
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 258 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2029
- U.S. locations
- 10
- States / cities
- Phoenix, Arizona • La Jolla, California • Oakland, California + 7 more
- Conditions
- Thalassemia Major
- Interventions
- Not listed
- Lead sponsor
- Dana-Farber Cancer Institute
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 40 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2004 – 2005
- U.S. locations
- 1
- States / cities
- Boston, Massachusetts
- Conditions
- Transfusional Iron Overload, Thalassemia Major, Sickle Cell Disease, Myelodysplastic Syndromes, Aplastic Anemia
- Interventions
- Hepatic biomagnetic susceptibility measurement
- Device
- Lead sponsor
- Columbia University
- Other
- Eligibility
- 5 Years to 80 Years
- Enrollment
- 99 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2011 – 2016
- U.S. locations
- 1
- States / cities
- New York, New York
- Conditions
- Transfusion-dependent Hemachromatosis, Thalassemia Major, Sickle Cell Disease
- Interventions
- Deferoxamine, Deferasirox, HIDA
- Drug · Radiation
- Lead sponsor
- Boston Children's Hospital
- Other
- Eligibility
- 6 Years and older
- Enrollment
- 15 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2008
- U.S. locations
- 1
- States / cities
- Boston, Massachusetts
- Conditions
- Sickle Cell Anemia
- Interventions
- Ambrisentan, Placebo
- Drug
- Lead sponsor
- Augusta University
- Other
- Eligibility
- 18 Years to 65 Years
- Enrollment
- 26 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2015 – 2019
- U.S. locations
- 1
- States / cities
- Augusta, Georgia
- Conditions
- Transfusional Iron Overload, Beta-thalassemia
- Interventions
- SPD602 (FBS0701, SSP-004184)
- Drug
- Lead sponsor
- Shire
- Industry
- Eligibility
- 18 Years to 60 Years
- Enrollment
- 51 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2010 – 2013
- U.S. locations
- 2
- States / cities
- Oakland, California • Boston, Massachusetts