- Conditions
- Sickle Cell Disease
- Interventions
- IMR-687, Placebo
- Drug
- Lead sponsor
- Cardurion Pharmaceuticals, Inc.
- Industry
- Eligibility
- 18 Years to 55 Years
- Enrollment
- 100 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2020
- U.S. locations
- 7
- States / cities
- Oakland, California • Farmington, Connecticut • Hollywood, Florida + 3 more
Search Results
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Showing 1–24
of 133
matching trials from the live ClinicalTrials.gov search.
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- Conditions
- Sickle Cell Disease, Thalassemia, Diamond-Blackfan Anemia
- Interventions
- Not listed
- Lead sponsor
- UCSF Benioff Children's Hospital Oakland
- Other
- Eligibility
- 16 Years and older
- Enrollment
- 20 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2009 – 2013
- U.S. locations
- 1
- States / cities
- Oakland, California
- Conditions
- Sickle Cell Disease
- Interventions
- Autologous CD34+ HSC cells transduced with the lentiviral vector containing a shRNA targeting BCL11a
- Biological
- Lead sponsor
- David Williams
- Other
- Eligibility
- 13 Years to 55 Years
- U.S. locations
- 1
- States / cities
- Boston, Massachusetts
- Conditions
- Sickle Cell Disease, Thalassemia
- Interventions
- Video recording, Medication Administration Log, Education
- Behavioral
- Lead sponsor
- Duke University
- Other
- Eligibility
- 5 Years and older
- Enrollment
- 46 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2014 – 2016
- U.S. locations
- 1
- States / cities
- Durham, North Carolina
- Conditions
- Anemia, Sickle Cell, Complex and Transfusion-dependent Hemoglobinopathies, Thalassemia, Diamond-Blackfan Anemia, Bone Marrow Failure Syndromes, Alpha-Thalassemia, Beta-Thalassemia
- Interventions
- Enriched Hematopoetic Stem Cell Infusion
- Biological
- Lead sponsor
- Talaris Therapeutics Inc.
- Industry
- Eligibility
- Up to 45 Years
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2011 – 2016
- U.S. locations
- 3
- States / cities
- Chicago, Illinois • Louisville, Kentucky • Durham, North Carolina
- Conditions
- Healthy Volunteers Sickle Cell Disease, Thalassemia
- Interventions
- Etavopivat, Digoxin, Pitavastatin, Metformin, Midazolam, Rosuvastatin
- Drug
- Lead sponsor
- Novo Nordisk A/S
- Industry
- Eligibility
- 18 Years to 55 Years
- Enrollment
- 37 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2025
- U.S. locations
- 1
- States / cities
- Salt Lake City, Utah
- Conditions
- Sickle Cell Disease
- Interventions
- Autologous CD34+ HSC cells transduced with the lentiviral vector containing a shRNA targeting BCL11a
- Biological
- Lead sponsor
- David Williams
- Other
- Eligibility
- 13 Years to 40 Years
- Enrollment
- 25 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022 – 2027
- U.S. locations
- 9
- States / cities
- Los Angeles, California • Oakland, California • Sacramento, California + 4 more
- Conditions
- Sickle Cell Disease, Thalassemia
- Interventions
- CordIn
- Biological
- Lead sponsor
- Gamida Cell ltd
- Industry
- Eligibility
- 2 Years to 25 Years
- Enrollment
- 1 participant
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2016 – 2017
- U.S. locations
- 2
- States / cities
- Oakland, California • Washington D.C., District of Columbia
- Conditions
- Sickle Cell Disease, HEMOGLOBIN SS, Hemoglobin S Beta-0 Thalassemia, Inflammation, Quality of Life
- Interventions
- Omega-3 Fatty Acids: Eicosapentaenoic Acid (EPA) and Docosahexaenoic Acid (DHA), Placebo Capsules
- Dietary Supplement · Other
- Lead sponsor
- Thomas Jefferson University
- Other
- Eligibility
- 10 Years to 19 Years
- Enrollment
- 48 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2010 – 2012
- U.S. locations
- 2
- States / cities
- Philadelphia, Pennsylvania
- Conditions
- Hematologic Disorder, Bleeding Disorder, Connective Tissue Disorder, Hemophilia, Thrombosis, Von Willebrand Diseases, Thrombophilia, Rare Bleeding Disorder, Platelet Disorder, Factor IX Deficiency, Factor VIII Deficiency, Thalassemia, Sickle Cell Disease
- Interventions
- Not listed
- Lead sponsor
- American Thrombosis and Hemostasis Network
- Network
- Eligibility
- Not listed
- Enrollment
- 3,000 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2020 – 2035
- U.S. locations
- 71
- States / cities
- Phoenix, Arizona • Little Rock, Arkansas • Los Angeles, California + 59 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
- 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
- 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
- Sickle Cell Anemia, Sickle ß0-Thalassemia
- Interventions
- L-glutamine, Placebo
- Drug
- Lead sponsor
- Emmaus Medical, Inc.
- Industry
- Eligibility
- 5 Years and older
- Enrollment
- 230 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2010 – 2014
- U.S. locations
- 31
- States / cities
- Mobile, Alabama • Phoenix, Arizona • Inglewood, California + 23 more
- Conditions
- Hematologic Malignancies, Nonmalignant Diseases, Immune Deficiency, Hemoglobinopathies, Genetic Inborn Errors of Metabolism, Fanconi Anemia, Thalassemia, Sickle Cell Disease
- Interventions
- Thiotepa
- Drug
- Lead sponsor
- University of California, San Francisco
- Other
- Eligibility
- Up to 17 Years
- Enrollment
- 25 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2017 – 2023
- U.S. locations
- 1
- States / cities
- San Francisco, California
- 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
- 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
- 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
- Sickle Cell Disease, Thalassemia, High Risk Hematologic Disorders, Cerebral Adrenoleukodystrophy, Inherited Metabolic Disorders
- Interventions
- Blood and Marrow Transplant
- Procedure
- Lead sponsor
- Masonic Cancer Center, University of Minnesota
- Other
- Eligibility
- 0 Years to 25 Years
- Enrollment
- 5 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2022
- U.S. locations
- 1
- States / cities
- Minneapolis, Minnesota
- Conditions
- Anemia, Sickle Cell
- Interventions
- Siplizumab, Exchange Transfusion, Total Body Irradiation, Stem Cell Infusion, Cyclophosphamide 50mg, Mesna, Sirolimus Oral Tablet, Rituximab or Biosimilar
- Drug · Procedure
- Lead sponsor
- Columbia University
- Other
- Eligibility
- 18 Years to 50 Years
- Enrollment
- 1 participant
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2023 – 2025
- U.S. locations
- 1
- States / cities
- New York, New York
- Conditions
- Sickle Cell Disease, Hemoglobin SC
- Interventions
- Busulfan, Campath 1H, Cyclophosphamide and MESNA
- Drug · Biological
- Lead sponsor
- Tami D. John
- Other
- Eligibility
- Up to 40 Years
- Enrollment
- 8 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2005 – 2012
- U.S. locations
- 2
- States / cities
- Houston, Texas
- Conditions
- Sickle Cell Anemia, Thalassemia
- Interventions
- L-Glutamine, Placebo
- Drug
- Lead sponsor
- Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 15 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2004 – 2009
- U.S. locations
- 1
- States / cities
- Torrance, California
- 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
- Transfusional Iron Overload, Beta-Thalassemia
- Interventions
- SPD602
- Drug
- Lead sponsor
- Shire
- Industry
- Eligibility
- 6 Years to 17 Years
- Enrollment
- 30 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2011 – 2014
- U.S. locations
- 2
- States / cities
- Boston, Massachusetts • Philadelphia, Pennsylvania