- Conditions
- Non-Transfusion Dependent Beta-Thalassemia (NTDT)
- Interventions
- REGN7999, Placebo
- Drug
- Lead sponsor
- Regeneron Pharmaceuticals
- Industry
- Eligibility
- 18 Years to 65 Years
- Enrollment
- 19 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024 – 2026
- U.S. locations
- 1
- States / cities
- New York, New York
Search Results
Search by objective public record fields.
Search by query text, NCT ID, condition, intervention, sponsor, city, state, recruitment status, phase, study type, healthy volunteer eligibility, sex, or age. Results come from ClinicalTrials.gov. When the live registry is unavailable, a clearly labeled stored copy may be shown.
These results were retrieved from the live ClinicalTrials.gov registry. Filters and sort order apply to this result list.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Showing 73–96
of 100
matching trials from the live ClinicalTrials.gov search.
- Conditions
- Sickle Cell Disease, Thalassemia, Beta, Thalassemia
- Interventions
- Fludarabine, Cyclophosphamide, Tacrolimus, Mycophenolate Mofetil, Rabbit ATG, Dexamethasone, Bortezomib, Rituximab
- Drug · Biological
- Lead sponsor
- Memorial Sloan Kettering Cancer Center
- Other
- Eligibility
- 2 Years to 50 Years
- Enrollment
- 24 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2023 – 2027
- U.S. locations
- 6
- States / cities
- Basking Ridge, New Jersey • Middletown, New Jersey • Montvale, New Jersey + 3 more
- Conditions
- Non Malignant Disorders, Immunodeficiencies, Congenital Marrow Failures, Hemoglobinopathies, Inborn Errors of Metabolism, Sickle Cell, Thalassemia, Lysosomal Storage Disease
- Interventions
- Unrelated Umbilical Cord Blood Transplant, Reduced Intensity Conditioning
- Biological · Drug
- Lead sponsor
- Duke University
- Other
- Eligibility
- Up to 21 Years
- Enrollment
- 22 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2008 – 2014
- U.S. locations
- 1
- States / cities
- Durham, North Carolina
- Conditions
- Transfusion-dependent Alpha-Thalassemia, Transfusion-dependent Beta-Thalassemia
- Interventions
- Mitapivat Matched Placebo, Mitapivat
- Drug
- Lead sponsor
- Agios Pharmaceuticals, Inc.
- Industry
- Eligibility
- 1 Year to 17 Years
- Enrollment
- 54 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2026 – 2032
- U.S. locations
- 4
- States / cities
- Atlanta, Georgia • New York, New York • Philadelphia, Pennsylvania + 1 more
- Conditions
- Alpha-Thalassemia, Beta-Thalassemia, Amyloidosis, Amyotrophic Lateral Sclerosis, Creutzfeld-Jakob Disease, Cystic Fibrosis, Duchenne Muscular Dystrophy, Early-Onset Alzheimer Disease, Ehlers-Danlos Syndrome, Huntington Disease, Gaucher Disease, GM1 Gangliosidosis, Myasthenia Gravis, Pompe Disease, Sickle Cell Disease, Transthyretin Amyloid Cardiomyopathy, Rare Diseases
- Interventions
- Not listed
- Lead sponsor
- xCures
- Industry
- Eligibility
- Not listed
- Enrollment
- 1,000 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024 – 2026
- U.S. locations
- 1
- States / cities
- Los Altos, California
- Conditions
- Thalassemia, Sickle Cell Disease, Diamond Blackfan Anemia, Myelofibrosis
- Interventions
- Deferasirox
- Drug
- Lead sponsor
- Novartis Pharmaceuticals
- Industry
- Eligibility
- 2 Years and older
- Enrollment
- 1,683 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2005 – 2008
- U.S. locations
- 27
- States / cities
- Little Rock, Arkansas • Berkeley, California • Orange, California + 23 more
- Conditions
- Thalassemia
- Interventions
- Luspatercept, Placebo, Best Supportive Care (BSC)
- Drug · Other
- Lead sponsor
- Celgene
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 145 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2022
- U.S. locations
- 4
- States / cities
- Los Angeles, California • Oakland, California • Chicago, Illinois
- Conditions
- Non-Neoplastic Hematologic and Lymphocytic Disorder
- Interventions
- Allogeneic Bone Marrow Transplantation, Anti-Thymocyte Globulin, Cyclosporine, Fludarabine Phosphate, Laboratory Biomarker Analysis, Methotrexate, Mycophenolate Mofetil, Peripheral Blood Stem Cell Transplantation, Tacrolimus, Total-Body Irradiation, Treosulfan, Umbilical Cord Blood Transplantation
- Procedure · Biological · Drug + 2 more
- Lead sponsor
- Fred Hutchinson Cancer Center
- Other
- Eligibility
- Up to 49 Years
- Enrollment
- 98 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2009 – 2020
- U.S. locations
- 6
- States / cities
- Aurora, Colorado • Portland, Oregon • Nashville, Tennessee + 2 more
- 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
- Primary Immune Deficiency Disorders, Hemophagocytic Lymphohistiocytosis, Inherited Bone Marrow Failure Syndrome, Hemoglobinopathies, Metabolic Disorders
- Interventions
- BPX-501 and Rimiducid
- Biological
- Lead sponsor
- Bellicum Pharmaceuticals
- Industry
- Eligibility
- 4 Months to 55 Years
- Enrollment
- 1 participant
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2015 – 2018
- U.S. locations
- 1
- States / cities
- Seattle, Washington
- Conditions
- Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes, Inherited Metabolic Disorders (IMD), Hereditary Anemias, Inflammatory Conditions
- Interventions
- data collection
- Drug
- Lead sponsor
- Paul Szabolcs
- Other
- Eligibility
- 2 Months to 60 Years
- Enrollment
- 50 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2020 – 2028
- U.S. locations
- 1
- States / cities
- Pittsburgh, Pennsylvania
- Conditions
- Hydrops Fetalis, Birth Defect, Fetal Anomaly
- Interventions
- Exome sequencing
- Diagnostic Test
- Lead sponsor
- University of California, San Francisco
- Other
- Eligibility
- 18 Years to 55 Years
- Enrollment
- 500 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2027
- U.S. locations
- 1
- States / cities
- San Francisco, California
- Conditions
- Sickle Cell Disease, Thalassemia, Hemoglobinopathies
- Interventions
- Busulfan; Fludarabine; cyclosporine A and MMF
- Other
- Lead sponsor
- University of Pittsburgh
- Other
- Eligibility
- 3 Years to 35 Years
- Enrollment
- 8 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2002 – 2014
- U.S. locations
- 1
- States / cities
- Pittsburgh, Pennsylvania
- Conditions
- Alpha Thalassemia Major, Hemoglobinopathy; With Thalassemia, Hemoglobinopathies, Fetal Anemia, Fetal Hydrops, Alpha; Thalassemia, Thalassemia Major, Thalassemia Alpha, A-Thalassemia
- Interventions
- in utero hematopoietic stem cell transplantation
- Biological
- Lead sponsor
- University of California, San Francisco
- Other
- Eligibility
- 18 Weeks to 26 Weeks
- Enrollment
- 6 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2017 – 2024
- U.S. locations
- 1
- States / cities
- San Francisco, California
- Conditions
- Beta-Thalassemia
- Interventions
- LentiGlobin BB305 Drug Product
- Genetic
- Lead sponsor
- Genetix Biotherapeutics Inc.
- Industry
- Eligibility
- 0 Years to 50 Years
- Enrollment
- 19 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2017 – 2022
- U.S. locations
- 3
- States / cities
- Oakland, California • Chicago, Illinois • Philadelphia, Pennsylvania
- 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
- Healthy Volunteers, Sickle Cell Disease, Thalassemia
- Interventions
- Etavopivat, Moxifloxacin, Placebo
- Drug
- Lead sponsor
- Novo Nordisk A/S
- Industry
- Eligibility
- 18 Years to 55 Years
- Enrollment
- 33 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2025
- U.S. locations
- 1
- States / cities
- Baltimore, Maryland
- 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
- 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
- Conditions
- Hematologic Malignancy, Acute Leukemia, Remission, Acute Myeloid Leukemia, Acute Lymphoblastic Leukemia, AML, TP53, Intrachromosomal Amplification of Chromosome 21, Cytogenetic Abnormality, CNS Leukemia, Minimal Residual Disease, Myelodysplasia, Juvenile Myelomonocytic Leukemia, Somatic Mutation, PTPN11 Gene Mutation, N-RAS Gene Amplification, Neurofibromatosis 1, NF1 Mutation, CBL Gene Mutation, Monosomy 7, Chromosome Abnormality, Fetal Hemoglobin, Lymphoblastic Lymphoma, High Grade Non-Hodgkin's Lymphoma, Adult
- Interventions
- Fludarabine, Busulfan, Melphalan, Rituximab, Levetiracetam, Alpha/Beta T Cell-Depleted Hematopoietic Stem Cells, Thymoglobulin, Cyclophosphamide
- Drug · Biological
- Lead sponsor
- Masonic Cancer Center, University of Minnesota
- Other
- Eligibility
- Up to 60 Years
- Enrollment
- 70 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2023 – 2030
- U.S. locations
- 1
- States / cities
- Minneapolis, Minnesota
- 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
- Thalassemia
- Interventions
- Not listed
- Lead sponsor
- Carelon Research
- Other
- Eligibility
- 5 Years and older
- Enrollment
- 416 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2007 – 2011
- U.S. locations
- 10
- States / cities
- Los Angeles, California • Oakland, California • Stanford, California + 7 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
- 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