- 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
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Showing 1–24
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matching trials from the live ClinicalTrials.gov search.
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- 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
- Sickle Cell Disease, Sickle Cell Anemia (HbSS, or HbSβ-thalassemia0), Beta-Thalassemia, Transfusion Dependent Beta-Thalassaemia
- Interventions
- autologous bone marrow derived CD34+ HSPCs electroporated with BCL11A enhancer targeting Cas9 ribonucleoprotein, Sequencing Assay for Variant rs114518452
- Biological · Device
- Lead sponsor
- Daniel Bauer
- Other
- Eligibility
- 13 Years to 40 Years
- Enrollment
- 10 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2025 – 2030
- U.S. locations
- 1
- States / cities
- Boston, Massachusetts
- Conditions
- Beta-Thalassemia
- Interventions
- ACE-536
- Drug
- Lead sponsor
- Celgene
- Industry
- Eligibility
- 6 Years to 17 Years
- Enrollment
- 99 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2035
- U.S. locations
- 2
- States / cities
- Los Angeles, California • New York, 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 21 Years
- Enrollment
- 68 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2025
- U.S. locations
- 1
- States / cities
- Chicago, Illinois
- 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
- Non-transfusion Dependent Thalassemia
- Interventions
- deferasirox, placebo
- Drug
- Lead sponsor
- Novartis Pharmaceuticals
- Industry
- Eligibility
- 10 Years and older
- Enrollment
- 166 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2008 – 2012
- U.S. locations
- 3
- States / cities
- Oakland, California • Chicago, Illinois • New York, New York
- Conditions
- Alpha and Beta Thalassemia, Sickle Cell Disease, Malaria, Human Physiology
- Interventions
- Not listed
- Lead sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
- NIH
- Eligibility
- 18 Years to 70 Years
- Enrollment
- 300 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2019 – 2029
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- 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
- Beta-Thalassemia, Thalassemia, Genetic Diseases, Inborn, Hematologic Diseases, Hemoglobinopathies
- Interventions
- CTX001
- Biological
- Lead sponsor
- Vertex Pharmaceuticals Incorporated
- Industry
- Eligibility
- 2 Years to 11 Years
- Enrollment
- 16 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022 – 2027
- U.S. locations
- 1
- States / cities
- Nashville, Tennessee
- Conditions
- Beta-Thalassemia
- Interventions
- VIT-2763 60 mg QD, VIT-2763 60 mg BID, VIT-2763 120 mg BID, Placebo
- Drug
- Lead sponsor
- Vifor (International) Inc.
- Industry
- Eligibility
- 18 Years to 65 Years
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2023
- U.S. locations
- 1
- States / cities
- Whittier, California
- Conditions
- Beta-Thalassemia
- Interventions
- ICL670, deferoxamine
- Drug
- Lead sponsor
- Novartis Pharmaceuticals
- Industry
- Eligibility
- 2 Years and older
- Enrollment
- 595 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- Started 2003
- U.S. locations
- 7
- States / cities
- Los Angeles, California • Oakland, California • Stanford, California + 4 more
- Conditions
- Sickle Red Blood Cell, Fetal Hemoglobin, Oxidative Stress
- Interventions
- Broccosprouts® (Brassica Protection Products LLC) homogenate
- Dietary Supplement
- Lead sponsor
- Duke University
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 21 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2012 – 2015
- U.S. locations
- 1
- States / cities
- Durham, North Carolina
- Conditions
- Sickle Cell Disease, Sickle Cell Anemia, Hemoglobin SS, Hemoglobin SC, Hemoglobin Beta Thalassemia
- Interventions
- Decision Aid Tool, Standard Practice
- Other
- Lead sponsor
- Emory University
- Other
- Eligibility
- 8 Years to 80 Years
- Enrollment
- 134 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2015 – 2017
- U.S. locations
- 1
- States / cities
- Atlanta, Georgia
- Conditions
- Beta-thalassemia, Myelodysplastic Syndromes, Fanconi Syndrome, Anemia, Diamond-Blackfan, Anemia, Aplastic
- Interventions
- Deferasirox
- Drug
- Lead sponsor
- Novartis Pharmaceuticals
- Industry
- Eligibility
- 2 Years and older
- Enrollment
- 175 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- Started 2003
- U.S. locations
- 6
- States / cities
- Oakland, California • Stanford, California • Arlington Heights, Illinois + 3 more
- Conditions
- Beta-Thalassemia
- Interventions
- LJPC-401
- Drug
- Lead sponsor
- La Jolla Pharmaceutical Company
- Industry
- Eligibility
- 14 Years and older
- Enrollment
- 84 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2017 – 2020
- U.S. locations
- 2
- States / cities
- Oakland, California • New York, New York
- Conditions
- Congenital Hemolytic Anemia, Sickle Cell Disease
- Interventions
- Peripheral blood hematopoietic progenitor cell (PBPC) transplant, Alemtuzumab, Peripheral blood hematopoietic progenitor cell Apheresis, Sirolimus
- Procedure · Drug
- Lead sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
- NIH
- Eligibility
- 2 Years to 80 Years
- Enrollment
- 130 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2004 – 2028
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- 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
- 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
- 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
- Hemoglobin SC Disease, Sickle Cell Anemia
- Interventions
- Recombinant-methionyl human stem cell factor
- Drug
- Lead sponsor
- National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
- NIH
- Eligibility
- Not listed
- Enrollment
- 50 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2000
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Sickle Cell Disease, Beta Thalassemia-Major
- Interventions
- alemtuzumab (Campath IH), Fludarabine, Melphalan, Cyclosporine, Mycophenolate mofetil, Tacrolimus, Hematopoietic Stem Cell Transplantation
- Drug · Biological
- Lead sponsor
- Northwell Health
- Other
- Eligibility
- 1 Year to 21 Years
- Enrollment
- 29 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2008 – 2019
- U.S. locations
- 1
- States / cities
- New Hyde Park, New York
- 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
- 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