- Conditions
- Sickle Cell Disease
- Interventions
- Pociredir
- Drug
- Lead sponsor
- Fulcrum Therapeutics
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 50 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2026 – 2030
- U.S. locations
- 8
- States / cities
- Little Rock, Arkansas • Los Angeles, California • Baton Rouge, Louisiana + 5 more
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 49–72
of 111
matching trials from the live ClinicalTrials.gov search.
- 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
- Age-Related Macular Degeneration, Allergies, Alpha-Gal Syndrome, Alzheimer Disease, Amyloidosis, Ankylosing Spondylitis, Arthritis, Alopecia Areata, Asthma, Atopic Dermatitis, Autism, Autoimmune Hepatitis, Behcet's Disease, Beta-Thalassemia, Cancer, Celiac Disease, Kidney Diseases, COPD, Crohn Disease, Cystic Fibrosis, Diabetes, Dravet Syndrome, DMD, Fibromyalgia, Graves Disease, Thyroid Diseases, Hepatitis, Hidradenitis Suppurativa, ITP, Leukemia, ALS, Lupus or SLE, Lymphoma, Multiple Sclerosis, Myasthenia Gravis, Heart Diseases, Parkinson Disease, Pemphigus Vulgaris, Cirrhosis, Psoriasis, Schizophrenia, Scleroderma, Sickle Cell Disease, Stroke, Ulcerative Colitis, Vasculitis, Vitiligo
- Interventions
- Specimen sample
- Diagnostic Test
- Lead sponsor
- Sanguine Biosciences
- Industry
- Eligibility
- 18 Years to 85 Years
- Enrollment
- 20,000 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2021 – 2025
- U.S. locations
- 1
- States / cities
- Waltham, Massachusetts
- Conditions
- Alpha-Thalassemia, Alpha Thalassemia Major, Alpha Thalassemia Minor
- Interventions
- Not listed
- Lead sponsor
- University of California, San Francisco
- Other
- Eligibility
- Not listed
- Enrollment
- 500 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2017 – 2037
- U.S. locations
- 1
- States / cities
- San Francisco, California
- 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
- Beta-Thalassemia, Thalassemia, Genetic Diseases, Inborn, Hematologic Diseases, Hemoglobinopathies
- Interventions
- CTX001
- Biological
- Lead sponsor
- Vertex Pharmaceuticals Incorporated
- Industry
- Eligibility
- 12 Years to 35 Years
- Enrollment
- 59 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2025
- U.S. locations
- 6
- States / cities
- Palo Alto, California • Chicago, Illinois • New York, New York + 2 more
- Conditions
- Sickle Cell Disease
- Interventions
- Defibrotide
- Drug
- Lead sponsor
- New York Medical College
- Other
- Eligibility
- 6 Months to 21 Years
- Enrollment
- 40 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2017 – 2027
- U.S. locations
- 4
- States / cities
- Los Angeles, California • Gainesville, Florida • Valhalla, New York + 1 more
- Conditions
- Thalassemia Major
- Interventions
- DST-0509, Jadenu, Exjade
- Drug
- Lead sponsor
- DisperSol Technologies, LLC
- Industry
- Eligibility
- 8 Years and older
- Enrollment
- 25 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2021
- U.S. locations
- 1
- States / cities
- New York, New York
- Conditions
- Beta-Thalassemia, Thalassemia, Sickle Cell Disease, Hematologic Diseases, Hemoglobinopathies, Genetic Diseases, Inborn, Sickle Cell Anemia
- Interventions
- CTX001
- Biological
- Lead sponsor
- Vertex Pharmaceuticals Incorporated
- Industry
- Eligibility
- 2 Years and older
- Enrollment
- 160 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2039
- U.S. locations
- 9
- States / cities
- Palo Alto, California • Chicago, Illinois • New York, New York + 5 more
- Conditions
- Hemoglobinopathies, Anemia, Sickle Cell, Hemoglobin SC Disease, Thalassemia, Thalassemia Major
- Interventions
- Busulfan, Fludarabine, FK506, Prednisone
- Drug
- Lead sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
- NIH
- Eligibility
- 18 Years and older
- Enrollment
- 2 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2001 – 2003
- U.S. locations
- 1
- States / cities
- Boston, Massachusetts
- 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 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 Disease, Beta Thalassemia
- Interventions
- Busulfan, Fludarabine, Alemtuzumab, Allogeneic stem cell transplant
- Drug · Procedure
- Lead sponsor
- Columbia University
- Other
- Eligibility
- 1 Month to 30 Years
- Enrollment
- 53 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2004 – 2025
- U.S. locations
- 1
- States / cities
- New York, New York
- Conditions
- Sickle Cell Disease, Beta-thalassemia
- Interventions
- Total Body Irradiation, Alemtuzumab, Abatacept, Research blood draw
- Radiation · Drug · Other
- Lead sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
- NIH
- Eligibility
- 4 Years to 65 Years
- Enrollment
- 90 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2026 – 2035
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- 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
- Erythrocyte Transfusion, Beta-Thalassemia
- Interventions
- Luspatercept, Placebo
- Drug · Other
- Lead sponsor
- Celgene
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 336 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2016 – 2021
- U.S. locations
- 6
- States / cities
- Los Angeles, California • Oakland, California • Chicago, Illinois + 3 more
- Conditions
- Sickle Cell Anemia
- Interventions
- PBSC Transplant, Alemtuzumab, Sirolimus, Cyclophosphamide, Low Dose Irradiation
- Procedure · Drug
- Lead sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
- NIH
- Eligibility
- 2 Years to 80 Years
- Enrollment
- 23 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2009 – 2026
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Transfusional Iron Overload, Beta-thalassemia
- Interventions
- FBS0701
- Drug
- Lead sponsor
- FerroKin BioSciences, Inc.
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 20 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- Started 2009
- U.S. locations
- 3
- States / cities
- Oakland, California • Boston, Massachusetts • Philadelphia, Pennsylvania
- Conditions
- Thrombocytopenia, Metachromatic Leukodystrophy, Fanconi's Anemia, Thalassemia Major, Pure Red-Cell Aplasia, Inborn Errors of Metabolism
- Interventions
- Stem Cell Transplantation
- Procedure
- Lead sponsor
- National Center for Research Resources (NCRR)
- NIH
- Eligibility
- 0 Years to 17 Years
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- Started 1995
- U.S. locations
- 1
- States / cities
- Los Angeles, California
- Conditions
- Beta Thalassemia, Hemoglobinopathy
- Interventions
- Hydroxyurea
- Drug
- Lead sponsor
- National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
- NIH
- Eligibility
- Not listed
- Enrollment
- 100 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 1999 – 2002
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Thalassemia
- Interventions
- Decitabine (USAN, INN)
- Drug
- Lead sponsor
- Carelon Research
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 6 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2008 – 2010
- U.S. locations
- 2
- States / cities
- Oakland, California • Philadelphia, Pennsylvania
- 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
- 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
- Non-Transfusion-dependent Alpha-Thalassemia, Non-Transfusion-dependent Beta-Thalassemia
- Interventions
- Mitapivat, Placebo Matching Mitapivat
- Drug
- Lead sponsor
- Agios Pharmaceuticals, Inc.
- Industry
- Eligibility
- 1 Year to 17 Years
- Enrollment
- 45 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2026 – 2032
- U.S. locations
- 5
- States / cities
- Palo Alto, California • Atlanta, Georgia • New York, New York + 2 more