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ClinicalTrials.gov public records Last synced Oct 14, 2026, 7:50 AM EDT

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Showing 217–240 of 1,044 matching trials from the live ClinicalTrials.gov search.
Recruiting No phase listed Observational Accepts healthy volunteers

The Congenital Dyserythropoietic Anemia Registry (CDAR)

NCT02964494
Conditions
Congenital Dyserythropoietic Anemia (CDA)
Interventions
Not listed
Lead sponsor
Children's Hospital Medical Center, Cincinnati
Other
Eligibility
Not listed
Enrollment
10,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2016 – 2031
U.S. locations
1
States / cities
Cincinnati, Ohio
Conditions
Pyruvate Kinase Deficiency, Glucose 6 Phosphate Dehydrogenase Deficiency
Interventions
Vitamin C
Drug
Lead sponsor
University of Utah
Other
Eligibility
18 Years and older
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2027
U.S. locations
1
States / cities
Salt Lake City, Utah
Conditions
Sickle Cell Disease
Interventions
GBT021601
Drug
Lead sponsor
Pfizer
Industry
Eligibility
18 Years to 55 Years
Enrollment
129 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2020 – 2023
U.S. locations
1
States / cities
San Antonio, Texas
Conditions
Sickle Cell Disease
Interventions
buprenorphine
Drug
Lead sponsor
Johns Hopkins University
Other
Eligibility
18 Years and older
Enrollment
47 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2022
U.S. locations
1
States / cities
Baltimore, Maryland
Completed Phase 1 Interventional Accepts healthy volunteers Results available

Voxelotor CYP and Transporter Cocktail Interaction Study

NCT05981365
Conditions
Sickle Cell Disease
Interventions
Voxelotor, Bupropion, Repaglinide, Flurbiprofen, Omeprazole, Midazolam, Metformin, Furosemide, Rosuvastatin
Drug
Lead sponsor
Pfizer
Industry
Eligibility
18 Years to 55 Years
Enrollment
44 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2023
U.S. locations
1
States / cities
San Antonio, Texas
Conditions
Sickle Cell Disease
Interventions
Not listed
Lead sponsor
St. Jude Children's Research Hospital
Other
Eligibility
Not listed
Enrollment
10,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2044
U.S. locations
6
States / cities
Peoria, Illinois • Baton Rouge, Louisiana • Charlotte, North Carolina + 1 more
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
Chronic Myeloproliferative Disorders, Graft Versus Host Disease, Leukemia, Lymphoma, Multiple Myeloma, Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Neoplasms
Interventions
graft-versus-tumor induction therapy, cyclophosphamide, cyclosporine, fludarabine phosphate, mycophenolate mofetil, umbilical cord blood transplantation, radiation therapy
Biological · Drug · Procedure + 1 more
Lead sponsor
University of Rochester
Other
Eligibility
Up to 75 Years
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2003 – 2016
U.S. locations
1
States / cities
Rochester, New York
Conditions
Hematologic Malignancies, Nonmalignant Diseases, Immunodeficiencies, Hemoglobinopathies, Genetic Inborn Errors of Metabolism, Fanconi's Anemia, Thalassemia, Sickle Cell Disease
Interventions
Clofarabine, Fludarabine Injection
Drug
Lead sponsor
University of California, San Francisco
Other
Eligibility
Up to 17 Years
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2020
U.S. locations
1
States / cities
San Francisco, California
Active, not recruiting Not applicable Interventional

Sickle Cell Children's Exercise Study (SuCCESs)

NCT06074198
Conditions
Sickle Cell Disease
Interventions
SuCCESs
Behavioral
Lead sponsor
University of Maryland, Baltimore
Other
Eligibility
6 Years to 17 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2026
U.S. locations
2
States / cities
Baltimore, Maryland
Conditions
Sickle Cell Disease (SCD)
Interventions
ALXN1820
Drug
Lead sponsor
Alexion Pharmaceuticals, Inc.
Industry
Eligibility
18 Years and older
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2024
U.S. locations
2
States / cities
Hollywood, Florida • Indianapolis, Indiana
Conditions
Fanconi Anemia
Interventions
JSP191, CliniMACS Prodigy System, Depleted Stem Cell Transplant, Rabbit Anti-Thymoglobulin (rATG), Cyclophosphamide, Fludarabine, Rituximab
Drug · Device · Biological
Lead sponsor
Porteus, Matthew, MD
Other
Eligibility
2 Years and older
Enrollment
18 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2028
U.S. locations
1
States / cities
Stanford, California
Conditions
Sickle Cell Disease
Interventions
Pociredir
Drug
Lead sponsor
Fulcrum Therapeutics
Industry
Eligibility
18 Years to 65 Years
Enrollment
13 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2026
U.S. locations
6
States / cities
Miami, Florida • Orlando, Florida • Riverdale, Georgia + 3 more
Conditions
Twin to Twin Transfusion Syndrome
Interventions
Curved KARL STORZ fetoscope, Conventional Scopes
Device
Lead sponsor
Jimmy Espinoza
Other
Eligibility
18 Years to 45 Years · Female only
Enrollment
160 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
1
States / cities
Houston, Texas
Conditions
Sickle Cell Disease
Interventions
Hydroxyurea
Drug
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
15 Years to 99 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2018
U.S. locations
1
States / cities
Bethesda, Maryland
Conditions
Sickle Cell Disease
Interventions
GBT021601
Drug
Lead sponsor
Pfizer
Industry
Eligibility
18 Years to 60 Years
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2022
U.S. locations
3
States / cities
Miami, Florida • Atlanta, Georgia
Conditions
Adult Acute Myeloid Leukemia in Remission, Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities, Adult Acute Myeloid Leukemia With Del(5q), Adult Acute Myeloid Leukemia With Inv(16)(p13;q22), Adult Acute Myeloid Leukemia With t(15;17)(q22;q12), Adult Acute Myeloid Leukemia With t(16;16)(p13;q22), Adult Acute Myeloid Leukemia With t(8;21)(q22;q22), Chronic Myelomonocytic Leukemia, de Novo Myelodysplastic Syndromes, Previously Treated Myelodysplastic Syndromes, Recurrent Adult Acute Myeloid Leukemia, Refractory Anemia With Excess Blasts, Refractory Anemia With Excess Blasts in Transformation, Refractory Anemia With Ringed Sideroblasts, Refractory Cytopenia With Multilineage Dysplasia, Secondary Myelodysplastic Syndromes, Untreated Adult Acute Myeloid Leukemia
Interventions
iodine I 131 monoclonal antibody BC8, total-body irradiation, allogeneic hematopoietic stem cell transplantation, peripheral blood stem cell transplantation, fludarabine phosphate, cyclosporine, mycophenolate mofetil, laboratory biomarker analysis
Radiation · Procedure · Drug + 1 more
Lead sponsor
Fred Hutchinson Cancer Center
Other
Eligibility
50 Years and older
Enrollment
79 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1999 – 2010
U.S. locations
1
States / cities
Seattle, Washington
Conditions
Hemoglobinopathy, Sickle Cell Disease
Interventions
Long-Term Follow-up Study of patients who received BEAM-101
Drug
Lead sponsor
Beam Therapeutics Inc.
Industry
Eligibility
14 Years to 37 Years
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2043
U.S. locations
17
States / cities
Birmingham, Alabama • Phoenix, Arizona • Jacksonville, Florida + 13 more
Conditions
Sickle Cell Disease
Interventions
OTQ923
Biological
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
2 Years to 40 Years
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2025
U.S. locations
3
States / cities
Chicago, Illinois • New York, New York • Memphis, Tennessee
Conditions
Sickle Cell Disease, Transfusion Dependent Alpha- or Beta- Thalassemia, Diamond Blackfan Anemia, Paroxysmal Nocturnal Hemoglobinuria, Glanzmann Thrombasthenia, Severe Congenital Neutropenia, Shwachman-Diamond Syndrome, Non-Malignant Hematologic Disorders
Interventions
Reduced Toxicity Ablative Regimen, Reduced Intensity Preparative Regimen, Myeloablative Preparative Regimen
Drug
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
Up to 55 Years
Enrollment
38 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2024
U.S. locations
1
States / cities
Minneapolis, Minnesota
Active, not recruiting Phase 2 Interventional Results available

Eltrombopag for Fanconi Anemia

NCT03206086
Conditions
Fanconi Anemia
Interventions
Eltrombopag
Drug
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
6 Years to 99 Years
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2028
U.S. locations
1
States / cities
Bethesda, Maryland
Conditions
G6PD, G6PD Deficiency
Interventions
G6PD assay
Diagnostic Test
Lead sponsor
Baebies, Inc.
Industry
Eligibility
35 Weeks to 100 Years
Enrollment
119 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2019 – 2020
U.S. locations
3
States / cities
Durham, North Carolina • Raleigh, North Carolina • Cleveland, Ohio
Conditions
Pain
Interventions
SMART app wearable
Other
Lead sponsor
Duke University
Other
Eligibility
8 Years to 80 Years
Enrollment
174 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2024
U.S. locations
1
States / cities
Durham, North Carolina
Conditions
Sickle Cell Disease
Interventions
Alemtuzumab, Total Body Irradiation, Sirolimus
Drug · Radiation
Lead sponsor
Kathleen Dorritie
Other
Eligibility
18 Years and older
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2022
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania