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ClinicalTrials.gov public records Last synced Oct 12, 2026, 10:36 PM EDT

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Showing 169–192 of 1,044 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Congenital Anemias Clear all
Recruiting Phase 4 Interventional

Clinical and Biomarker Effects of Depot Medroxyprogesterone Acetate in Females With Sickle Cell Disease

NCT06665997
View directory record Official record
Conditions
Sickle Cell Disease (SCD), Vaso-Occlusive Pain Episode in Sickle Cell Disease
Interventions
Depot medroxyprogesterone acetate (DMPA)
Drug
Lead sponsor
University of Pennsylvania
Other
Eligibility
18 Years to 50 Years · Female only
Enrollment
65 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2028
U.S. locations
2
States / cities
Atlanta, Georgia • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated May 28, 2026 · Synced Oct 12, 2026, 10:36 PM EDT
Completed Phase 1 Interventional

Evaluation of Hydroxyurea Plus L-arginine or Sildenafil to Treat Sickle Cell Anemia

NCT00056433
View directory record Official record
Conditions
Sickle Cell Anemia
Interventions
Hydroxyurea, L-Arginine, Sildenafil
Drug
Lead sponsor
National Institutes of Health Clinical Center (CC)
NIH
Eligibility
16 Years and older
Enrollment
39 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2003 – 2006
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Nov 22, 2019 · Synced Oct 12, 2026, 10:36 PM EDT
Completed Phase 1 Interventional

XK469R in Treating Patients With Refractory Hematologic Cancer

NCT00095797
View directory record Official record
Conditions
Adult Acute Basophilic Leukemia, Adult Acute Eosinophilic Leukemia, Adult Acute Megakaryoblastic Leukemia (M7), Adult Acute Minimally Differentiated Myeloid Leukemia (M0), Adult Acute Monoblastic Leukemia (M5a), Adult Acute Monocytic Leukemia (M5b), Adult Acute Myeloblastic Leukemia With Maturation (M2), Adult Acute Myeloblastic Leukemia Without Maturation (M1), Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities, Adult Acute Myeloid Leukemia With Inv(16)(p13;q22), Adult Acute Myeloid Leukemia With t(16;16)(p13;q22), Adult Acute Myeloid Leukemia With t(8;21)(q22;q22), Adult Acute Myelomonocytic Leukemia (M4), Adult Erythroleukemia (M6a), Adult Pure Erythroid Leukemia (M6b), Blastic Phase Chronic Myelogenous Leukemia, Chronic Myelomonocytic Leukemia, de Novo Myelodysplastic Syndromes, Previously Treated Myelodysplastic Syndromes, Recurrent Adult Acute Lymphoblastic Leukemia, Recurrent Adult Acute Myeloid Leukemia, Refractory Anemia With Excess Blasts, Refractory Anemia With Excess Blasts in Transformation, Refractory Chronic Lymphocytic Leukemia, Relapsing Chronic Myelogenous Leukemia, Secondary Myelodysplastic Syndromes, Stage III Chronic Lymphocytic Leukemia, Stage IV Chronic Lymphocytic Leukemia, Untreated Adult Acute Myeloid Leukemia
Interventions
R(+)XK469, pharmacological study, laboratory biomarker analysis
Drug · Other
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
18 Years and older
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2004
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Feb 8, 2013 · Synced Oct 12, 2026, 10:36 PM EDT
Recruiting No phase listed Observational

Tissue, Blood, and Body Fluid Sample Collection From Patients With Hematologic Cancer

NCT01137643
View directory record Official record
Conditions
Chronic Myeloproliferative Disorders, Leukemia, Lymphoma, Lymphoproliferative Disorder, Multiple Myeloma and Plasma Cell Neoplasm, Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Neoplasms, Nonmalignant Neoplasm
Interventions
biologic sample preservation procedure, cytology specimen collection procedure
Other
Lead sponsor
UNC Lineberger Comprehensive Cancer Center
Other
Eligibility
18 Years to 120 Years
Enrollment
15,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2040
U.S. locations
1
States / cities
Chapel Hill, North Carolina
Source: ClinicalTrials.gov public record
Updated Oct 27, 2025 · Synced Oct 12, 2026, 10:36 PM EDT
Terminated Phase 2 Interventional

Effects of HQK-1001 in Patients With Sickle Cell Disease

NCT01601340
View directory record Official record
Conditions
Sickle Cell Disease, Sickle Cell Anemia, Sickle Cell Disorders, Hemoglobin S Disease, Sickling Disorder Due to Hemoglobin S
Interventions
HQK-1001, Placebo
Drug
Lead sponsor
HemaQuest Pharmaceuticals Inc.
Industry
Eligibility
12 Years to 60 Years
Enrollment
77 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2013
U.S. locations
11
States / cities
Mobile, Alabama • Oakland, California • Washington D.C., District of Columbia + 7 more
Source: ClinicalTrials.gov public record
Updated Mar 18, 2015 · Synced Oct 12, 2026, 10:36 PM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Sickle Cell Kidney Biorepository

NCT07064174
View directory record Official record
Conditions
Sickle Cell Trait, Sickle Cell Disease (SCD), Chronic Kidney Disease
Interventions
Not listed
Lead sponsor
University of Texas Southwestern Medical Center
Other
Eligibility
18 Years to 65 Years
Enrollment
800 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2024 – 2049
U.S. locations
2
States / cities
Dallas, Texas
Source: ClinicalTrials.gov public record
Updated Apr 13, 2026 · Synced Oct 12, 2026, 10:36 PM EDT
Completed Phase 2 Interventional

Hydroxyurea for the Treatment of Patients With Sickle Cell Anemia

NCT00001197
View directory record Official record
Conditions
Sickle Cell Anemia
Interventions
Hydroxyurea
Drug
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
18 Years and older
Enrollment
41 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1984 – 2015
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Dec 16, 2019 · Synced Oct 12, 2026, 10:36 PM EDT
Active, not recruiting Phase 1Phase 2 Interventional Accepts healthy volunteers Results available

Improving the Results of Bone Marrow Transplantation for Patients With Severe Congenital Anemias

NCT00061568
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Sep 9, 2026 · Synced Oct 12, 2026, 10:36 PM EDT
Terminated Phase 3 Interventional

A Stratified Sickle Event Randomized Trial (ASSERT)

NCT00102791
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
ICA-17043
Drug
Lead sponsor
Icagen
Industry
Eligibility
16 Years to 65 Years
Enrollment
297 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2007
U.S. locations
42
States / cities
Mobile, Alabama • Little Rock, Arkansas • Davis, California + 39 more
Source: ClinicalTrials.gov public record
Updated Mar 27, 2008 · Synced Oct 12, 2026, 10:36 PM EDT
Terminated Phase 2 Interventional Results available

Low-Dose Ketamine Infusion for Children With Sickle Cell Disease-Related Pain

NCT00595530
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
ketamine
Drug
Lead sponsor
Connecticut Children's Medical Center
Other
Eligibility
7 Years and older
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2010
U.S. locations
2
States / cities
Farmington, Connecticut • Hartford, Connecticut
Source: ClinicalTrials.gov public record
Updated Aug 21, 2019 · Synced Oct 12, 2026, 10:36 PM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

The Congenital Dyserythropoietic Anemia Registry (CDAR)

NCT02964494
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Jun 16, 2026 · Synced Oct 12, 2026, 10:36 PM EDT
Completed Phase 2 Interventional

Study of the Effect of Etavopivat on Cerebral Hemodynamic Response in Children With Sickle Cell Disease

NCT05725902
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
Etavopivat
Drug
Lead sponsor
Novo Nordisk A/S
Industry
Eligibility
12 Years to 21 Years
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
1
States / cities
Atlanta, Georgia
Source: ClinicalTrials.gov public record
Updated Apr 8, 2026 · Synced Oct 12, 2026, 10:36 PM EDT
Completed No phase listed Observational

Analysis of T-Cell Immune Reconstitution Following Allogeneic Hematopoietic BMT for Severe SCD

NCT00228631
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
Not listed
Lead sponsor
Emory University
Other
Eligibility
6 Months to 21 Years
Enrollment
7 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2012
U.S. locations
1
States / cities
Atlanta, Georgia
Source: ClinicalTrials.gov public record
Updated May 26, 2014 · Synced Oct 12, 2026, 10:36 PM EDT
Completed Phase 1 Interventional

Radiolabeled Monoclonal Antibody Therapy, Fludarabine Phosphate, and Low-Dose Total-Body Irradiation Followed by Donor Stem Cell Transplant and Immunosuppression Therapy in Treating Older Patients With Advanced Acute Myeloid Leukemia or High-Risk Myelodysplastic Syndromes

NCT00008177
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Nov 13, 2019 · Synced Oct 12, 2026, 10:36 PM EDT
Terminated Phase 1 Interventional

CSL200 Gene Therapy in Adults With Severe Sickle Cell Disease

NCT04091737
View directory record Official record
Conditions
Anemia, Sickle Cell
Interventions
Autologous enriched CD34+ cell fraction that contains CD34+ cells transduced with lentiviral vector encoding human γ-globinG16D and short-hairpin RNA734
Biological
Lead sponsor
CSL Behring
Industry
Eligibility
18 Years to 45 Years
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2021
U.S. locations
1
States / cities
Duarte, California
Source: ClinicalTrials.gov public record
Updated Jun 18, 2021 · Synced Oct 12, 2026, 10:36 PM EDT
Completed Not applicable Interventional Results available

Treatment for Relapsed/Refractory AML Based on a High Throughput Drug Sensitivity Assay

NCT01872819
View directory record Official record
Conditions
Adult Acute Megakaryoblastic Leukemia (M7), Adult Acute Minimally Differentiated Myeloid Leukemia (M0), Adult Acute Monoblastic Leukemia (M5a), Adult Acute Monocytic Leukemia (M5b), Adult Acute Myeloblastic Leukemia With Maturation (M2), Adult Acute Myeloblastic Leukemia Without Maturation (M1), 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(16;16)(p13;q22), Adult Acute Myeloid Leukemia With t(8;21)(q22;q22), Adult Acute Myelomonocytic Leukemia (M4), Adult Erythroleukemia (M6a), Adult Pure Erythroid Leukemia (M6b), Chronic Myelomonocytic Leukemia, Myelodysplastic/Myeloproliferative Neoplasm, Unclassifiable, Previously Treated Myelodysplastic Syndromes, Recurrent Adult Acute Myeloid Leukemia, Refractory Anemia With Excess Blasts
Interventions
antitumor drug screening assay, chemotherapy, biological therapy
Other · Drug · Biological
Lead sponsor
University of Washington
Other
Eligibility
18 Years and older
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2014
U.S. locations
1
States / cities
Seattle, Washington
Source: ClinicalTrials.gov public record
Updated Jul 11, 2018 · Synced Oct 12, 2026, 10:36 PM EDT
Completed No phase listed Observational

Obesity in Pediatric Sickle Cell Disease: A New Phenomenon

NCT04676113
View directory record Official record
Conditions
Sickle Cell Disease, Obesity, Childhood, Hypertension, Hyperlipidemia in Children, Hyperglycemia
Interventions
Not listed
Lead sponsor
University of Mississippi Medical Center
Other
Eligibility
10 Years to 19 Years
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2022
U.S. locations
1
States / cities
Jackson, Mississippi
Source: ClinicalTrials.gov public record
Updated May 12, 2023 · Synced Oct 12, 2026, 10:36 PM EDT
Terminated Phase 1 Interventional Accepts healthy volunteers

Use of Rft5-Dga to Deplete Alloreactive Cells for Pts With Fanconi Anemia After Haploidentical SCT

NCT00586274
View directory record Official record
Conditions
FANCONI ANEMIA
Interventions
CD34 selected haploidentical PBSCT, Fludarabine, T cell infusion, Campath 1h, anti-CD45
Procedure · Drug · Biological
Lead sponsor
Baylor College of Medicine
Other
Eligibility
Up to 64 Years
Enrollment
1 participant
Healthy volunteers
Accepts healthy volunteers
Timeline
2002 – 2009
U.S. locations
2
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Mar 4, 2014 · Synced Oct 12, 2026, 10:36 PM EDT
Completed Not applicable Interventional

Blood Cell Collection for Future Use in Individuals With Fanconi Anemia

NCT00271089
View directory record Official record
Conditions
Fanconi Anemia
Interventions
PBSC Collection, Bone Marrow Harvest, CliniMacs Cell Selection System
Procedure · Device
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
1 Year to 35 Years
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2007
U.S. locations
1
States / cities
Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Jul 12, 2016 · Synced Oct 12, 2026, 10:36 PM EDT
Completed Phase 1 Interventional

Bone Marrow Transplant With Abatacept for Non-Malignant Diseases

NCT01917708
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Dec 26, 2019 · Synced Oct 12, 2026, 10:36 PM EDT
Completed Phase 2 Interventional Results available

Daily Vitamin D for Sickle-cell Respiratory Complications

NCT04170348
View directory record Official record
Conditions
Sickle Cell Disease, Anemia, Sickle Cell, Anemia, Hemolytic, Congenital, Respiratory Tract Diseases, Respiration Disorders, Acute Chest Syndrome, Lung Diseases, Asthma, Respiratory Tract Infections, Nutrition Disorders, Deficiency Diseases Vitamin, Vitamin D Deficiency
Interventions
Daily oral vitamin D3, 3,333 IU, Monthly oral vitamin D3, 100,000 IU, Placebo oral tablet
Drug
Lead sponsor
Columbia University
Other
Eligibility
3 Years to 20 Years
Enrollment
58 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2024
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Sep 24, 2025 · Synced Oct 12, 2026, 10:36 PM EDT
Not listed Phase 2 Interventional Accepts healthy volunteers

Outcomes Mandate National Integration With Cannabis as Medicine

NCT03944447
View directory record Official record
Conditions
Chronic Pain, Chronic Pain Syndrome, Chronic Pain Due to Injury, Chronic Pain Due to Trauma, Fibromyalgia, Seizures, Hepatitis C, Cancer, Crohn Disease, HIV/AIDS, Multiple Sclerosis, Traumatic Brain Injury, Sickle Cell Disease, Post Traumatic Stress Disorder, Tourette Syndrome, Ulcerative Colitis, Glaucoma, Epilepsy, Inflammatory Bowel Diseases, Parkinson Disease, Amyotrophic Lateral Sclerosis, Chronic Traumatic Encephalopathy, Anxiety, Depression, Insomnia, Autism, Opioid-use Disorder, Bipolar Disorder, Covid19, SARS-CoV Infection, COVID-19, Corona Virus Infection, Coronavirus
Interventions
Cannabis, Medical, RYAH-Medtech Inhaler
Drug · Device
Lead sponsor
OMNI Medical Services, LLC
Network
Eligibility
7 Years and older
Enrollment
200,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2018 – 2025
U.S. locations
17
States / cities
Boca Raton, Florida • Bradenton, Florida • Fort Lauderdale, Florida + 13 more
Source: ClinicalTrials.gov public record
Updated Jun 2, 2022 · Synced Oct 12, 2026, 10:36 PM EDT
Withdrawn Phase 1Phase 2 Interventional

Phase I/II Pilot Study of Mixed Chimerism to Treat Hemoglobinopathies

NCT01419704
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Mar 3, 2023 · Synced Oct 12, 2026, 10:36 PM EDT
Terminated Phase 2 Interventional Results available

Erythropoietin to Prevent Unnecessary Transfusions In Patients With Cyanotic CHD - A Prospective Control Trial

NCT02564796
View directory record Official record
Conditions
Cyanotic Congenital Heart Disease, Anemia, Cyanosis, Congenital Heart Disease
Interventions
Epoetin Alfa and Iron, Iron
Drug
Lead sponsor
University of California, San Diego
Other
Eligibility
Up to 8 Weeks
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2020
U.S. locations
1
States / cities
San Diego, California
Source: ClinicalTrials.gov public record
Updated Dec 2, 2021 · Synced Oct 12, 2026, 10:36 PM EDT