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ClinicalTrials.gov public records Last synced Oct 20, 2026, 8:23 AM EDT

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Showing 1–24 of 1,052 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Anemia, Neonatal Clear all
Completed Phase 1Phase 2 Interventional Results available

Cytoxan, Fludara, and Antithymocyte Globulin Conditioning Followed By Stem Cell Transplant in Treating Fanconi Anemia

NCT00630253
View directory record Official record
Conditions
Fanconi Anemia
Interventions
Anti-Thymocyte Globulin, Cyclophosphamide, Fludarabine, Hematopoietic Stem Cell Transplantation, Methylprednisolone, Filgrastim, Cyclosporine, Mycophenolate Mofetil
Biological · Drug · Procedure
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
Up to 59 Years
Enrollment
31 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2000 – 2020
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Oct 12, 2021 · Synced Oct 20, 2026, 8:23 AM EDT
Not yet recruiting Phase 1 Interventional

High-Dose Vitamin C in G6PDA and Pyruvate Kinase Deficiency: A Safety Study

NCT07612345
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated May 28, 2026 · Synced Oct 20, 2026, 8:23 AM EDT
Recruiting Phase 2 Interventional

Cognitive Remediation Intervention to Prepare for Transition of Care

NCT05336201
View directory record Official record
Conditions
Sickle Cell Disease, Cognitive Impairment, Adolescent Behavior, Self Efficacy, Health-Related Behavior, Coping Skills
Interventions
CREADY (Cognitive-Remediation of Executive and Adaptive Deficits in Youth)
Behavioral
Lead sponsor
University of Alabama at Birmingham
Other
Eligibility
10 Years to 18 Years
Enrollment
120 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2027
U.S. locations
1
States / cities
Birmingham, Alabama
Source: ClinicalTrials.gov public record
Updated Feb 27, 2026 · Synced Oct 20, 2026, 8:23 AM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

A Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of AG-946 in Healthy Volunteers and in Participants With Sickle Cell Disease

NCT04536792
View directory record Official record
Conditions
Healthy Volunteers, Anemia, Sickle Cell
Interventions
AG-946, AG-946 Matched Placebo
Drug
Lead sponsor
Agios Pharmaceuticals, Inc.
Industry
Eligibility
18 Years to 70 Years
Enrollment
122 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2020 – 2023
U.S. locations
8
States / cities
La Jolla, California • Boston, Massachusetts • New York, New York + 4 more
Source: ClinicalTrials.gov public record
Updated Apr 16, 2024 · Synced Oct 20, 2026, 8:23 AM EDT
Completed Phase 2 Interventional

Non-Ablative Allo HSCT For Hematologic Malignancies or SAA

NCT00006379
View directory record Official record
Conditions
Chronic Myeloproliferative Disorders, Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm, Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Diseases, Precancerous/Nonmalignant Condition, Small Intestine Cancer
Interventions
anti-thymocyte globulin, graft-versus-tumor induction therapy, cyclophosphamide, fludarabine phosphate, peripheral blood stem cell transplantation
Biological · Drug · Procedure
Lead sponsor
Case Comprehensive Cancer Center
Other
Eligibility
Up to 70 Years
Enrollment
58 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2000 – 2011
U.S. locations
1
States / cities
Cleveland, Ohio
Source: ClinicalTrials.gov public record
Updated Dec 8, 2011 · Synced Oct 20, 2026, 8:23 AM EDT
Completed Phase 2 Interventional

Donor Bone Marrow Transplant in Treating Patients With Leukemia, Lymphoma, or Nonmalignant Hematologic Disorders

NCT00005622
View directory record Official record
Conditions
Chronic Myeloproliferative Disorders, Leukemia, Multiple Myeloma and Malignant Plasma Cell Neoplasms, Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Neoplasms
Interventions
cyclophosphamide, TBI
Drug · Radiation
Lead sponsor
H. Lee Moffitt Cancer Center and Research Institute
Other
Eligibility
15 Years to 50 Years
Enrollment
72 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1996 – 2009
U.S. locations
1
States / cities
Tampa, Florida
Source: ClinicalTrials.gov public record
Updated Oct 25, 2012 · Synced Oct 20, 2026, 8:23 AM EDT
Terminated Phase 1 Interventional

Dronabinol for Pain and Inflammation in Adults Living With Sickle Cell Disease

NCT03978156
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
Dronabinol 2.5 MG, Microcrystalline cellulose
Drug
Lead sponsor
Yale University
Other
Eligibility
18 Years and older
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2021
U.S. locations
1
States / cities
New Haven, Connecticut
Source: ClinicalTrials.gov public record
Updated Jan 26, 2021 · Synced Oct 20, 2026, 8:23 AM EDT
Recruiting Phase 2 Interventional

Minimizing Toxicity in HLA-identical Sibling Donor Transplantation for Children With Sickle Cell Disease

NCT03587272
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
Alemtuzumab, low dose total body irradiation, Sirolimus
Drug
Lead sponsor
Robert Nickel
Other
Eligibility
2 Years to 25 Years
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2030
U.S. locations
5
States / cities
Washington D.C., District of Columbia • Chicago, Illinois • New York, New York + 2 more
Source: ClinicalTrials.gov public record
Updated Mar 10, 2026 · Synced Oct 20, 2026, 8:23 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Hydoxyurea Exposure in Lactation A Pharmacokinetics Study (HELPS)

NCT02990598
View directory record Official record
Conditions
Sickle Cell Anemia
Interventions
Not listed
Lead sponsor
Children's Hospital Medical Center, Cincinnati
Other
Eligibility
18 Years to 99 Years · Female only
Enrollment
16 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2017 – 2018
U.S. locations
1
States / cities
Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Aug 26, 2019 · Synced Oct 20, 2026, 8:23 AM EDT
Completed Phase 2 Interventional Results available

Treatment of Sickle Cell Patients Hospitalized in Pain Crisis With Prophylactic Dose Low-molecular-weight Heparin (LMWH) Versus Placebo

NCT01419977
View directory record Official record
Conditions
Sickle Cell Disease, Vaso-occlusive Crisis
Interventions
Placebo, Dalteparin
Drug
Lead sponsor
Duke University
Other
Eligibility
18 Years and older
Enrollment
34 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2014
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Feb 16, 2015 · Synced Oct 20, 2026, 8:23 AM EDT
Recruiting No phase listed Observational

Study to Understand the Genetic Risk of Developing an Immune Response After Blood Transfusions Among Individuals With Sickle Cell Disease

NCT06944067
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
Not listed
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
2 Years to 99 Years
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2030
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Sep 11, 2026 · Synced Oct 20, 2026, 8:23 AM EDT
Withdrawn Phase 4 Interventional

Assessment of Opioid Analgesia in Sickle Cell

NCT00513864
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
Dextromethorphan, Codeine, Morphine
Drug
Lead sponsor
Children's National Research Institute
Other
Eligibility
7 Years to 18 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2012
U.S. locations
1
States / cities
Washington D.C., District of Columbia
Source: ClinicalTrials.gov public record
Updated Dec 23, 2014 · Synced Oct 20, 2026, 8:23 AM EDT
Completed Phase 1 Interventional

Gene Therapy for the Treatment of Fanconi's Anemia Type C

NCT00001399
View directory record Official record
Conditions
Fanconi's Anemia, Pancytopenia
Interventions
Transduced CD34+ Cells
Drug
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
5 Years and older
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1993 – 2009
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 2, 2017 · Synced Oct 20, 2026, 8:23 AM EDT
Completed No phase listed Observational

Improving Sickle Cell Disease (SCD) Care Using Web-based Guidelines

NCT03037021
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
Not listed
Lead sponsor
Duke University
Other
Eligibility
15 Years to 45 Years
Enrollment
213 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Nov 1, 2023 · Synced Oct 20, 2026, 8:23 AM EDT
Terminated Phase 1Phase 2 Interventional Results available

Safety and Efficacy Study of Sotatercept in Adults With Transfusion Dependent Diamond Blackfan Anemia

NCT01464164
View directory record Official record
Conditions
Diamond Blackfan Anemia
Interventions
Sotatercept, Sotatercept with prednisone boost
Drug
Lead sponsor
Northwell Health
Other
Eligibility
18 Years and older
Enrollment
19 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2021
U.S. locations
1
States / cities
Manhasset, New York
Source: ClinicalTrials.gov public record
Updated Mar 17, 2023 · Synced Oct 20, 2026, 8:23 AM EDT
Completed Phase 3 Interventional Results available

An Efficacy and Safety Study of Luspatercept (ACE-536) Versus Placebo in Adults Who Require Regular Red Blood Cell Transfusions Due to Beta (β) Thalassemia

NCT02604433
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Apr 18, 2023 · Synced Oct 20, 2026, 8:23 AM EDT
Completed Not applicable Interventional

The Feasibility of the Pray Until Something Happens (PUSH) Intervention-OUTPATIENT

NCT06580067
View directory record Official record
Conditions
Sickle Cell Disease, Stress, Pain
Interventions
Pray Until Something Happens (PUSH), Self-monitoring
Behavioral
Lead sponsor
University of Florida
Other
Eligibility
18 Years to 99 Years
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025
U.S. locations
1
States / cities
Gainesville, Florida
Source: ClinicalTrials.gov public record
Updated Mar 30, 2026 · Synced Oct 20, 2026, 8:23 AM EDT
Terminated Phase 1 Interventional

A Study to Investigate the Safety, Tolerability, and Pharmacokinetics of Oral GSK4172239D Compared With Placebo in Sickle Cell Disease Participants Aged 18 to 50 Years

NCT05660265
View directory record Official record
Conditions
Hematologic Diseases, Anaemia, Sickle Cell
Interventions
GSK4172239D, Placebo
Drug · Other
Lead sponsor
GlaxoSmithKline
Industry
Eligibility
18 Years to 50 Years
Enrollment
11 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2025
U.S. locations
6
States / cities
Miami, Florida • Tamarac, Florida • Atlanta, Georgia + 3 more
Source: ClinicalTrials.gov public record
Updated Jan 22, 2026 · Synced Oct 20, 2026, 8:23 AM EDT
Completed Phase 1 Interventional

Combination Chemotherapy Followed By Umbilical Cord Blood Transplantation in Treating Patients With Hematologic Cancer or Severe Aplastic Anemia

NCT00054236
View directory record Official record
Conditions
Chronic Myeloproliferative Disorders, Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm, Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Diseases
Interventions
anti-thymocyte globulin, filgrastim, cyclophosphamide, fludarabine phosphate, umbilical cord blood transplantation, methylprednisolone
Biological · Drug · Procedure
Lead sponsor
Case Comprehensive Cancer Center
Other
Eligibility
Up to 120 Years
Enrollment
55 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2002 – 2011
U.S. locations
1
States / cities
Cleveland, Ohio
Source: ClinicalTrials.gov public record
Updated Jul 27, 2020 · Synced Oct 20, 2026, 8:23 AM EDT
Completed Phase 1Phase 2 Interventional Results available

Combination Deferasirox and Deferiprone for Severe Iron Overload in Thalassemia

NCT01709032
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Mar 6, 2019 · Synced Oct 20, 2026, 8:23 AM EDT
Completed Phase 2 Interventional

Study of GMI-1070 for the Treatment of Sickle Cell Pain Crisis

NCT01119833
View directory record Official record
Conditions
Sickle Cell Disease, Vaso-occlusive Crisis, Pain Crisis
Interventions
GMI-1070, Placebo
Drug
Lead sponsor
GlycoMimetics Incorporated
Industry
Eligibility
12 Years to 60 Years
Enrollment
81 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2013
U.S. locations
21
States / cities
Birmingham, Alabama • Berkeley, California • Sacramento, California + 18 more
Source: ClinicalTrials.gov public record
Updated May 13, 2020 · Synced Oct 20, 2026, 8:23 AM EDT
Completed Phase 1 Interventional

Alemtuzumab and Combination Chemotherapy Followed By Donor Lymphocytes in Treating Patients Who Are Undergoing Donor Stem Cell Transplant for Hematologic Cancer

NCT00104975
View directory record Official record
Conditions
Chronic Myeloproliferative Disorders, Leukemia, Lymphoma, Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Neoplasms
Interventions
alemtuzumab, therapeutic allogeneic lymphocytes, fludarabine phosphate, melphalan, tacrolimus, thiotepa, peripheral blood stem cell transplantation
Biological · Drug · Procedure
Lead sponsor
Yale University
Other
Eligibility
18 Years to 55 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2008
U.S. locations
1
States / cities
New Haven, Connecticut
Source: ClinicalTrials.gov public record
Updated Dec 15, 2016 · Synced Oct 20, 2026, 8:23 AM EDT
Completed Phase 1 Interventional Results available

Reduced Intensity Conditioning for Umbilical Cord Blood Transplant in Pediatric Patients With Non-Malignant Disorders

NCT00744692
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Aug 13, 2014 · Synced Oct 20, 2026, 8:23 AM EDT
Completed Phase 2 Interventional

5-Azacytidine and Phenylbutyrate to Treat Severe Thalassemia

NCT00005934
View directory record Official record
Conditions
Beta Thalassemia
Interventions
5-Azacytidine
Drug
Lead sponsor
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
NIH
Eligibility
Not listed
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2000 – 2003
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 4, 2008 · Synced Oct 20, 2026, 8:23 AM EDT