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ClinicalTrials.gov public records Last synced Oct 15, 2026, 7:08 PM EDT

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Showing 625–648 of 1,045 matching trials from the live ClinicalTrials.gov search.
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Recruiting Phase 2 Interventional

Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT

NCT01962415
View directory record Official record
Conditions
Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes, Inherited Metabolic Disorders (IMD), Hereditary Anemias, Inflammatory Conditions, Systemic Juvenile Idiopathic Arthritis (sJIA), Juvenile Rheumatoid Arthritis (JRA)
Interventions
Hydroxyurea, Alemtuzumab, Fludarabine, Melphalan, Thiotepa
Drug
Lead sponsor
Paul Szabolcs
Other
Eligibility
2 Months to 55 Years
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2027
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Dec 15, 2025 · Synced Oct 15, 2026, 7:08 PM EDT
Recruiting Phase 1Phase 2 Interventional

Arginine Therapy for the Treatment of Pain in Children With Sickle Cell Disease

NCT02447874
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
Arginine, Arginine (Loading), Arginine (Continuous)
Drug
Lead sponsor
Emory University
Other
Eligibility
7 Years to 21 Years
Enrollment
21 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2028
U.S. locations
2
States / cities
Atlanta, Georgia
Source: ClinicalTrials.gov public record
Updated Jul 29, 2026 · Synced Oct 15, 2026, 7:08 PM EDT
Recruiting Phase 2 Interventional

Safety, Efficacy, and Pharmacokinetics of CSL889 in Adults and Adolescents With Sickle Cell Disease During Vaso-Occlusive Crisis

NCT06699849
View directory record Official record
Conditions
Sickle Cell Disease Vaso-occlusive Crisis
Interventions
CSL889, Placebo
Biological · Drug
Lead sponsor
CSL Behring
Industry
Eligibility
12 Years and older
Enrollment
70 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
20
States / cities
Oakland, California • Orange, California • Washington D.C., District of Columbia + 15 more
Source: ClinicalTrials.gov public record
Updated Oct 8, 2026 · Synced Oct 15, 2026, 7:08 PM EDT
Completed Phase 2 Interventional

Treosulfan-Based Conditioning Regimen Before a Blood or Bone Marrow Transplant for the Treatment of Bone Marrow Failure Diseases (BMT CTN 1904)

NCT04965597
View directory record Official record
Conditions
Bone Marrow Failure Syndrome, Congenital Amegakaryocytic Thrombocytopenia, Diamond-Blackfan Anemia, Hereditary Sideroblastic Anemia, Paroxysmal Nocturnal Hemoglobinuria, Shwachman-Diamond Syndrome, Hematologic Neoplasm With Germline GATA2 Mutation, Hematologic Neoplasm With Germline SAMD9 Mutation, Hematologic Neoplasm With Germline SAMD9L Mutation
Interventions
Treosulfan, Fludarabine Phosphate, Tacrolimus, Methotrexate, Lapine T-Lymphocyte Immune Globulin, Peripheral Blood Stem Cell Transplantation, Allogeneic Bone Marrow Transplantation, Quality-of-Life Assessment, Echocardiography, Multigated Acquisition Scan, Bone Marrow Biopsy, Bone Marrow Aspiration, Biospecimen Collection, X-Ray Imaging, Computed Tomography
Drug · Biological · Procedure + 1 more
Lead sponsor
Fred Hutchinson Cancer Center
Other
Eligibility
1 Year to 49 Years
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2026
U.S. locations
24
States / cities
Los Angeles, California • San Diego, California • San Francisco, California + 20 more
Source: ClinicalTrials.gov public record
Updated Mar 9, 2026 · Synced Oct 15, 2026, 7:08 PM EDT
Recruiting Phase 3 Interventional

A Study to Investigate the Effect of Mitapivat on Transfusion Burden in Subjects With Sickle Cell Disease (SCD)

NCT07656415
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
Mitapivat Matched Placebo, Mitapivat
Drug
Lead sponsor
Agios Pharmaceuticals, Inc.
Industry
Eligibility
12 Years and older
Enrollment
159 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2030
U.S. locations
3
States / cities
Indianapolis, Indiana • Grand Rapids, Michigan • Flowood, Mississippi
Source: ClinicalTrials.gov public record
Updated Sep 28, 2026 · Synced Oct 15, 2026, 7:08 PM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

Metabolism and Pharmacokinetics of Primaquine Enantiomers in Human Volunteers Receiving a Seven Day Dose Regimen

NCT03934450
View directory record Official record
Conditions
Malaria, Glucose 6 Phosphate Dehydrogenase Deficiency
Interventions
RPQ, SPQ, Primaquine Phosphate, Placebo
Drug
Lead sponsor
University of Mississippi, Oxford
Other
Eligibility
18 Years to 65 Years
Enrollment
36 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2018 – 2019
U.S. locations
1
States / cities
University, Mississippi
Source: ClinicalTrials.gov public record
Updated May 14, 2019 · Synced Oct 15, 2026, 7:08 PM EDT
Completed Not applicable Interventional Results available

Evaluation of the AMICUS RBCx System in Sickle Cell Patients

NCT02372877
View directory record Official record
Conditions
Sickle Cell Anemia
Interventions
Amicus Red Cell Exchange in SCD patients
Device
Lead sponsor
Fenwal, Inc.
Industry
Eligibility
6 Years and older
Enrollment
83 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2018
U.S. locations
6
States / cities
Phoenix, Arizona • Detroit, Michigan • St Louis, Missouri + 3 more
Source: ClinicalTrials.gov public record
Updated Jul 29, 2022 · Synced Oct 15, 2026, 7:08 PM EDT
Terminated Phase 2 Interventional

Bone Marrow Transplant From Related Donor for Patients With High Risk Hemoglobinopathies

NCT00040469
View directory record Official record
Conditions
Sickle Cell Anemia, Hemoglobinopathy, Thalassemia
Interventions
Campath -1H, Dilantin, Busulfan, Cyclophosphamide
Drug
Lead sponsor
Baylor College of Medicine
Other
Eligibility
1 Day to 64 Years
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2000 – 2003
U.S. locations
2
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Jan 18, 2020 · Synced Oct 15, 2026, 7:08 PM EDT
Terminated Phase 2 Interventional

Haploidentical Allogeneic Hematopoietic Stem Cell Transplantation (HaploHCT) Following Reduced Intensity Conditioning (RIC) for Selected High Risk Non-Malignant Diseases

NCT03367546
View directory record Official record
Conditions
Sickle Cell Disease, Thalassemia, High Risk Hematologic Disorders, Cerebral Adrenoleukodystrophy, Inherited Metabolic Disorders
Interventions
Blood and Marrow Transplant
Procedure
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
0 Years to 25 Years
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2022
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Jul 12, 2024 · Synced Oct 15, 2026, 7:08 PM EDT
Terminated Phase 2 Interventional Results available

A Study of the Effect of Blood Stem Cell Transplant After Chemotherapy Alone in Patients With Fanconi Anemia

NCT03600909
View directory record Official record
Conditions
Fanconi Anemia, Myelodysplastic Syndrome (MDS), Acute Myelogenous Leukemia (AML)
Interventions
Busulfan, Fludarabine, Cyclophosphamide, Anti-Thymocyte Globulin (Rabbit), The CliniMACS device, G-CSF
Drug · Device
Lead sponsor
Memorial Sloan Kettering Cancer Center
Other
Eligibility
1 Month and older
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2021
U.S. locations
3
States / cities
New York, New York • Cincinnati, Ohio • Seattle, Washington
Source: ClinicalTrials.gov public record
Updated Apr 12, 2022 · Synced Oct 15, 2026, 7:08 PM EDT
Recruiting Phase 1Phase 2 Interventional

Depleted Donor Stem Cell Transplant in Children and Adults With Fanconi Anemia After Being Conditioned With a Regimen Containing Briquilimab

NCT04784052
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Jan 27, 2026 · Synced Oct 15, 2026, 7:08 PM EDT
Completed Not applicable Interventional

Prevention of Graft-Versus-Host Disease in Patients Undergoing Bone Marrow Transplantation

NCT00003538
View directory record Official record
Conditions
Cancer
Interventions
graft versus host disease prophylaxis/therapy, allogeneic bone marrow transplantation
Biological · Procedure
Lead sponsor
Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
Other
Eligibility
Up to 65 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
1997 – 2004
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated May 2, 2014 · Synced Oct 15, 2026, 7:08 PM EDT
Recruiting Not applicable Interventional

Virtual Reality Devices as an Adjunct to Usual Care for Patients With Sickle Cell Disease Experiencing Vaso-Occlusive Crises

NCT06184126
View directory record Official record
Conditions
Sickle Cell Disease, Vaso-occlusive Pain Episode in Sickle Cell Disease, Acute Pain
Interventions
Virtual Reality Headset and Hand-Held Controllers, Blindfolding and Noise Cancelling
Device · Other
Lead sponsor
University of Maryland, Baltimore
Other
Eligibility
18 Years and older
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Dec 24, 2025 · Synced Oct 15, 2026, 7:08 PM EDT
Recruiting Phase 1Phase 2 Interventional Accepts healthy volunteers

A Study to Evaluate BMS-986470 in Healthy Volunteers and Participants With Sickle Cell Disease

NCT06481306
View directory record Official record
Conditions
Anemia, Sickle Cell, Healthy Volunteers
Interventions
BMS-986470, Placebo, Famotidine, Pantoprazole
Drug
Lead sponsor
Bristol-Myers Squibb
Industry
Eligibility
18 Years and older
Enrollment
224 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2024 – 2027
U.S. locations
19
States / cities
Birmingham, Alabama • La Jolla, California • Oakland, California + 14 more
Source: ClinicalTrials.gov public record
Updated Aug 18, 2026 · Synced Oct 15, 2026, 7:08 PM EDT
Terminated Phase 2 Interventional

A Study With LJPC-401 for the Treatment of Myocardial Iron Overload in Patients With Transfusion-Dependent Beta Thalassemia

NCT03381833
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Jul 29, 2021 · Synced Oct 15, 2026, 7:08 PM EDT
Recruiting Phase 4 Interventional

Effectiveness of Nontraditional Hydroxyurea Algorithms: Novel and Clinical Evaluations (ENHANCE)

NCT07177300
View directory record Official record
Conditions
Sickle Cell Anemia (HbSS), Sickle-β0-thalassemia (HbSβ0)
Interventions
PK-optimized oral hydroxyurea at MTD until 15 years of age.
Drug
Lead sponsor
Children's Hospital Medical Center, Cincinnati
Other
Eligibility
6 Months and older
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2028
U.S. locations
1
States / cities
Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Sep 16, 2025 · Synced Oct 15, 2026, 7:08 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 15, 2026, 7:08 PM EDT
Terminated Phase 3 Interventional Results available

A Study of a Single Dose of Inclacumab to Reduce Re-admission in Participants With Sickle Cell Disease and Recurrent Vaso-occlusive Crises

NCT04927247
View directory record Official record
Conditions
Sickle Cell Disease, Vaso-occlusive Crisis, Vaso-occlusive Pain Episode in Sickle Cell Disease
Interventions
Inclacumab, Placebo
Drug
Lead sponsor
Pfizer
Industry
Eligibility
12 Years and older
Enrollment
72 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2023
U.S. locations
17
States / cities
Mobile, Alabama • Phoenix, Arizona • Little Rock, Arkansas + 11 more
Source: ClinicalTrials.gov public record
Updated Dec 11, 2024 · Synced Oct 15, 2026, 7:08 PM EDT
Completed Not applicable Interventional

The Longitudinal Relationship of HU Adherence to HRQOL, Barriers to Adherence and Habit in SCD.

NCT04691323
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Jul 31, 2026 · Synced Oct 15, 2026, 7:08 PM EDT
Completed Not applicable Interventional

Health Literacy - Neurocognitive Screening in Pediatric SCD

NCT04917783
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
Passport Card, No Passport card
Behavioral
Lead sponsor
Medical College of Wisconsin
Other
Eligibility
6 Years to 17 Years
Enrollment
46 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2021
U.S. locations
2
States / cities
Milwaukee, Wisconsin
Source: ClinicalTrials.gov public record
Updated Apr 5, 2022 · Synced Oct 15, 2026, 7:08 PM EDT
Completed Not applicable Interventional Results available

Transfusion of Prematures Trial

NCT01702805
View directory record Official record
Conditions
Infant, Newborn, Diseases, Infant, Extremely Low Birth Weight, Infant, Small for Gestational Age, Bronchopulmonary Dysplasia (BPD), Anemia
Interventions
Liberal Cell Transfusion, Restricted red cell transfusion
Procedure
Lead sponsor
NICHD Neonatal Research Network
Network
Eligibility
Up to 48 Hours
Enrollment
1,824 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2025
U.S. locations
20
States / cities
Birmingham, Alabama • Los Angeles, California • Palo Alto, California + 16 more
Source: ClinicalTrials.gov public record
Updated Nov 14, 2025 · Synced Oct 15, 2026, 7:08 PM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

A Study on the Effect of Etavopivat on Heart Rhythm in Healthy Participants

NCT07023029
View directory record Official record
Conditions
Healthy Volunteers, Sickle Cell Disease, Thalassemia
Interventions
Etavopivat, Moxifloxacin, Placebo
Drug
Lead sponsor
Novo Nordisk A/S
Industry
Eligibility
18 Years to 55 Years
Enrollment
33 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2025
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Jan 2, 2026 · Synced Oct 15, 2026, 7:08 PM EDT
Completed Phase 2 Interventional Results available

Pain Management in Children and Young Adults With Sickle Cell Disease

NCT01954927
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
Gabapentin, Placebo
Drug
Lead sponsor
St. Jude Children's Research Hospital
Other
Eligibility
1 Year to 20 Years
Enrollment
90 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2018
U.S. locations
1
States / cities
Memphis, Tennessee
Source: ClinicalTrials.gov public record
Updated Apr 2, 2019 · Synced Oct 15, 2026, 7:08 PM EDT
Recruiting Phase 1 Interventional

Escalating Doses of VAS-101 in Subjects With Stable Sickle Cell Disease

NCT07566494
View directory record Official record
Conditions
Sickle Cell Disease, Hemolytic Anemia
Interventions
VAS-101
Drug
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
18 Years to 90 Years
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2027
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Aug 24, 2026 · Synced Oct 15, 2026, 7:08 PM EDT