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ClinicalTrials.gov public records Last synced Oct 14, 2026, 9:54 AM EDT

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Showing 265–288 of 1,044 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Congenital Anemias Clear all
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 14, 2026, 9:54 AM EDT
Completed Phase 1 Interventional

Sickle Cell Disease Conditioning for Bone Marrow Transplant

NCT00968162
View directory record Official record
Conditions
Bone Marrow Transplantation, Sickle Cell Disease
Interventions
fludarabine
Drug
Lead sponsor
Emory University
Other
Eligibility
Up to 18 Years
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2016
U.S. locations
10
States / cities
Birmingham, Alabama • Washington D.C., District of Columbia • St. Petersburg, Florida + 7 more
Source: ClinicalTrials.gov public record
Updated Jul 25, 2017 · Synced Oct 14, 2026, 9:54 AM EDT
Completed Not applicable Interventional

The Feasibility of the PAINReportIt Guided Relaxation Intervention-INPATIENT

NCT02729350
View directory record Official record
Conditions
Sickle Cell Disease, Stress, Pain
Interventions
Guided Relaxation video clip, Sickle Cell Disease Experience Discussion
Behavioral
Lead sponsor
University of Florida
Other
Eligibility
18 Years and older
Enrollment
46 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2019
U.S. locations
1
States / cities
Gainesville, Florida
Source: ClinicalTrials.gov public record
Updated Oct 28, 2025 · Synced Oct 14, 2026, 9:54 AM EDT
Not listed Phase 1 Interventional

A Clinical Trial to Evaluate the Safety of RP-L102 in Pediatric Subjects With Fanconi Anemia Subtype A

NCT03814408
View directory record Official record
Conditions
Fanconi Anemia Complementation Group A
Interventions
RP-L102
Biological
Lead sponsor
Rocket Pharmaceuticals Inc.
Industry
Eligibility
1 Year to 12 Years
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2022
U.S. locations
1
States / cities
Stanford, California
Source: ClinicalTrials.gov public record
Updated Nov 24, 2020 · Synced Oct 14, 2026, 9:54 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Quantitative Susceptibility Mapping (QSM) to Guide Iron Chelating Therapy

NCT04171635
View directory record Official record
Conditions
MRI Scans
Interventions
Quantitative Susceptibility Mapping (QSM) Magnetic Resonance Imaging (MRI), R2* Magnetic Resonance Imaging (MRI)
Radiation
Lead sponsor
Weill Medical College of Cornell University
Other
Eligibility
2 Years and older
Enrollment
55 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2019 – 2025
U.S. locations
2
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Feb 27, 2026 · Synced Oct 14, 2026, 9:54 AM EDT
Completed Early Phase 1 Interventional Accepts healthy volunteers

Milking the Umbilical Cord at Term Cesarean Birth

NCT01630993
View directory record Official record
Conditions
Anemia
Interventions
Umbilical Cord Milking
Other
Lead sponsor
University of Rhode Island
Other
Eligibility
18 Years to 44 Years · Female only
Enrollment
24 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2008 – 2009
U.S. locations
1
States / cities
Providence, Rhode Island
Source: ClinicalTrials.gov public record
Updated Oct 5, 2015 · Synced Oct 14, 2026, 9:54 AM EDT
Completed Not applicable Interventional

The Feasibility of the PAINReportIt Guided Relaxation Intervention-Outpatient

NCT02729363
View directory record Official record
Conditions
Sickle Cell Disease, Stress, Pain
Interventions
Guided Relaxation video clip, Sickle Cell Disease Experience Discussion
Behavioral
Lead sponsor
University of Florida
Other
Eligibility
18 Years and older
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2017
U.S. locations
1
States / cities
Gainesville, Florida
Source: ClinicalTrials.gov public record
Updated Jan 22, 2020 · Synced Oct 14, 2026, 9:54 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 14, 2026, 9:54 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 14, 2026, 9:54 AM EDT
Completed Phase 1Phase 2 Interventional Results available

Assessment of Algorithm-Based Hydroxyurea Dosing on Fetal Hemoglobin Response, Acute Complications, and Organ Function in People With Sickle Cell Disease

NCT02225132
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Aug 6, 2019 · Synced Oct 14, 2026, 9:54 AM EDT
Completed Not applicable Interventional

Sleep and Pain in Sickle Cell Disease

NCT03150433
View directory record Official record
Conditions
Sickle Cell Disease, Sleep Disturbance, Pain
Interventions
Behavioral symptom management, Sickle cell disease management
Behavioral · Other
Lead sponsor
Johns Hopkins University
Other
Eligibility
18 Years to 100 Years
Enrollment
57 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2023
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Jan 24, 2025 · Synced Oct 14, 2026, 9:54 AM EDT
Active, not recruiting Phase 2 Interventional Results available

A Study to Determine the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of AG-348 in Adult Participants With Non-transfusion-dependent Thalassemia

NCT03692052
View directory record Official record
Conditions
Thalassemia
Interventions
AG-348
Drug
Lead sponsor
Agios Pharmaceuticals, Inc.
Industry
Eligibility
18 Years and older
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2030
U.S. locations
2
States / cities
Oakland, California • Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Mar 30, 2026 · Synced Oct 14, 2026, 9:54 AM EDT
Completed No phase listed Observational

Long Term Effects of Erythrocyte Lysis

NCT00842621
View directory record Official record
Conditions
Sickle Cell Disease, Hemolytic Anemia
Interventions
Clinical Evaluations, Laboratory Studies
Procedure · Other
Lead sponsor
St. Jude Children's Research Hospital
Other
Eligibility
5 Years and older
Enrollment
390 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2016
U.S. locations
1
States / cities
Memphis, Tennessee
Source: ClinicalTrials.gov public record
Updated Jun 27, 2016 · Synced Oct 14, 2026, 9:54 AM EDT
Terminated Phase 3 Interventional Results available

Effect of Ticagrelor vs. Placebo in the Reduction of Vaso-occlusive Crises in Pediatric Patients With Sickle Cell Disease

NCT03615924
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
Ticagrelor, Placebo
Drug
Lead sponsor
AstraZeneca
Industry
Eligibility
2 Years to 17 Years
Enrollment
193 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2020
U.S. locations
7
States / cities
Fort Lauderdale, Florida • Miami, Florida • Chicago, Illinois + 4 more
Source: ClinicalTrials.gov public record
Updated Apr 9, 2021 · Synced Oct 14, 2026, 9:54 AM EDT
Completed Phase 2 Interventional

Assessing Physical Function in Sickle Cell Patients Taking Voxelotor

NCT06023199
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
Voxelotor
Drug
Lead sponsor
Inova Health Care Services
Other
Eligibility
18 Years to 65 Years
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2026
U.S. locations
1
States / cities
Falls Church, Virginia
Source: ClinicalTrials.gov public record
Updated Aug 31, 2026 · Synced Oct 14, 2026, 9:54 AM EDT
Completed Phase 2Phase 3 Interventional Results available

TBI Dose De-escalation for Fanconi Anemia

NCT00352976
View directory record Official record
Conditions
Fanconi Anemia
Interventions
Cyclophosphamide, Fludarabine, Total Body Irradiation, Bone Marrow Transplantation, Mycophenolate Mofetil, Sirolimus
Drug · Procedure
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
Not listed
Enrollment
83 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2020
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Nov 24, 2021 · Synced Oct 14, 2026, 9:54 AM EDT
No Longer Available No phase listed Expanded access

Expanded Access Protocol (EAP) Using the CliniMACS® Device for Pediatric Haplocompatible Donor Stem Cell Transplant

NCT01200017
View directory record Official record
Conditions
Acute Lymphoblastic Leukemia, Acute Myeloid Leukemia, Chronic Myeloid Leukemia, Myelodysplastic Syndrome, Lymphomas, Bone Marrow Failure, Hemoglobinopathy, Immune Deficiency, Osteopetrosis, Cytopenias, Leukocyte Disorders, Anemia Due to Intrinsic Red Cell Abnormality
Interventions
CD34+ enriched, T Cell Depleted donor stem cell product
Biological
Lead sponsor
Christopher Dvorak
Other
Eligibility
2 Months to 30 Years
Healthy volunteers
Healthy volunteers not accepted
U.S. locations
1
States / cities
San Francisco, California
Source: ClinicalTrials.gov public record
Updated May 27, 2021 · Synced Oct 14, 2026, 9:54 AM EDT
Terminated Not applicable Interventional Accepts healthy volunteers Results available

Effects of Transfusion of Older Stored Red Cells

NCT01534676
View directory record Official record
Conditions
Sickle Cell Disease, Thalassemia
Interventions
Transfusion, Blood, Chelation therapy
Procedure · Biological
Lead sponsor
Columbia University
Other
Eligibility
1 Year to 65 Years
Enrollment
3 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2012 – 2013
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Oct 5, 2016 · Synced Oct 14, 2026, 9:54 AM EDT
Completed Phase 1Phase 2 Interventional

Unrelated Hematopoietic Stem Cell Transplantation(HSCT) for Genetic Diseases of Blood Cells

NCT00730314
View directory record Official record
Conditions
Sickle Cell Disease, Thalassemia, Anemia, Granuloma, Wiskott-Aldrich Syndrome, Chediak Higashi Syndrome, Osteopetrosis, Neutropenia, Thrombocytopenia, Hurler Disease, Niemann-Pick Disease, Fucosidosis
Interventions
Hematopoietic stem cell transplantation
Procedure
Lead sponsor
Children's Hospital Los Angeles
Other
Eligibility
Up to 21 Years
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2015
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Jun 23, 2016 · Synced Oct 14, 2026, 9:54 AM 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 14, 2026, 9:54 AM EDT
Completed Not applicable Interventional Accepts healthy volunteers Results available

Sickle Cell Anemia WE CARE

NCT03716726
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
WE CARE SDoH Screening Survey, Family Resource Book, Standard of care
Behavioral · Other
Lead sponsor
Boston Medical Center
Other
Eligibility
18 Years and older
Enrollment
112 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2021 – 2023
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Mar 26, 2024 · Synced Oct 14, 2026, 9:54 AM EDT
Completed Phase 2 Interventional Results available

Bexarotene and GM-CSF in Treating Patients With Myelodysplastic Syndrome or Acute Myeloid Leukemia

NCT00425477
View directory record Official record
Conditions
Leukemia, Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Diseases
Interventions
sargramostim, bexarotene, cytogenetic analysis, fluorescence in situ hybridization, flow cytometry, laboratory biomarker analysis, biopsy
Biological · Drug · Genetic + 2 more
Lead sponsor
Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
Other
Eligibility
18 Years to 120 Years
Enrollment
26 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2016
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 5, 2018 · Synced Oct 14, 2026, 9:54 AM EDT
Completed No phase listed Observational

Quality of Life of Children With Sickle Cell Disease Who Are Getting Chronic Transfusions With a Lifeport

NCT00246077
View directory record Official record
Conditions
Anemia, Sickle Cell
Interventions
Not listed
Lead sponsor
Children's Mercy Hospital Kansas City
Other
Eligibility
8 Years to 18 Years
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005
U.S. locations
1
States / cities
Kansas City, Missouri
Source: ClinicalTrials.gov public record
Updated Apr 11, 2007 · Synced Oct 14, 2026, 9:54 AM EDT
Recruiting No phase listed Observational

HPC Offered for PRESERVE Expansion

NCT07145411
View directory record Official record
Conditions
Bone Marrow Transplant (BMT)
Interventions
Bone Marrow Transplant with Ossium's HPC, Marrow (bone marrow recovered from deceased organ and tissue donors)
Other
Lead sponsor
Ossium Health, Inc.
Industry
Eligibility
12 Years to 80 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2029
U.S. locations
4
States / cities
Birmingham, Alabama • Detroit, Michigan • Columbus, Ohio + 1 more
Source: ClinicalTrials.gov public record
Updated Aug 28, 2025 · Synced Oct 14, 2026, 9:54 AM EDT