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ClinicalTrials.gov public records Last synced May 21, 2026, 7:40 PM EDT

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Showing 25–48 of 24 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Hereditary Anemias Clear all
Completed Phase 2 Interventional Results available
View directory record Official record
Conditions
Accelerated Phase Chronic Myelogenous Leukemia, 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), Adult Grade III Lymphomatoid Granulomatosis, B-cell Chronic Lymphocytic Leukemia, Chronic Myelogenous Leukemia, BCR-ABL1 Positive, Chronic Myelomonocytic Leukemia, Chronic Phase Chronic Myelogenous Leukemia, Contiguous Stage II Adult Burkitt Lymphoma, Contiguous Stage II Adult Diffuse Large Cell Lymphoma, Contiguous Stage II Adult Diffuse Mixed Cell Lymphoma, Contiguous Stage II Adult Diffuse Small Cleaved Cell Lymphoma, Contiguous Stage II Adult Immunoblastic Large Cell Lymphoma, Contiguous Stage II Adult Lymphoblastic Lymphoma, Contiguous Stage II Grade 1 Follicular Lymphoma, Contiguous Stage II Grade 2 Follicular Lymphoma, Contiguous Stage II Grade 3 Follicular Lymphoma, Contiguous Stage II Mantle Cell Lymphoma, Contiguous Stage II Marginal Zone Lymphoma, Contiguous Stage II Small Lymphocytic Lymphoma, Cutaneous B-cell Non-Hodgkin Lymphoma, Extranodal Marginal Zone B-cell Lymphoma of Mucosa-associated Lymphoid Tissue, Graft Versus Host Disease, Intraocular Lymphoma, Myelodysplastic Syndrome With Isolated Del(5q), Myelodysplastic/Myeloproliferative Neoplasm, Unclassifiable, Nodal Marginal Zone B-cell Lymphoma, Noncontiguous Stage II Adult Burkitt Lymphoma, Noncontiguous Stage II Adult Diffuse Large Cell Lymphoma, Noncontiguous Stage II Adult Diffuse Mixed Cell Lymphoma, Noncontiguous Stage II Adult Diffuse Small Cleaved Cell Lymphoma, Noncontiguous Stage II Adult Immunoblastic Large Cell Lymphoma, Noncontiguous Stage II Adult Lymphoblastic Lymphoma, Noncontiguous Stage II Grade 1 Follicular Lymphoma, Noncontiguous Stage II Grade 2 Follicular Lymphoma, Noncontiguous Stage II Grade 3 Follicular Lymphoma, Noncontiguous Stage II Mantle Cell Lymphoma, Noncontiguous Stage II Marginal Zone Lymphoma, Noncontiguous Stage II Small Lymphocytic Lymphoma, Post-transplant Lymphoproliferative Disorder, Primary Central Nervous System Hodgkin Lymphoma, Primary Central Nervous System Non-Hodgkin Lymphoma, Recurrent Adult Acute Myeloid Leukemia, Recurrent Adult Burkitt Lymphoma, Recurrent Adult Diffuse Large Cell Lymphoma, Recurrent Adult Diffuse Mixed Cell Lymphoma, Recurrent Adult Diffuse Small Cleaved Cell Lymphoma, Recurrent Adult Grade III Lymphomatoid Granulomatosis, Recurrent Adult Hodgkin Lymphoma, Recurrent Adult Immunoblastic Large Cell Lymphoma, Recurrent Adult Lymphoblastic Lymphoma, Recurrent Grade 1 Follicular Lymphoma, Recurrent Grade 2 Follicular Lymphoma, Recurrent Grade 3 Follicular Lymphoma, Recurrent Mantle Cell Lymphoma, Recurrent Marginal Zone Lymphoma, Recurrent Small Lymphocytic Lymphoma, Refractory Anemia, Refractory Anemia With Excess Blasts, Refractory Anemia With Ringed Sideroblasts, Refractory Chronic Lymphocytic Leukemia, Refractory Cytopenia With Multilineage Dysplasia, Refractory Hairy Cell Leukemia, Relapsing Chronic Myelogenous Leukemia, Secondary Central Nervous System Hodgkin Lymphoma, Secondary Central Nervous System Non-Hodgkin Lymphoma, Small Intestine Lymphoma, Splenic Marginal Zone Lymphoma, Stage I Adult Burkitt Lymphoma, Stage I Adult Diffuse Large Cell Lymphoma, Stage I Adult Diffuse Mixed Cell Lymphoma, Stage I Adult Diffuse Small Cleaved Cell Lymphoma, Stage I Adult Hodgkin Lymphoma, Stage I Adult Immunoblastic Large Cell Lymphoma, Stage I Adult Lymphoblastic Lymphoma, Stage I Chronic Lymphocytic Leukemia, Stage I Grade 1 Follicular Lymphoma, Stage I Grade 2 Follicular Lymphoma, Stage I Grade 3 Follicular Lymphoma, Stage I Mantle Cell Lymphoma, Stage I Marginal Zone Lymphoma, Stage I Small Lymphocytic Lymphoma, Stage II Adult Hodgkin Lymphoma, Stage II Chronic Lymphocytic Leukemia, Stage III Adult Burkitt Lymphoma, Stage III Adult Diffuse Large Cell Lymphoma, Stage III Adult Diffuse Mixed Cell Lymphoma, Stage III Adult Diffuse Small Cleaved Cell Lymphoma, Stage III Adult Hodgkin Lymphoma, Stage III Adult Immunoblastic Large Cell Lymphoma, Stage III Adult Lymphoblastic Lymphoma, Stage III Chronic Lymphocytic Leukemia, Stage III Grade 1 Follicular Lymphoma, Stage III Grade 2 Follicular Lymphoma, Stage III Grade 3 Follicular Lymphoma, Stage III Mantle Cell Lymphoma, Stage III Marginal Zone Lymphoma, Stage IV Adult Burkitt Lymphoma, Stage IV Adult Diffuse Large Cell Lymphoma, Stage IV Adult Diffuse Mixed Cell Lymphoma, Stage IV Adult Diffuse Small Cleaved Cell Lymphoma, Stage IV Adult Hodgkin Lymphoma, Stage IV Adult Immunoblastic Large Cell Lymphoma, Stage IV Adult Lymphoblastic Lymphoma, Stage IV Chronic Lymphocytic Leukemia, Stage IV Grade 1 Follicular Lymphoma, Stage IV Grade 2 Follicular Lymphoma, Stage IV Grade 3 Follicular Lymphoma, Stage IV Mantle Cell Lymphoma, Stage IV Marginal Zone Lymphoma, Stage IV Small Lymphocytic Lymphoma, Testicular Lymphoma, Waldenström Macroglobulinemia
Interventions
vorinostat, tacrolimus, cyclosporine, methotrexate, laboratory biomarker analysis, pharmacological study
Drug · Other
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
18 Years to 75 Years
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2014
U.S. locations
1
States / cities
Ann Arbor, Michigan
Source: ClinicalTrials.gov public record
Updated Jul 23, 2018 · Synced May 21, 2026, 7:40 PM EDT
Completed No phase listed Observational
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
Brief Cognitive-Behavioral Pain Management Training
Behavioral
Lead sponsor
University of South Carolina
Other
Eligibility
8 Years to 18 Years
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2012
U.S. locations
1
States / cities
Columbia, South Carolina
Source: ClinicalTrials.gov public record
Updated Apr 27, 2021 · Synced May 21, 2026, 7:40 PM EDT
Completed Phase 2Phase 3 Interventional
View directory record Official record
Conditions
Sickle Cell Disease, Hematological Diseases, Hemoglobinopathies
Interventions
CTX001
Biological
Lead sponsor
Vertex Pharmaceuticals Incorporated
Industry
Eligibility
12 Years to 35 Years
Enrollment
63 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2025
U.S. locations
9
States / cities
Palo Alto, California • Chicago, Illinois • New York, New York + 4 more
Source: ClinicalTrials.gov public record
Updated Aug 10, 2025 · Synced May 21, 2026, 7:40 PM EDT
Completed No phase listed Observational
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
Not listed
Lead sponsor
Children's Hospital Medical Center, Cincinnati
Other
Eligibility
Not listed
Enrollment
320 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2020
U.S. locations
4
States / cities
Atlanta, Georgia • Louisville, Kentucky • Bethesda, Maryland + 1 more
Source: ClinicalTrials.gov public record
Updated Mar 28, 2021 · Synced May 21, 2026, 7:40 PM EDT
Completed No phase listed Observational Accepts healthy volunteers
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
Brigance Preschool Screen -II
Other
Lead sponsor
St. Jude Children's Research Hospital
Other
Eligibility
3 Years to 4 Years
Enrollment
248 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2010 – 2015
U.S. locations
1
States / cities
Memphis, Tennessee
Source: ClinicalTrials.gov public record
Updated Jun 27, 2018 · Synced May 21, 2026, 7:40 PM EDT
Active, not recruiting Phase 3 Interventional
View directory record Official record
Conditions
Beta-Thalassemia, Thalassemia, Genetic Diseases, Inborn, Hematologic Diseases, Hemoglobinopathies
Interventions
CTX001
Biological
Lead sponsor
Vertex Pharmaceuticals Incorporated
Industry
Eligibility
2 Years to 11 Years
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2026
U.S. locations
1
States / cities
Nashville, Tennessee
Source: ClinicalTrials.gov public record
Updated Mar 2, 2026 · Synced May 21, 2026, 7:40 PM EDT
Not listed Not applicable Interventional
View directory record Official record
Conditions
Pediatric Cancer, Sickle Cell Disease, Quality of Life, Depressive Symptoms, Coping Skills
Interventions
FOCUS
Behavioral
Lead sponsor
Georgia State University
Other
Eligibility
12 Years to 18 Years
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2021
U.S. locations
2
States / cities
Atlanta, Georgia
Source: ClinicalTrials.gov public record
Updated Nov 12, 2020 · Synced May 21, 2026, 7:40 PM EDT
Completed Early Phase 1 Interventional Accepts healthy volunteers Results available
View directory record Official record
Conditions
Sickle Cell Disease, Sickle Cell Anemia
Interventions
Biotin label
Drug
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
18 Years to 99 Years
Enrollment
22 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2021 – 2023
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Feb 12, 2024 · Synced May 21, 2026, 7:40 PM EDT
Completed Phase 1 Interventional Results available
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
TAK-755, Placebo
Drug · Other
Lead sponsor
Shire
Industry
Eligibility
18 Years to 65 Years
Enrollment
19 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2022
U.S. locations
14
States / cities
Birmingham, Alabama • Little Rock, Arkansas • Aurora, Colorado + 11 more
Source: ClinicalTrials.gov public record
Updated Apr 22, 2024 · Synced May 21, 2026, 7:40 PM EDT
Completed Phase 1Phase 2 Interventional Results available
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
Simvastatin
Drug
Lead sponsor
UCSF Benioff Children's Hospital Oakland
Other
Eligibility
13 Years and older
Enrollment
42 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2011
U.S. locations
1
States / cities
Oakland, California
Source: ClinicalTrials.gov public record
Updated Sep 16, 2013 · Synced May 21, 2026, 7:40 PM EDT
Terminated Phase 2 Interventional Results available
View directory record Official record
Conditions
Pulmonary Hypertension, Sickle Cell Disease
Interventions
macitentan
Drug
Lead sponsor
Boston University
Other
Eligibility
18 Years and older
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2019
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Dec 7, 2020 · Synced May 21, 2026, 7:40 PM EDT
Completed Phase 2 Interventional
View directory record Official record
Conditions
Thalassemia Major
Interventions
DST-0509, Jadenu, Exjade
Drug
Lead sponsor
DisperSol Technologies, LLC
Industry
Eligibility
8 Years and older
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2021
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jan 31, 2022 · Synced May 21, 2026, 7:40 PM EDT
Terminated Phase 3 Interventional Results available
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
Voxelotor Only Product in Oral Dose Form, Placebo
Drug
Lead sponsor
Pfizer
Industry
Eligibility
8 Years to 18 Years
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated May 1, 2024 · Synced May 21, 2026, 7:40 PM EDT
Withdrawn Phase 2 Interventional
View directory record Official record
Conditions
Sickle Cell Disease, Vaso-occlusive Crisis
Interventions
Patient controlled analgesia, nurse-administered intermittent IV bolus opioid therapy (NAIBOD)
Procedure · Drug
Lead sponsor
Johns Hopkins University
Other
Eligibility
18 Years to 100 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2009
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 23, 2017 · Synced May 21, 2026, 7:40 PM EDT
Completed Not applicable Interventional
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 25 Years
Enrollment
33 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2020
U.S. locations
1
States / cities
Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated Jul 28, 2021 · Synced May 21, 2026, 7:40 PM EDT
Completed No phase listed Observational
View directory record Official record
Conditions
Anemia, Sickle Cell, Sickling Disorder Due to Hemoglobin S
Interventions
Rapid manual partial exchange transfusion, Simple Transfusion
Other
Lead sponsor
Chattanooga-Hamilton County Hospital Authority
Other
Eligibility
3 Years to 25 Years
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2017
U.S. locations
1
States / cities
Chattanooga, Tennessee
Source: ClinicalTrials.gov public record
Updated Jul 26, 2018 · Synced May 21, 2026, 7:40 PM EDT
Completed Phase 1 Interventional Accepts healthy volunteers
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 1, 2026 · Synced May 21, 2026, 7:40 PM EDT
Terminated No phase listed Observational Results available
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
Oxbryta® (voxelotor) 500mg Tablets
Drug
Lead sponsor
Pfizer
Industry
Eligibility
4 Years and older
Enrollment
265 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2024
U.S. locations
43
States / cities
Mobile, Alabama • Phoenix, Arizona • La Jolla, California + 24 more
Source: ClinicalTrials.gov public record
Updated Dec 30, 2025 · Synced May 21, 2026, 7:40 PM EDT
Recruiting No phase listed Observational
View directory record Official record
Conditions
Beta-Thalassemia
Interventions
No Intervention
Other
Lead sponsor
Genetix Biotherapeutics Inc.
Industry
Eligibility
Not listed
Enrollment
150 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2043
U.S. locations
6
States / cities
Oakland, California • Palo Alto, California • Minneapolis, Minnesota + 3 more
Source: ClinicalTrials.gov public record
Updated Feb 10, 2025 · Synced May 21, 2026, 7:40 PM EDT
Completed Phase 1 Interventional Accepts healthy volunteers Results available
View directory record Official record
Conditions
Anemia, Sickle Cell
Interventions
HBI-002
Drug
Lead sponsor
Hillhurst Biopharmaceuticals, Inc.
Industry
Eligibility
18 Years to 55 Years
Enrollment
20 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2022 – 2023
U.S. locations
1
States / cities
San Diego, California
Source: ClinicalTrials.gov public record
Updated May 22, 2025 · Synced May 21, 2026, 7:40 PM EDT
Completed Phase 2 Interventional Results available
View directory record Official record
Conditions
Priapism
Interventions
Crizanlizumab
Drug
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
12 Years to 100 Years · Male only
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2023
U.S. locations
13
States / cities
Birmingham, Alabama • Farmington, Connecticut • Washington D.C., District of Columbia + 10 more
Source: ClinicalTrials.gov public record
Updated Jan 12, 2026 · Synced May 21, 2026, 7:40 PM EDT
Completed Phase 1 Interventional
View directory record Official record
Conditions
Transfusional Iron Overload, Beta-thalassemia
Interventions
FBS0701
Drug
Lead sponsor
FerroKin BioSciences, Inc.
Industry
Eligibility
18 Years and older
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2009
U.S. locations
3
States / cities
Oakland, California • Boston, Massachusetts • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Feb 9, 2012 · Synced May 21, 2026, 7:40 PM EDT
Terminated Not applicable Interventional Results available
View directory record Official record
Conditions
Transfusion Dependent Thalassemia
Interventions
Mirasol Red Blood Cells (MIR RBCs), Reference Red Blood Cells (REF RBCs)
Device
Lead sponsor
Terumo BCTbio
Industry
Eligibility
12 Years and older
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018
U.S. locations
4
States / cities
Oakland, California • Boston, Massachusetts • New York, New York + 1 more
Source: ClinicalTrials.gov public record
Updated Feb 13, 2024 · Synced May 21, 2026, 7:40 PM EDT
Completed Not applicable Interventional
View directory record Official record
Conditions
To Assess the Safety and Tolerabiltiy of an Amino Acid Composition in Subjects With Sickle Cell Disease, Anemia, Sickle Cell, Sickle Cell Anemia, Sickle Cell Disorders, Sickle Cell Disease
Interventions
AXA4010
Dietary Supplement
Lead sponsor
Axcella Health, Inc
Industry
Eligibility
12 Years and older
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2021
U.S. locations
4
States / cities
Hollywood, Florida • Miami, Florida • Atlanta, Georgia + 1 more
Source: ClinicalTrials.gov public record
Updated Mar 31, 2021 · Synced May 21, 2026, 7:40 PM EDT