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ClinicalTrials.gov public records Last synced Oct 6, 2026, 10:42 PM EDT

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Showing 73–96 of 831 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Hemoglobinopathy (Disorder) Clear all
Completed No phase listed Observational

Phosphodiesterase Type-5 Inhibitor Therapy in Sickle Cell People With Pulmonary Hypertension

NCT03572036
View directory record Official record
Conditions
Pulmonary Hypertension, Sickle Cell Disease
Interventions
Not listed
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
18 Years to 99 Years
Enrollment
38 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2021
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Apr 19, 2021 · Synced Oct 6, 2026, 10:42 PM EDT
Completed Not applicable Interventional

Applying Directly Observed Therapy to Hydroxyurea to Realize Effectiveness

NCT06264700
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
Video Directly Observed Therapy (VDOT), Health Reminder Tip Alerts
Behavioral
Lead sponsor
Nationwide Children's Hospital
Other
Eligibility
11 Years and older
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
3
States / cities
Chicago, Illinois • Columbus, Ohio • Providence, Rhode Island
Source: ClinicalTrials.gov public record
Updated Aug 21, 2026 · Synced Oct 6, 2026, 10:42 PM EDT
Completed No phase listed Observational

Developing the Family Map: Looking at Communal Coping

NCT01633021
View directory record Official record
Conditions
Sickle Cell, Diabetes, Cancer, Cardiovascular Disease, Genetic Screening
Interventions
Not listed
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
18 Years to 100 Years
Enrollment
1,061 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2012
U.S. locations
3
States / cities
Bethesda, Maryland • Cincinnati, Ohio • Lewisburg, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Oct 2, 2026 · Synced Oct 6, 2026, 10:42 PM EDT
Completed Phase 3 Interventional Results available

A Study Evaluating the Efficacy and Safety of the LentiGlobin® BB305 Drug Product in Participants With Transfusion-Dependent β-Thalassemia, Who do Not Have a β0/β0 Genotype

NCT02906202
View directory record Official record
Conditions
Beta-Thalassemia
Interventions
LentiGlobin BB305 Drug Product
Genetic
Lead sponsor
Genetix Biotherapeutics Inc.
Industry
Eligibility
0 Years to 50 Years
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2022
U.S. locations
3
States / cities
Oakland, California • Chicago, Illinois • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jun 18, 2023 · Synced Oct 6, 2026, 10:42 PM EDT
Completed Phase 1 Interventional Results available

A Study to Evaluate GBT021601-012 Single Dose and Multiple Dose in Participants With Sickle Cell Disease (SCD)

NCT04983264
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
GBT021601
Drug
Lead sponsor
Pfizer
Industry
Eligibility
18 Years to 60 Years
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2022
U.S. locations
3
States / cities
Miami, Florida • Atlanta, Georgia
Source: ClinicalTrials.gov public record
Updated Jun 10, 2024 · Synced Oct 6, 2026, 10:42 PM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

A SAD/MAD to Assess the Safety, PK/PD of FT-4202 in Healthy Volunteers and Sickle Cell Disease Patients

NCT03815695
View directory record Official record
Conditions
Healthy Volunteers, Sickle Cell Disease
Interventions
FT-4202/Placebo
Drug
Lead sponsor
Forma Therapeutics, Inc.
Industry
Eligibility
12 Years to 65 Years
Enrollment
130 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2018 – 2021
U.S. locations
18
States / cities
Little Rock, Arkansas • Long Beach, California • Oakland, California + 12 more
Source: ClinicalTrials.gov public record
Updated Apr 10, 2024 · Synced Oct 6, 2026, 10:42 PM EDT
Active, not recruiting Phase 3 Interventional

Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Transfusion-Dependent β-Thalassemia (TDT)

NCT05356195
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 – 2027
U.S. locations
1
States / cities
Nashville, Tennessee
Source: ClinicalTrials.gov public record
Updated Jul 1, 2026 · Synced Oct 6, 2026, 10:42 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

Spectralis HRA+OCT Imaging of the Retina With Autofluorescence in Sickle Cell Disease

NCT01123369
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
Not listed
Lead sponsor
Medical University of South Carolina
Other
Eligibility
12 Years and older
Enrollment
8 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2010
U.S. locations
2
States / cities
Charleston, South Carolina
Source: ClinicalTrials.gov public record
Updated Sep 14, 2010 · Synced Oct 6, 2026, 10:42 PM EDT
Completed Phase 2Phase 3 Interventional Results available

Age of Blood in Sickle Cell Transfusion

NCT03704922
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
Transfusion
Biological
Lead sponsor
University of North Carolina, Chapel Hill
Other
Eligibility
16 Years to 60 Years
Enrollment
26 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2024
U.S. locations
3
States / cities
Atlanta, Georgia • Chapel Hill, North Carolina • Milwaukee, Wisconsin
Source: ClinicalTrials.gov public record
Updated Nov 19, 2024 · Synced Oct 6, 2026, 10:42 PM EDT
Terminated Early Phase 1 Interventional

Stem Cell Transplant in Patients With Severe Sickle Cell Disease

NCT03421756
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
Alemtuzumab, Total Body Irradiation, Sirolimus
Drug · Radiation
Lead sponsor
Kathleen Dorritie
Other
Eligibility
18 Years and older
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2022
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jun 28, 2022 · Synced Oct 6, 2026, 10:42 PM EDT
Completed No phase listed Observational

Bone Marrow for Hemoglobinopathy Research

NCT00669305
View directory record Official record
Conditions
Sickle Cell Anemia, Thalassemia
Interventions
Not listed
Lead sponsor
St. Jude Children's Research Hospital
Other
Eligibility
Not listed
Enrollment
26 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2019
U.S. locations
1
States / cities
Memphis, Tennessee
Source: ClinicalTrials.gov public record
Updated Feb 25, 2020 · Synced Oct 6, 2026, 10:42 PM EDT
Completed Phase 3 Interventional Results available

Sickle Cell Disease Treatment With Arginine Therapy (STArT) Trial

NCT04839354
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
L-Arginine Hydrochloride, Normal saline
Drug · Other
Lead sponsor
Claudia R. Morris
Other
Eligibility
3 Years to 21 Years
Enrollment
271 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2024
U.S. locations
10
States / cities
Los Angeles, California • San Francisco, California • Washington D.C., District of Columbia + 6 more
Source: ClinicalTrials.gov public record
Updated Jun 23, 2026 · Synced Oct 6, 2026, 10:42 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 6, 2026, 10:42 PM EDT
Recruiting Phase 1 Interventional

131I-apamistamab-based Conditioning for Hematopoietic Stem Cell Transplant (HSCT) in Advanced Sickle Cell Disease (SCD)

NCT07015684
View directory record Official record
Conditions
Sickling Disorder Due to Hemoglobin S
Interventions
131I-apamistmab, Sirolimus, Campath, Total Body Irradiation, Exchange Transfusion, Planar gamma imaging
Drug · Radiation · Procedure
Lead sponsor
Columbia University
Other
Eligibility
12 Years to 50 Years
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2032
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jun 10, 2026 · Synced Oct 6, 2026, 10:42 PM EDT
Active, not recruiting No phase listed Observational Accepts healthy volunteers

Sickle Cell Hemoglobinopathies and Bone Health

NCT02306993
View directory record Official record
Conditions
Sickle Cell Disease, Sickle Cell Trait
Interventions
Healthy volunteers without SCT, Healthy volunteers with SCT, Volunteers with SCD
Other
Lead sponsor
UConn Health
Other
Eligibility
18 Years to 45 Years · Female only
Enrollment
45 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2014 – 2028
U.S. locations
1
States / cities
Farmington, Connecticut
Source: ClinicalTrials.gov public record
Updated Aug 24, 2026 · Synced Oct 6, 2026, 10:42 PM EDT
Completed Phase 4 Interventional Accepts healthy volunteers Results available

Does IV Acetaminophen Reduce Opioid Requirement in Pediatric Patients With Acute Sickle Cell Crises?

NCT03541980
View directory record Official record
Conditions
Sickle Cell Anemia Crisis
Interventions
Acetaminophen, Normal saline
Drug
Lead sponsor
Newark Beth Israel Medical Center
Other
Eligibility
4 Years to 16 Years
Enrollment
71 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2018 – 2020
U.S. locations
1
States / cities
Newark, New York
Source: ClinicalTrials.gov public record
Updated Apr 2, 2021 · Synced Oct 6, 2026, 10:42 PM EDT
Completed No phase listed Observational

Role of Placenta Growth Factor in Sickle Acute Chest Syndrome

NCT00448370
View directory record Official record
Conditions
Anemia, Sickle Cell
Interventions
Not listed
Lead sponsor
Children's Hospital Medical Center, Cincinnati
Other
Eligibility
66 Months to 30 Years
Enrollment
136 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2007
U.S. locations
1
States / cities
Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Dec 21, 2017 · Synced Oct 6, 2026, 10:42 PM EDT
Not yet recruiting Phase 2 Interventional

Naive T Cell Deplete Grafts for GVHD Prevention in Non-Malignant Diseases

NCT07660783
View directory record Official record
Conditions
Bone Marrow Failure, Hemoglobinopathies, Primary Immune Deficiency (PID), Autoimmune Cytopenia, Immune Dysregulation, Hemophagocytic Lymphohistiocytosis (HLH), Non Malignant Disorders
Interventions
ALLOGENEIC CD34+ ENRICHED AND CD45RA- DEPLETED PBSCs
Biological
Lead sponsor
Fred Hutchinson Cancer Center
Other
Eligibility
6 Months to 50 Years
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2035
U.S. locations
2
States / cities
Seattle, Washington
Source: ClinicalTrials.gov public record
Updated Jun 22, 2026 · Synced Oct 6, 2026, 10:42 PM EDT
Completed No phase listed Observational

Functional Outcomes Following Hip Core Decompression in Younger Participants With Osteonecrosis

NCT02458937
View directory record Official record
Conditions
Osteonecrosis
Interventions
Functional Mobility Assessment, GAITRite® System, Range of Motion
Other
Lead sponsor
St. Jude Children's Research Hospital
Other
Eligibility
8 Years to 29 Years
Enrollment
17 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2018
U.S. locations
1
States / cities
Memphis, Tennessee
Source: ClinicalTrials.gov public record
Updated Nov 13, 2018 · Synced Oct 6, 2026, 10:42 PM EDT
Completed Not applicable Interventional Accepts healthy volunteers

Effect of Exercise on Biomarkers in SCT

NCT04273022
View directory record Official record
Conditions
Sickle Cell Trait
Interventions
Exercise
Other
Lead sponsor
St. Louis University
Other
Eligibility
18 Years to 70 Years
Enrollment
12 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2021 – 2024
U.S. locations
1
States / cities
St Louis, Missouri
Source: ClinicalTrials.gov public record
Updated Jan 28, 2025 · Synced Oct 6, 2026, 10:42 PM EDT
Active, not recruiting No phase listed Observational

Long - Term Follow Up of Sickle Cell Disease and Beta-thalassemia Subjects Previously Exposed to BIVV003 or ST-400.

NCT05145062
View directory record Official record
Conditions
Blood and Lymphatic Diseases
Interventions
BIVV003, ST-400
Drug
Lead sponsor
Sangamo Therapeutics
Industry
Eligibility
18 Years to 45 Years
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2038
U.S. locations
7
States / cities
Oakland, California • Sacramento, California • Atlanta, Georgia + 3 more
Source: ClinicalTrials.gov public record
Updated Nov 12, 2025 · Synced Oct 6, 2026, 10:42 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 6, 2026, 10:42 PM EDT
Not yet recruiting Not applicable Interventional Accepts healthy volunteers

Development and Evaluation of an Information Management System and Communication System for Population-wide Point-of-care Infant Sickle Cell Disease Screening

NCT06563440
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
Digital app and information system
Other
Lead sponsor
Makerere University
Other
Eligibility
Up to 5 Years
Enrollment
24,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2024 – 2028
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Aug 22, 2024 · Synced Oct 6, 2026, 10:42 PM EDT
Completed Not applicable Interventional Accepts healthy volunteers

SCD-PROMIS: A Software Platform to Enhance Self-efficacy and Patient-provider Engagement for Patients With Sickle Cell Pain

NCT03006718
View directory record Official record
Conditions
Sickle Cell Disease, Anemia, Anemia, Hemolytic, Anemia, Sickle Cell, Hematologic Diseases
Interventions
PROMIS for Pain Management App
Other
Lead sponsor
Julia Finkel
Other
Eligibility
8 Years to 21 Years
Enrollment
150 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2016 – 2019
U.S. locations
1
States / cities
Washington D.C., District of Columbia
Source: ClinicalTrials.gov public record
Updated Feb 24, 2021 · Synced Oct 6, 2026, 10:42 PM EDT