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ClinicalTrials.gov public records Last synced Oct 4, 2026, 9:26 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
Recruiting Not applicable Interventional

A Socio-ecological Approach for Improving Self-management in Adolescents With SCD

NCT06290401
View directory record Official record
Conditions
Anemia, Sickle Cell
Interventions
SCThrive, SCHealthED
Behavioral · Other
Lead sponsor
Children's Hospital Medical Center, Cincinnati
Other
Eligibility
13 Years to 21 Years
Enrollment
310 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2028
U.S. locations
4
States / cities
Wilmington, Delaware • Atlanta, Georgia • Cincinnati, Ohio + 1 more
Source: ClinicalTrials.gov public record
Updated May 11, 2026 · Synced Oct 4, 2026, 9:26 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 4, 2026, 9:26 PM EDT
Not yet recruiting Phase 3 Interventional

Optimal Timing of Ketamine Initiation for SCD Pain

NCT07450430
View directory record Official record
Conditions
Sickle Cell Disease, Sickle Cell Crisis
Interventions
Ketamine hydrochloride injection, Placebo
Drug · Other
Lead sponsor
Boston Children's Hospital
Other
Eligibility
6 Years to 24 Years
Enrollment
90 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2030
U.S. locations
1
States / cities
Brookline, Massachusetts
Source: ClinicalTrials.gov public record
Updated Mar 9, 2026 · Synced Oct 4, 2026, 9:26 PM EDT
Completed Phase 2 Interventional

CD34+Selection for Partially Matched Family or Matched Unrelated Adult Donor Transplant

NCT01049854
View directory record Official record
Conditions
Leukemia, Lymphoma, Bone Marrow Failure, Immunodeficiencies, Histiocytosis, Sickle Cell Disease, Beta Thalassemia, Inborn Errors of Metabolism
Interventions
Full Intensity with TBI, Full Intensity, Reduced Intensity, Reduced Intensity (Fanconi)
Drug
Lead sponsor
New York Medical College
Other
Eligibility
Up to 70 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2018
U.S. locations
1
States / cities
Valhalla, New York
Source: ClinicalTrials.gov public record
Updated Aug 21, 2018 · Synced Oct 4, 2026, 9:26 PM EDT
Completed Not applicable Interventional

A Stress and Pain Self-management m-Health App for Adult Outpatients With Sickle Cell Disease

NCT04484272
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
Self-monitoring of pain, stress, and opioid use + alerts/reminders + use of YCWS. [three video banks of RDE + Support])., Self-monitoring of pain, stress, and opioid use + alerts/reminders.
Behavioral
Lead sponsor
University of Florida
Other
Eligibility
18 Years to 99 Years
Enrollment
239 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2026
U.S. locations
1
States / cities
Gainesville, Florida
Source: ClinicalTrials.gov public record
Updated Sep 1, 2026 · Synced Oct 4, 2026, 9:26 PM EDT
Completed No phase listed Observational Results available

A Study to Learn About Sickle Cell Disease In Adult Patients

NCT05407805
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
Electronic Diary
Other
Lead sponsor
Pfizer
Industry
Eligibility
18 Years and older
Enrollment
98 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2024
U.S. locations
4
States / cities
Hollywood, Florida • Upper Marlboro, Maryland • Waltham, Massachusetts + 1 more
Source: ClinicalTrials.gov public record
Updated Jun 10, 2026 · Synced Oct 4, 2026, 9:26 PM EDT
Not listed Phase 4 Interventional

The Effect of Vitamin D Supplementation on Calcium Excretion in Thalassemia: a Dose Response Study

NCT01323608
View directory record Official record
Conditions
Thalassemia Major
Interventions
Vitamin D3, Placebo
Drug
Lead sponsor
Weill Medical College of Cornell University
Other
Eligibility
6 Years to 60 Years
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2014
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Apr 19, 2011 · Synced Oct 4, 2026, 9:26 PM EDT
Active, not recruiting Phase 2 Interventional

A Study to Test the Safety, Tolerability, and Efficacy of an Antibody, REGN7999, Injected Under the Skin for the Treatment of Iron Overload in Adult Participants With Non-Transfusion Dependent β-thalassemia, Using MRI Scans to Measure Iron Levels in the Body

NCT06421636
View directory record Official record
Conditions
Non-Transfusion Dependent Beta-Thalassemia (NTDT)
Interventions
REGN7999, Placebo
Drug
Lead sponsor
Regeneron Pharmaceuticals
Industry
Eligibility
18 Years to 65 Years
Enrollment
19 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jul 1, 2026 · Synced Oct 4, 2026, 9:26 PM 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 4, 2026, 9:26 PM EDT
Not listed Phase 1Phase 2 Interventional

CD34+ (Malignant) Stem Cell Selection for Patients Receiving Allogenic Stem Cell Transplant

NCT02061800
View directory record Official record
Conditions
Chronic Myeloid Leukemia (CML), Acute Myelogenous Leukemia (AML), Myelodysplastic Syndrome (MDS), Juvenile Myelomonocytic Leukemia (JMML), Acute Lymphoblastic Leukemia (ALL), Lymphoma (Hodgkin's and Non-Hodgkin's)
Interventions
CliniMACS CD34+ Reagent System, Thiotepa, Cyclophosphamide, Alemtuzumab, Tacrolimus, Melphalan, Busulfan, Fludarabine, Methylprednisolone
Device · Drug
Lead sponsor
Diane George
Other
Eligibility
Up to 22 Years
Enrollment
14 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2026
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jun 4, 2024 · Synced Oct 4, 2026, 9:26 PM EDT
Completed Not applicable Interventional Results available

Home-Based Intervention for Chronic Pain in Adults With Sickle Cell Disease

NCT04906707
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
EaseVRx, Audio-only version of EaseVRx
Device · Behavioral
Lead sponsor
Emory University
Other
Eligibility
18 Years to 50 Years
Enrollment
44 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2024
U.S. locations
1
States / cities
Atlanta, Georgia
Source: ClinicalTrials.gov public record
Updated Apr 20, 2025 · Synced Oct 4, 2026, 9:26 PM EDT
Completed Phase 2 Interventional

Hydroxyurea to Treat Beta-Thalassemia (Cooley's Anemia)

NCT00001958
View directory record Official record
Conditions
Beta Thalassemia, Hemoglobinopathy
Interventions
Hydroxyurea
Drug
Lead sponsor
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
NIH
Eligibility
Not listed
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1999 – 2002
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 4, 2008 · Synced Oct 4, 2026, 9:26 PM EDT
Completed No phase listed Observational

A 3-year, Prospective, Non-interventional, Multicenter Registry in Sickle Cell Disease (SCD) Patients

NCT01220115
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
Not listed
Lead sponsor
Novartis
Industry
Eligibility
2 Years and older
Enrollment
498 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2014
U.S. locations
42
States / cities
Little Rock, Arkansas • Oakland, California • Orange, California + 31 more
Source: ClinicalTrials.gov public record
Updated Dec 9, 2019 · Synced Oct 4, 2026, 9:26 PM EDT
Completed Phase 2 Interventional

Study of SANGUINATE™ In the Treatment of Sickle Cell Disease Patients With Vaso-Occlusive Crisis

NCT02411708
View directory record Official record
Conditions
Anemia, Sickle Cell
Interventions
SANGUINATE, Placebo
Drug
Lead sponsor
Prolong Pharmaceuticals
Industry
Eligibility
18 Years and older
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2017
U.S. locations
9
States / cities
Hollywood, Florida • Tampa, Florida • Baltimore, Maryland + 5 more
Source: ClinicalTrials.gov public record
Updated May 23, 2018 · Synced Oct 4, 2026, 9:26 PM EDT
Recruiting Not applicable Interventional

Peer Support for Adolescents and Emerging Adults With Sickle Cell Pain

NCT06374238
View directory record Official record
Conditions
Pain, Sickle Cell Disease
Interventions
CBT+ Health coach, CBT w/o Health Coach ( self-guided), Usual Care
Behavioral
Lead sponsor
University of Pittsburgh
Other
Eligibility
16 Years to 30 Years
Enrollment
470 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2028
U.S. locations
14
States / cities
Mobile, Alabama • Los Angeles, California • Hartford, Connecticut + 10 more
Source: ClinicalTrials.gov public record
Updated Jan 7, 2026 · Synced Oct 4, 2026, 9:26 PM EDT
Active, not recruiting Phase 1Phase 2 Interventional

A Study Evaluating the Safety and Efficacy of EDIT-301 in Participants With Severe Sickle Cell Disease (RUBY)

NCT04853576
View directory record Official record
Conditions
Sickle Cell Disease, Hemoglobinopathies
Interventions
EDIT-301
Genetic
Lead sponsor
Editas Medicine, Inc.
Industry
Eligibility
12 Years to 50 Years
Enrollment
45 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2025
U.S. locations
21
States / cities
Oakland, California • Aurora, Colorado • New Haven, Connecticut + 15 more
Source: ClinicalTrials.gov public record
Updated Jan 31, 2025 · Synced Oct 4, 2026, 9:26 PM EDT
Completed Phase 2 Interventional

Study of Deferasirox in Iron Overload From Beta-thalassemia Unable to be Treated With Deferoxamine or Chronic Anemias

NCT00061763
View directory record Official record
Conditions
Beta-thalassemia, Myelodysplastic Syndromes, Fanconi Syndrome, Anemia, Diamond-Blackfan, Anemia, Aplastic
Interventions
Deferasirox
Drug
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
2 Years and older
Enrollment
175 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2003
U.S. locations
6
States / cities
Oakland, California • Stanford, California • Arlington Heights, Illinois + 3 more
Source: ClinicalTrials.gov public record
Updated Aug 22, 2017 · Synced Oct 4, 2026, 9:26 PM EDT
Completed No phase listed Observational

Heart Disease in Sickle Cell Anemia

NCT00113152
View directory record Official record
Conditions
Sickle Cell Anemia, Pulmonary Hypertension
Interventions
Not listed
Lead sponsor
National Institutes of Health Clinical Center (CC)
NIH
Eligibility
18 Years to 60 Years
Enrollment
120 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2007
U.S. locations
2
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 2, 2017 · Synced Oct 4, 2026, 9:26 PM EDT
Completed No phase listed Observational Results available

Priapism in Boys and Men With Sickle Cell Disease - Demographics, Characteristics and Prevalence

NCT00300235
View directory record Official record
Conditions
Priapism, Anemia, Sickle Cell
Interventions
Not listed
Lead sponsor
University of Texas Southwestern Medical Center
Other
Eligibility
5 Years and older · Male only
Enrollment
1,464 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2008
U.S. locations
27
States / cities
Mobile, Alabama • Davis, California • Oakland, California + 16 more
Source: ClinicalTrials.gov public record
Updated Feb 1, 2019 · Synced Oct 4, 2026, 9:26 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

Microvascular and Cardiac Dysfunction in Paroxysmal Nocturnal Hemoglobinuria and Sickle Cell Disease

NCT01294891
View directory record Official record
Conditions
Rheologic Disease, Sickle Cell Disease, Paroxysmal Nocturnal Hemoglobinuria
Interventions
Imaging
Other
Lead sponsor
Oregon Health and Science University
Other
Eligibility
19 Years and older
Enrollment
6 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2011 – 2014
U.S. locations
1
States / cities
Portland, Oregon
Source: ClinicalTrials.gov public record
Updated Oct 28, 2014 · Synced Oct 4, 2026, 9:26 PM 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 4, 2026, 9:26 PM EDT
Completed Not applicable Interventional

Parent Educational Program for Children With Sickle Cell Disease

NCT00860782
View directory record Official record
Conditions
Hemoglobin SC Disease, Anemia, Sickle Cell
Interventions
Parent Educational Support
Behavioral
Lead sponsor
University of Miami
Other
Eligibility
6 Years to 12 Years
Enrollment
73 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2016
U.S. locations
1
States / cities
Miami, Florida
Source: ClinicalTrials.gov public record
Updated Aug 2, 2021 · Synced Oct 4, 2026, 9:26 PM EDT
Recruiting Phase 2 Interventional

TCR Alpha Beta T-cell Depleted Haploidentical HCT in the Treatment of Non-Malignant Hematological Disorders in Children

NCT04356469
View directory record Official record
Conditions
Hemoglobinopathy (Disorder), Severe Aplastic Anemia, Bone Marrow Failure Syndrome
Interventions
Haploidentical Hematopoietic Cell Transplantation
Biological
Lead sponsor
Johns Hopkins All Children's Hospital
Other
Eligibility
0 Years to 21 Years
Enrollment
17 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2027
U.S. locations
1
States / cities
St. Petersburg, Florida
Source: ClinicalTrials.gov public record
Updated Jul 1, 2026 · Synced Oct 4, 2026, 9:26 PM EDT
Terminated Phase 3 Interventional Results available

Dexamethasone to Treat Acute Chest Syndrome in People With Sickle Cell Disease

NCT00530270
View directory record Official record
Conditions
Anemia, Sickle Cell
Interventions
Dexamethasone, Placebo
Drug
Lead sponsor
Children's Hospital Medical Center, Cincinnati
Other
Eligibility
5 Years and older
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2008
U.S. locations
6
States / cities
Sacramento, California • Louisville, Kentucky • Boston, Massachusetts + 3 more
Source: ClinicalTrials.gov public record
Updated Apr 9, 2013 · Synced Oct 4, 2026, 9:26 PM EDT