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ClinicalTrials.gov public records Last synced Oct 17, 2026, 6:54 PM EDT

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Showing 745–768 of 1,045 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Congenital Anemias Clear all
Completed Not applicable Interventional

Web-MAP Intervention for Youth With Sickle Cell Disease

NCT02511769
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
WEB-MAP Educational Control Group, WEB-MAP CBT
Behavioral
Lead sponsor
Connecticut Children's Medical Center
Other
Eligibility
11 Years to 18 Years
Enrollment
42 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2016
U.S. locations
1
States / cities
Hartford, Connecticut
Source: ClinicalTrials.gov public record
Updated Feb 1, 2017 · Synced Oct 17, 2026, 6:54 PM EDT
Withdrawn Phase 1 Interventional

A Pilot Study of Azithromycin Prophylaxis for Acute Chest Syndrome in Sickle Cell Disease

NCT02630394
View directory record Official record
Conditions
Sickle Cell Disease, Acute Chest Syndrome
Interventions
Azithromycin
Drug
Lead sponsor
University of Mississippi Medical Center
Other
Eligibility
6 Years to 16 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2017
U.S. locations
2
States / cities
Jackson, Mississippi • Nashville, Tennessee
Source: ClinicalTrials.gov public record
Updated Jun 21, 2018 · Synced Oct 17, 2026, 6:54 PM EDT
Completed Phase 1Phase 2 Interventional Results available

A Study Evaluating the Safety and Efficacy of Lovo-cel in Severe Sickle Cell Disease

NCT02140554
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
lovo-cel
Genetic
Lead sponsor
Genetix Biotherapeutics Inc.
Industry
Eligibility
12 Years to 50 Years
Enrollment
54 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2024
U.S. locations
11
States / cities
Birmingham, Alabama • Oakland, California • Atlanta, Georgia + 8 more
Source: ClinicalTrials.gov public record
Updated Mar 14, 2025 · Synced Oct 17, 2026, 6:54 PM EDT
Recruiting No phase listed Observational

Natural History of Acquired and Inherited Bone Marrow Failure Syndromes

NCT05012111
View directory record Official record
Conditions
Severe Aplastic Anemia, Telomere Biology Disorders, Inherited Bone Marrow Failure Syndromes
Interventions
Not listed
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
2 Years to 99 Years
Enrollment
1,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2041
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Sep 30, 2026 · Synced Oct 17, 2026, 6:54 PM EDT
Recruiting Phase 2 Interventional

A Study of Immune Suppression Treatment for People With Sickle Cell Disease or β-Thalassemia Who Are Going to Receive an Allogeneic Hematopoietic Cell Transplantation (HCT)

NCT05736419
View directory record Official record
Conditions
Sickle Cell Disease, Thalassemia, Beta, Thalassemia
Interventions
Fludarabine, Cyclophosphamide, Tacrolimus, Mycophenolate Mofetil, Rabbit ATG, Dexamethasone, Bortezomib, Rituximab
Drug · Biological
Lead sponsor
Memorial Sloan Kettering Cancer Center
Other
Eligibility
2 Years to 50 Years
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2027
U.S. locations
6
States / cities
Basking Ridge, New Jersey • Middletown, New Jersey • Montvale, New Jersey + 3 more
Source: ClinicalTrials.gov public record
Updated Jun 3, 2026 · Synced Oct 17, 2026, 6:54 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 17, 2026, 6:54 PM EDT
Recruiting No phase listed Observational

MRI as Noninvasive Innovative Approach in Detection and Monitoring of Malignant Oral Lesions in Fanconi Anemia Patients

NCT07649031
View directory record Official record
Conditions
Fanconi Anemia
Interventions
MRIs
Diagnostic Test
Lead sponsor
University of Minnesota
Other
Eligibility
18 Years and older
Enrollment
80 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2030
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Jul 2, 2026 · Synced Oct 17, 2026, 6:54 PM EDT
Completed Not applicable Interventional Results available

LCI-HEM-SCD-ST3P-UP-001: The Sickle Cell Trevor Thompson Transition Project (ST3P-UP Study)

NCT03593395
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
Peer Mentoring [PM], Structured Education Based Transition Program STE
Other
Lead sponsor
Wake Forest University Health Sciences
Other
Eligibility
16 Years and older
Enrollment
291 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2023
U.S. locations
14
States / cities
Birmingham, Alabama • Mobile, Alabama • Miami, Florida + 10 more
Source: ClinicalTrials.gov public record
Updated Sep 3, 2024 · Synced Oct 17, 2026, 6:54 PM EDT
Completed Not applicable Interventional

Stem Cell Transplantation (SCT) for Genetic Diseases

NCT00004378
View directory record Official record
Conditions
Thrombocytopenia, Metachromatic Leukodystrophy, Fanconi's Anemia, Thalassemia Major, Pure Red-Cell Aplasia, Inborn Errors of Metabolism
Interventions
Stem Cell Transplantation
Procedure
Lead sponsor
National Center for Research Resources (NCRR)
NIH
Eligibility
0 Years to 17 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 1995
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Jun 24, 2005 · Synced Oct 17, 2026, 6:54 PM EDT
Completed Phase 2 Interventional Results available

NMA Allogeneic Hematopoietic Cell Transplant in Hematologic Cancer/Disorders

NCT00053989
View directory record Official record
Conditions
Chronic Myeloproliferative Disorders, Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm, Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Diseases, Fanconi Anemia, Aplastic Anemia
Interventions
anti-thymocyte globulin, graft-versus-tumor induction therapy, sargramostim, therapeutic allogeneic lymphocytes, cyclophosphamide, fludarabine phosphate, methylprednisolone, mycophenolate mofetil, tacrolimus, allogeneic bone marrow transplantation, peripheral blood stem cell transplantation, umbilical cord blood transplantation
Biological · Drug · Procedure
Lead sponsor
Roswell Park Cancer Institute
Other
Eligibility
4 Years to 75 Years
Enrollment
41 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2002 – 2018
U.S. locations
1
States / cities
Buffalo, New York
Source: ClinicalTrials.gov public record
Updated Feb 10, 2020 · Synced Oct 17, 2026, 6:54 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 39 Years
Enrollment
390 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 Oct 8, 2026 · Synced Oct 17, 2026, 6:54 PM EDT
Completed No phase listed Observational Results available

Novel Cardiac Magnetic Resonance Imaging to Define a Unique Restrictive Cardiomyopathy in Sickle Cell Disease

NCT02410811
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
Cardiac magnetic resonance imaging (CMR)
Other
Lead sponsor
Children's Hospital Medical Center, Cincinnati
Other
Eligibility
6 Years to 65 Years
Enrollment
33 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2019
U.S. locations
1
States / cities
Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Nov 2, 2020 · Synced Oct 17, 2026, 6:54 PM EDT
Completed Not applicable Interventional

An mHealth Strategy to Improve Medication Adherence in Adolescents With Sickle Cell Disease

NCT04688411
View directory record Official record
Conditions
Sickle Cell Disease, Sickle Beta Zero Thalassemia, Sickle B+ Thalassemia, Sickle Cell Hemoglobin C
Interventions
MED-Go App
Behavioral
Lead sponsor
Ann & Robert H Lurie Children's Hospital of Chicago
Other
Eligibility
12 Years to 21 Years
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2025
U.S. locations
1
States / cities
Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated Jul 31, 2026 · Synced Oct 17, 2026, 6:54 PM EDT
Completed Phase 1Phase 2 Interventional Results available

Pilot Phase I/II Study of Amino Acid Leucine in Treatment of Patients With Transfusion-Dependent Diamond Blackfan Anemia

NCT01362595
View directory record Official record
Conditions
Diamond Blackfan Anemia, Blackfan Diamond Syndrome, DBA, Congenital Hypoplastic Anemia, Pure Red Cell Aplasia
Interventions
leucine
Drug
Lead sponsor
Northwell Health
Other
Eligibility
2 Years and older
Enrollment
55 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2020
U.S. locations
11
States / cities
Phoenix, Arizona • Palo Alto, California • Indianapolis, Indiana + 8 more
Source: ClinicalTrials.gov public record
Updated Dec 2, 2022 · Synced Oct 17, 2026, 6:54 PM EDT
Completed Not applicable Interventional Accepts healthy volunteers Results available

Gaming Technology to Engage Adolescent Sickle Cell Patients in Pain Management

NCT03291613
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
Pinpoint App
Other
Lead sponsor
Klein Buendel, Inc.
Industry
Eligibility
13 Years and older
Enrollment
13 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2017
U.S. locations
2
States / cities
Golden, Colorado • Munster, Indiana
Source: ClinicalTrials.gov public record
Updated Feb 6, 2019 · Synced Oct 17, 2026, 6:54 PM 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 17, 2026, 6:54 PM EDT
Completed No phase listed Observational

Early Diagnosis of Sickle Acute Chest Syndrome Using a Combination of Plasma Bimarkers and Chest Imaging

NCT03478917
View directory record Official record
Conditions
Sickle Cell Disease (SCD)
Interventions
Not listed
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
10 Years to 85 Years
Enrollment
65 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2021
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 17, 2023 · Synced Oct 17, 2026, 6:54 PM EDT
Completed Not applicable Interventional

COVID-19 Vaccination Hesitancy in Adults With Sickle Cell Disease

NCT05998824
View directory record Official record
Conditions
Sickle Cell Disease, COVID-19 Vaccine, Vaccine Hesitancy
Interventions
SCD-specific COVID-19 vaccination information (SCVI) video
Behavioral
Lead sponsor
Duke University
Other
Eligibility
18 Years to 100 Years
Enrollment
33 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2024
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Dec 24, 2024 · Synced Oct 17, 2026, 6:54 PM EDT
Completed No phase listed Observational

A New Reagent Assay Examining Natural Parvovirus B19 Infection in Sickle Cell Disease

NCT02261480
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
Blood draw, Nasopharyngeal wash
Other
Lead sponsor
St. Jude Children's Research Hospital
Other
Eligibility
1 Year and older
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2016
U.S. locations
1
States / cities
Memphis, Tennessee
Source: ClinicalTrials.gov public record
Updated Sep 19, 2016 · Synced Oct 17, 2026, 6:54 PM EDT
Recruiting Phase 1 Interventional

SCD Stem Cell Mobilization and Apheresis Using Motixafortide

NCT06442761
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
Motixafortide
Drug
Lead sponsor
St. Jude Children's Research Hospital
Other
Eligibility
18 Years and older
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2028
U.S. locations
3
States / cities
Birmingham, Alabama • Bethesda, Maryland • Memphis, Tennessee
Source: ClinicalTrials.gov public record
Updated Sep 8, 2026 · Synced Oct 17, 2026, 6:54 PM EDT
Completed Phase 2 Interventional

A Study of Etavopivat in Patients With Thalassemia or Sickle Cell Disease

NCT04987489
View directory record Official record
Conditions
Sickle Cell Disease, Thalassemia
Interventions
Etavopivat tablets
Drug
Lead sponsor
Forma Therapeutics, Inc.
Industry
Eligibility
12 Years to 65 Years
Enrollment
53 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2025
U.S. locations
14
States / cities
Cerritos, California • Los Angeles, California • Oakland, California + 9 more
Source: ClinicalTrials.gov public record
Updated Dec 16, 2025 · Synced Oct 17, 2026, 6:54 PM EDT
Completed Phase 1 Interventional

Safety, Pharmacokinetic, and Pharmacodynamic Study of NKTT120 in Adult Patients With Stable Sickle Cell Disease (SCD)

NCT01783691
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
NKTT120
Drug
Lead sponsor
NKT Therapeutics
Industry
Eligibility
18 Years to 60 Years
Enrollment
21 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2015
U.S. locations
6
States / cities
Oakland, California • Baltimore, Maryland • Boston, Massachusetts + 3 more
Source: ClinicalTrials.gov public record
Updated Jun 26, 2015 · Synced Oct 17, 2026, 6:54 PM EDT
Completed Phase 3 Interventional Results available

Stem Cell Transplantation for Sickle Cell Anemia

NCT01877837
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
Alemtuzumab, Fludarabine, Melphalan, Stem Cells
Drug · Procedure
Lead sponsor
Hackensack Meridian Health
Other
Eligibility
2 Years to 30 Years
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2021
U.S. locations
1
States / cities
Hackensack, New Jersey
Source: ClinicalTrials.gov public record
Updated Nov 7, 2022 · Synced Oct 17, 2026, 6:54 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

Effect of G6PD Deficiency on Red Blood Cell Storage

NCT04081272
View directory record Official record
Conditions
G6PD Deficiency
Interventions
Sodium Chromate Cr51
Drug
Lead sponsor
Columbia University
Other
Eligibility
18 Years to 70 Years · Male only
Enrollment
40 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2012 – 2017
U.S. locations
2
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Sep 17, 2019 · Synced Oct 17, 2026, 6:54 PM EDT