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ClinicalTrials.gov public records Last synced Sep 14, 2026, 8:53 AM EDT

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Showing 1–24 of 1,049 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Hereditary Anemias Clear all
Completed Phase 2Phase 3 Interventional Accepts healthy volunteers

The Effects of Erythropoietin (EPO) on the Transfusion Requirements of Very Low Birth Weight Infants

NCT01203514
View directory record Official record
Conditions
Infant, Newborn, Infant, Low Birth Weight, Infant, Small for Gestational Age, Infant, Premature, Anemia, Neonatal
Interventions
Erythropoietin, Sham Comparator
Drug · Other
Lead sponsor
NICHD Neonatal Research Network
Network
Eligibility
24 Hours to 96 Days
Enrollment
318 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
1997 – 2000
U.S. locations
9
States / cities
New Haven, Connecticut • Washington D.C., District of Columbia • Atlanta, Georgia + 6 more
Source: ClinicalTrials.gov public record
Updated Sep 25, 2017 · Synced Sep 14, 2026, 8:53 AM EDT
Completed Phase 2 Interventional Results available

A Study of the Effect of IW-1701 (Olinciguat), a Stimulator of Soluble Guanylate Cyclase (sGC), on Patients With Sickle Cell Disease (SCD)

NCT03285178
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
IW-1701, Placebo
Drug
Lead sponsor
Cyclerion Therapeutics
Industry
Eligibility
16 Years to 70 Years
Enrollment
88 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2020
U.S. locations
30
States / cities
Orange, California • Washington D.C., District of Columbia • Aventura, Florida + 23 more
Source: ClinicalTrials.gov public record
Updated Jul 20, 2023 · Synced Sep 14, 2026, 8:53 AM EDT
Completed Phase 2Phase 3 Interventional Results available

Stem Cell Transplant for Hemoglobinopathy

NCT00176852
View directory record Official record
Conditions
Sickle Cell Disease, Thalassemia, Severe Congenital Neutropenia, Diamond-Blackfan Anemia, Shwachman-Diamond Syndrome
Interventions
Busulfan, Fludarabine, ATG, TLI, Busulfan, Cyclophosphamide, ATG, GCSF, Campath, Fludarabine, Cyclophosphamide, Total Body Irradiation, Stem cell infusion
Drug · Radiation · Procedure
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
Up to 50 Years
Enrollment
22 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2002 – 2020
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Feb 26, 2020 · Synced Sep 14, 2026, 8:53 AM EDT
Completed No phase listed Observational

Pyruvate Kinase Deficiency Global Longitudinal Registry Substudy of Protocol AG348-C-008

NCT04995315
View directory record Official record
Conditions
Pyruvate Kinase Deficiency
Interventions
Not listed
Lead sponsor
Agios Pharmaceuticals, Inc.
Industry
Eligibility
18 Years and older
Enrollment
13 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2023
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Oct 15, 2023 · Synced Sep 14, 2026, 8:53 AM EDT
Completed Phase 1Phase 2 Interventional

Stem Cell Gene Therapy for Sickle Cell Disease

NCT02247843
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
βAS3-FB vector transduced peripheral blood CD34+ cells
Biological
Lead sponsor
Donald B. Kohn, M.D.
Other
Eligibility
18 Years and older
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2025
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Dec 16, 2025 · Synced Sep 14, 2026, 8:53 AM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

A Study to Evaluate GBT021601 in Single and Multiple Doses in Healthy Participants

NCT05036512
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
GBT021601
Drug
Lead sponsor
Pfizer
Industry
Eligibility
18 Years to 55 Years
Enrollment
129 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2020 – 2023
U.S. locations
1
States / cities
San Antonio, Texas
Source: ClinicalTrials.gov public record
Updated Jul 24, 2023 · Synced Sep 14, 2026, 8:53 AM EDT
Not listed No phase listed Observational

A Single-arm Safety Study of Transplantation Using Umbilical Cord Blood and Human Placental-derived Stem Cells From Partially Matched Related Donors in Persons With Certain Malignant Blood Diseases and Non-malignant Disorders

NCT00596999
View directory record Official record
Conditions
Hematologic Malignancies
Interventions
UCB and HPDSC
Procedure
Lead sponsor
Celgene Corporation
Industry
Eligibility
Up to 55 Years
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2013
U.S. locations
1
States / cities
New Orleans, Louisiana
Source: ClinicalTrials.gov public record
Updated Apr 9, 2012 · Synced Sep 14, 2026, 8:53 AM EDT
Completed Phase 1Phase 2 Interventional Results available

A Study to Assess the Safety, Tolerability, and Efficacy of ST-400 for Treatment of Transfusion-Dependent Beta-thalassemia (TDT)

NCT03432364
View directory record Official record
Conditions
Transfusion Dependent Beta-thalassemia
Interventions
ST-400 Investigational product
Genetic
Lead sponsor
Sangamo Therapeutics
Industry
Eligibility
18 Years to 40 Years
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2022
U.S. locations
6
States / cities
Los Angeles, California • Oakland, California • Atlanta, Georgia + 3 more
Source: ClinicalTrials.gov public record
Updated Dec 13, 2023 · Synced Sep 14, 2026, 8:53 AM EDT
Terminated Phase 2 Interventional Results available

Safety, Efficacy, Pharmacokinetic, and Pharmacodynamic Study of ALXN1820 in Adult Participants With Sickle Cell Disease

NCT05565092
View directory record Official record
Conditions
Sickle Cell Disease (SCD)
Interventions
ALXN1820
Drug
Lead sponsor
Alexion Pharmaceuticals, Inc.
Industry
Eligibility
18 Years and older
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2024
U.S. locations
2
States / cities
Hollywood, Florida • Indianapolis, Indiana
Source: ClinicalTrials.gov public record
Updated Jan 12, 2025 · Synced Sep 14, 2026, 8:53 AM EDT
Completed Not applicable Interventional Accepts healthy volunteers

Sequential vs. Standard Laser Treatment of Twin-twin Transfusion Syndrome

NCT02122328
View directory record Official record
Conditions
Twin Twin Transfusion Syndrome
Interventions
Sequential laser photocoagulation of communicating vessels., Selective laser photocoagulation of communicating vessels
Procedure
Lead sponsor
University of Southern California
Other
Eligibility
16 Years to 55 Years · Female only
Enrollment
642 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2010 – 2023
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Jul 19, 2025 · Synced Sep 14, 2026, 8:53 AM EDT
Completed No phase listed Observational

Identification of Early Predictors of Fetomaternal Hemorrhage

NCT01232387
View directory record Official record
Conditions
Fetomaternal Hemorrhage, Neonatal Anemia
Interventions
Not listed
Lead sponsor
Icahn School of Medicine at Mount Sinai
Other
Eligibility
Up to 60 Years
Enrollment
39 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2013
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Sep 19, 2013 · Synced Sep 14, 2026, 8:53 AM EDT
Active, not recruiting Early Phase 1 Interventional

Fludarabine Based RIC for Bone Marrow Failure Syndromes

NCT02928991
View directory record Official record
Conditions
Bone Marrow Failure Syndromes
Interventions
MRD-BMT with Fludarabine-based RIC for Acquired AA, MRD-BMT with Fludarabine-based RIC for iBMF with trilineage aplasia, MRD-BMT with Fludarabine-based RIC for iBMF without trilineage aplasia
Other
Lead sponsor
Children's Hospital of Philadelphia
Other
Eligibility
Up to 22 Years
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2028
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated May 27, 2026 · Synced Sep 14, 2026, 8:53 AM 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 5, 2019 · Synced Sep 14, 2026, 8:53 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Microvascular and Fibrosis Imaging Study

NCT01758250
View directory record Official record
Conditions
Autoimmune Diseases, Sickle Cell Disease, Chronic Graft Versus Host Disease
Interventions
Acoustic Radiation Force Impulse (ARFI), Laser Doppler Flowmetry (LDF), Laser Doppler Perfusion Imaging (LDI), Optical Coherence Tomography (OCT), Orthogonal Polarization Spectral Imaging (OPSI), Nail fold video capillaroscopy (NVC)
Other
Lead sponsor
Duke University
Other
Eligibility
18 Years and older
Enrollment
63 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2013 – 2018
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Sep 10, 2020 · Synced Sep 14, 2026, 8:53 AM EDT
Terminated Phase 2 Interventional

Effects of HQK-1001 in Patients With Sickle Cell Disease

NCT01601340
View directory record Official record
Conditions
Sickle Cell Disease, Sickle Cell Anemia, Sickle Cell Disorders, Hemoglobin S Disease, Sickling Disorder Due to Hemoglobin S
Interventions
HQK-1001, Placebo
Drug
Lead sponsor
HemaQuest Pharmaceuticals Inc.
Industry
Eligibility
12 Years to 60 Years
Enrollment
77 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2013
U.S. locations
11
States / cities
Mobile, Alabama • Oakland, California • Washington D.C., District of Columbia + 7 more
Source: ClinicalTrials.gov public record
Updated Mar 17, 2015 · Synced Sep 14, 2026, 8:53 AM EDT
Not yet recruiting Phase 1 Interventional

High-Dose Vitamin C in G6PDA and Pyruvate Kinase Deficiency: A Safety Study

NCT07612345
View directory record Official record
Conditions
Pyruvate Kinase Deficiency, Glucose 6 Phosphate Dehydrogenase Deficiency
Interventions
Vitamin C
Drug
Lead sponsor
University of Utah
Other
Eligibility
18 Years and older
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2027
U.S. locations
1
States / cities
Salt Lake City, Utah
Source: ClinicalTrials.gov public record
Updated May 27, 2026 · Synced Sep 14, 2026, 8:53 AM EDT
Terminated Not applicable Interventional

Donor Umbilical Cord Blood Transplant in Treating Patients With Hematologic Cancer

NCT00290628
View directory record Official record
Conditions
Chronic Myeloproliferative Disorders, Diamond-blackfan Anemia, Fanconi Anemia, Graft Versus Host Disease, Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm, Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Diseases
Interventions
anti-thymocyte globulin, busulfan, cyclophosphamide, cyclosporine, filgrastim, melphalan, methylprednisolone, mycophenolate mofetil, radiation therapy, umbilical cord blood transplantation
Drug · Procedure
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
Up to 45 Years
Enrollment
43 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1999 – 2007
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Nov 28, 2017 · Synced Sep 14, 2026, 8:53 AM EDT
Completed Not applicable Interventional Results available

iCanCope With Sickle Cell Pain

NCT03201874
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
Pain self-management, Education
Behavioral
Lead sponsor
Seattle Children's Hospital
Other
Eligibility
12 Years to 18 Years
Enrollment
137 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2022
U.S. locations
3
States / cities
Hartford, Connecticut • Atlanta, Georgia • Seattle, Washington
Source: ClinicalTrials.gov public record
Updated Dec 12, 2023 · Synced Sep 14, 2026, 8:53 AM EDT
Completed Phase 2 Interventional

Non-Ablative Allo HSCT For Hematologic Malignancies or SAA

NCT00006379
View directory record Official record
Conditions
Chronic Myeloproliferative Disorders, Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm, Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Diseases, Precancerous/Nonmalignant Condition, Small Intestine Cancer
Interventions
anti-thymocyte globulin, graft-versus-tumor induction therapy, cyclophosphamide, fludarabine phosphate, peripheral blood stem cell transplantation
Biological · Drug · Procedure
Lead sponsor
Case Comprehensive Cancer Center
Other
Eligibility
Up to 70 Years
Enrollment
58 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2000 – 2011
U.S. locations
1
States / cities
Cleveland, Ohio
Source: ClinicalTrials.gov public record
Updated Dec 7, 2011 · Synced Sep 14, 2026, 8:53 AM EDT
Recruiting No phase listed Observational

Comparing the Effectiveness of Matched Related Donor Hematopoietic Stem Cell Transplantation to Disease Modifying Therapy in Pediatric Patients With Sickle Cell Disease

NCT06941389
View directory record Official record
Conditions
Sickle Cell Disease (SCD)
Interventions
Not listed
Lead sponsor
University of Rochester
Other
Eligibility
3 Years to 20 Years
Enrollment
480 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2030
U.S. locations
31
States / cities
Birmingham, Alabama • Wilmington, Delaware • Washington D.C., District of Columbia + 19 more
Source: ClinicalTrials.gov public record
Updated Jun 3, 2026 · Synced Sep 14, 2026, 8:53 AM EDT
Completed Phase 1Phase 2 Interventional

Phase 1/2 Study to Evaluate the Safety, Tolerability and Pharmacokinetics of HQK-1001 Administered Daily in Patients With Sickle Cell Disease

NCT00842088
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
Placebo, HQK-1001
Drug
Lead sponsor
HemaQuest Pharmaceuticals Inc.
Industry
Eligibility
12 Years to 60 Years
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2009
U.S. locations
8
States / cities
Madison, Alabama • Oakland, California • Daytona Beach, Florida + 5 more
Source: ClinicalTrials.gov public record
Updated Jul 31, 2011 · Synced Sep 14, 2026, 8:53 AM EDT
Completed Phase 2 Interventional Results available

Prasugrel Versus Placebo in Adult Sickle Cell Disease

NCT01167023
View directory record Official record
Conditions
Sickle Cell Anemia
Interventions
Prasugrel, Placebo
Drug
Lead sponsor
Eli Lilly and Company
Industry
Eligibility
18 Years to 55 Years
Enrollment
62 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2011
U.S. locations
14
States / cities
Birmingham, Alabama • Little Rock, Arkansas • Sacramento, California + 11 more
Source: ClinicalTrials.gov public record
Updated May 2, 2012 · Synced Sep 14, 2026, 8:53 AM EDT
Completed Phase 2 Interventional Results available

Hydroxyurea to Prevent Brain Injury in Sickle Cell Disease

NCT01389024
View directory record Official record
Conditions
Sickle Cell Disease, Stroke
Interventions
Hydroxyurea, Placebo
Drug
Lead sponsor
Johns Hopkins University
Other
Eligibility
12 Months to 54 Months
Enrollment
28 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2022
U.S. locations
5
States / cities
Birmingham, Alabama • Baltimore, Maryland • Kansas City, Missouri + 2 more
Source: ClinicalTrials.gov public record
Updated Jul 9, 2024 · Synced Sep 14, 2026, 8:53 AM EDT
Completed Phase 1 Interventional Results available

Biotin-RBC Transfusion in SCD

NCT04426591
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
Biotin Labeled Red Blood Cells, Pathogen-reduced Biotin Labeled Red Blood Cells
Drug · Device
Lead sponsor
Marianne Yee
Other
Eligibility
2 Years to 65 Years
Enrollment
22 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2025
U.S. locations
3
States / cities
Atlanta, Georgia
Source: ClinicalTrials.gov public record
Updated May 21, 2026 · Synced Sep 14, 2026, 8:53 AM EDT