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ClinicalTrials.gov public records Last synced Oct 14, 2026, 12:43 PM EDT

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Showing 169–192 of 2,013 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Inherited Hematological Diseases Clear all
Completed Phase 3 Interventional Results available

A Study to Learn How Safe the Study Treatment BAY94-9027 is and How it Affects the Body in Previously Treated Children Aged 7 to Less Than 12 Years With Severe Hemophilia A, a Genetic Bleeding Disorder That is Caused by the Lack of a Protein Called Clotting Factor 8 (FVIII) in the Blood

NCT05147662
View directory record Official record
Conditions
Treatment of Bleeding, Prophylaxis of Bleeding, Hemophilia A, Children
Interventions
Damoctocog alfa pegol (Jivi, BAY94-9027)
Biological
Lead sponsor
Bayer
Industry
Eligibility
7 Years to 11 Years · Male only
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2025
U.S. locations
1
States / cities
Orlando, Florida
Source: ClinicalTrials.gov public record
Updated Jul 2, 2026 · Synced Oct 14, 2026, 12:43 PM 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 4, 2026 · Synced Oct 14, 2026, 12:43 PM EDT
Active, not recruiting Not applicable Interventional

Hydrops: Diagnosing & Redefining Outcomes With Precision Study

NCT03412760
View directory record Official record
Conditions
Hydrops Fetalis, Birth Defect, Fetal Anomaly
Interventions
Exome sequencing
Diagnostic Test
Lead sponsor
University of California, San Francisco
Other
Eligibility
18 Years to 55 Years
Enrollment
500 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2027
U.S. locations
1
States / cities
San Francisco, California
Source: ClinicalTrials.gov public record
Updated Apr 17, 2026 · Synced Oct 14, 2026, 12:43 PM EDT
Enrolling by invitation Phase 1Phase 2 Interventional

The BENeFiTS Trial in Beta Thalassemia Intermedia

NCT04432623
View directory record Official record
Conditions
Beta Thalassemia Intermedia, Sickle Cell Disease
Interventions
Benserazide Only Product
Drug
Lead sponsor
Phoenicia BioScience
Other
Eligibility
18 Years and older
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2026
U.S. locations
4
States / cities
Oakland, California • Boston, Massachusetts • Weston, Massachusetts + 1 more
Source: ClinicalTrials.gov public record
Updated Aug 15, 2025 · Synced Oct 14, 2026, 12:43 PM EDT
Completed Phase 2 Interventional Results available

Oral Pirfenidone for the Pulmonary Fibrosis of Hermansky-Pudlak Syndrome

NCT00001596
View directory record Official record
Conditions
Albinism, Inborn Errors of Metabolism, Oculocutaneous Albinism, Platelet Storage Pool Deficiency, Pulmonary Fibrosis
Interventions
Pirfenidone, Placebo
Drug
Lead sponsor
William Gahl, M.D.
NIH
Eligibility
18 Years and older
Enrollment
35 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2016
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 16, 2017 · Synced Oct 14, 2026, 12:43 PM EDT
Completed Phase 4 Interventional Accepts healthy volunteers Results available

Transfusion of Pathogen Reduced Cryoprecipitated Fibrinogen to Expedite Product Availability in Perioperative Bleeding

NCT05711524
View directory record Official record
Conditions
Hypofibrinogenemia, Bleeding
Interventions
Traditional Cryoprecipitate, Pathogen-Reduced Cryoprecipitate
Biological
Lead sponsor
Weill Medical College of Cornell University
Other
Eligibility
18 Years and older
Enrollment
208 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2023 – 2025
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Nov 21, 2025 · Synced Oct 14, 2026, 12:43 PM EDT
Not yet recruiting Phase 4 Interventional

Low Dose Bolus Ketamine For Use In Sickle Cell Pain Crisis

NCT07682662
View directory record Official record
Conditions
Vaso-Occlusive Pain Episode in Sickle Cell Disease, Sickle Cell Disease (SCD)
Interventions
Standardized Low-dose bolus IV Ketamine within 30 minutes of arrival and every 1 hour as needed for max of 3 doses., Standard Care (in control arm)
Drug · Other
Lead sponsor
University of Mississippi Medical Center
Other
Eligibility
2 Years to 21 Years
Enrollment
400 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2029
U.S. locations
1
States / cities
Jackson, Mississippi
Source: ClinicalTrials.gov public record
Updated Jul 6, 2026 · Synced Oct 14, 2026, 12:43 PM EDT
Terminated Phase 2 Interventional

Cilengitide in Treating Patients With Acute Myeloid Leukemia

NCT00089388
View directory record Official record
Conditions
Adult Acute Basophilic Leukemia, Adult Acute Eosinophilic Leukemia, Adult Acute Megakaryoblastic Leukemia (M7), Adult Acute Minimally Differentiated Myeloid Leukemia (M0), Adult Acute Monoblastic Leukemia (M5a), Adult Acute Monocytic Leukemia (M5b), Adult Acute Myeloblastic Leukemia Without Maturation (M1), Adult Acute Myeloid Leukemia in Remission, Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities, Adult Acute Myeloid Leukemia With t(15;17)(q22;q12), Adult Erythroleukemia (M6a), Adult Pure Erythroid Leukemia (M6b)
Interventions
cilengitide
Drug
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
50 Years and older
Enrollment
70 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2004
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Jan 24, 2013 · Synced Oct 14, 2026, 12:43 PM EDT
Completed No phase listed Observational

Study of Melphalan Drug Exposure in Pediatric Hematopoietic Stem Cell Transplant Patients

NCT03609827
View directory record Official record
Conditions
Hematologic Malignancies, Nonmalignant Diseases, Immunodeficiencies, Hemoglobinopathies, Genetic Inborn Errors of Metabolism, Fanconi's Anemia, Thalassemia, Sickle Cell Disease
Interventions
Melphalan
Drug
Lead sponsor
University of California, San Francisco
Other
Eligibility
Up to 17 Years
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2021
U.S. locations
1
States / cities
San Francisco, California
Source: ClinicalTrials.gov public record
Updated Oct 4, 2021 · Synced Oct 14, 2026, 12:43 PM EDT
Completed Phase 2Phase 3 Interventional

Safety and Efficacy Study of a Recombinant and Protein-Free Factor VIII (rAHF-PFM) in Hemophilia A Patients Undergoing Surgery

NCT00157105
View directory record Official record
Conditions
Hemophilia A
Interventions
Antihemophilic factor, recombinant, manufactured protein-free
Drug
Lead sponsor
Baxalta now part of Shire
Industry
Eligibility
5 Years and older · Male only
Enrollment
59 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2004
U.S. locations
10
States / cities
Los Angeles, California • Atlanta, Georgia • Indianapolis, Indiana + 7 more
Source: ClinicalTrials.gov public record
Updated Aug 24, 2021 · Synced Oct 14, 2026, 12:43 PM EDT
Completed Phase 2 Interventional Results available

Matched Unrelated or Non-Genotype Identical Related Donor Transplantation For Chronic Granulomatous Disease

NCT00578643
View directory record Official record
Conditions
Chronic Granulomatous Disease
Interventions
Busulfan, Alemtuzumab, Cyclophosphamide, Fludarabine, Cyclosporine, Stem Cell Infusion
Drug · Biological · Procedure
Lead sponsor
Baylor College of Medicine
Other
Eligibility
Not listed
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2017
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Nov 9, 2018 · Synced Oct 14, 2026, 12:43 PM EDT
Recruiting Not applicable Interventional

Trans-auricular Nerve Stimulation as an Innovative Approach to the Treatment of Pain in Pediatric Patients Suffering From Sickle Cell Disease

NCT07090668
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
tAN device
Device
Lead sponsor
Emory University
Other
Eligibility
7 Years to 18 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2026
U.S. locations
1
States / cities
Atlanta, Georgia
Source: ClinicalTrials.gov public record
Updated Jan 26, 2026 · Synced Oct 14, 2026, 12:43 PM EDT
Active, not recruiting Phase 2 Interventional Accepts healthy volunteers

Pilot Study of Reduced-Intensity Hematopoietic Stem Cell Transplant of DOCK8 Deficiency

NCT01176006
View directory record Official record
Conditions
DOCK8 Deficiency
Interventions
Reduced-intensity hematopoietic stem cell, Fludarabine(Fludara, Berlex Laboratories), Cyclophosphamide(CTX, Cytoxan), Total Body Irradiation (TBI), Busulfan (Busulfex), Donor peripheral blood stem cell mobiliation and collection, Bone Marrow Harvest Procedure
Procedure · Drug
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
4 Years to 120 Years
Enrollment
70 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2010 – 2028
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Sep 28, 2026 · Synced Oct 14, 2026, 12:43 PM EDT
Completed Phase 3 Interventional Results available

Pharmacokinetic, Efficacy, and Safety Study of Recombinant Factor VIII Single Chain (rVIII-SingleChain) in Children With Severe Hemophilia A

NCT02093897
View directory record Official record
Conditions
Congenital Hemophilia A
Interventions
rVIII-SingleChain
Biological
Lead sponsor
CSL Behring
Industry
Eligibility
Up to 11 Years · Male only
Enrollment
84 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2015
U.S. locations
2
States / cities
Aurora, Colorado • Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated Jan 27, 2017 · Synced Oct 14, 2026, 12:43 PM EDT
Completed No phase listed Observational

Eligibility Screening for a NCI Pediatric Oncology Branch Research Study

NCT00026780
View directory record Official record
Conditions
Ewing Sarcoma, Osteosarcoma, Neuroblastoma, Acute Lymphoblastic Leukemia, Neurofibromatosis Type 1
Interventions
Not listed
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
Up to 35 Years
Enrollment
1,720 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2020
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 8, 2020 · Synced Oct 14, 2026, 12:43 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

Erythroferrone and Its Impact on Maternal and Neonatal Iron Homeostasis

NCT04517734
View directory record Official record
Conditions
Pregnancy Anemia, Newborn; Anemia
Interventions
Not listed
Lead sponsor
Cornell University
Other
Eligibility
11 Years to 45 Years · Female only
Enrollment
338 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2006 – 2017
U.S. locations
2
States / cities
Rochester, New York
Source: ClinicalTrials.gov public record
Updated Mar 22, 2022 · Synced Oct 14, 2026, 12:43 PM EDT
Completed Phase 3 Interventional Results available

A Study of Fitusiran in Severe Hemophilia A and B Patients Previously Receiving Factor or Bypassing Agent Prophylaxis

NCT03549871
View directory record Official record
Conditions
Hemophilia
Interventions
Fitusiran, BPA prophylaxis, Factor (FVIII or FIX) prophylaxis
Drug
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
12 Years and older · Male only
Enrollment
80 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2022
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Feb 6, 2023 · Synced Oct 14, 2026, 12:43 PM EDT
Terminated Phase 2 Interventional Results available

A Study of the Effect of Blood Stem Cell Transplant After Chemotherapy Alone in Patients With Fanconi Anemia

NCT03600909
View directory record Official record
Conditions
Fanconi Anemia, Myelodysplastic Syndrome (MDS), Acute Myelogenous Leukemia (AML)
Interventions
Busulfan, Fludarabine, Cyclophosphamide, Anti-Thymocyte Globulin (Rabbit), The CliniMACS device, G-CSF
Drug · Device
Lead sponsor
Memorial Sloan Kettering Cancer Center
Other
Eligibility
1 Month and older
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2021
U.S. locations
3
States / cities
New York, New York • Cincinnati, Ohio • Seattle, Washington
Source: ClinicalTrials.gov public record
Updated Apr 12, 2022 · Synced Oct 14, 2026, 12:43 PM EDT
Completed Phase 1 Interventional

Temozolomide and VNP40101M in Treating Patients With Relapsed or Refractory Leukemias

NCT00098436
View directory record Official record
Conditions
Leukemia
Interventions
laromustine, temozolomide
Drug
Lead sponsor
Vion Pharmaceuticals
Industry
Eligibility
18 Years and older
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2008
U.S. locations
4
States / cities
Indianapolis, Indiana • Durham, North Carolina • Cleveland, Ohio
Source: ClinicalTrials.gov public record
Updated Jul 18, 2013 · Synced Oct 14, 2026, 12:43 PM EDT
Not listed No phase listed Observational Accepts healthy volunteers

300 Antibody Diagnostic Test Kit

NCT01646411
View directory record Official record
Conditions
Acute Bacterial Infections, Acute Viral Infections
Interventions
Not listed
Lead sponsor
The Plasma Source
Industry
Eligibility
18 Years to 70 Years
Enrollment
300 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2012 – 2013
U.S. locations
1
States / cities
Southampton, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jul 20, 2012 · Synced Oct 14, 2026, 12:43 PM EDT
Completed Phase 3 Interventional Results available

Study to Investigate Immunogenicity, Efficacy and Safety of Treatment With Human-cl rhFVIII

NCT01992549
View directory record Official record
Conditions
Severe Hemophilia A
Interventions
Human-cl rhFVIII
Biological
Lead sponsor
Octapharma
Industry
Eligibility
Male only
Enrollment
48 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2018
U.S. locations
1
States / cities
Sacramento, California
Source: ClinicalTrials.gov public record
Updated Jan 19, 2021 · Synced Oct 14, 2026, 12:43 PM EDT
Recruiting Not applicable Interventional

Complex Aortic Aneurysm Repair Using Physician Modified Endografts and Custom Made Devices

NCT02050113
View directory record Official record
Conditions
Complex Aortic Aneurysms, Thoracoabdominal Aneurysms, Pararenal Aneurysms, Juxtarenal Aneurysms, Marfan Syndrome, Ehlers-Danlos Syndrome, Loeys-Dietz Syndrome, Aortic Arch Aneurysm
Interventions
Custom Made Fenestrated or Branched Stent Graft
Device
Lead sponsor
Andres Schanzer
Other
Eligibility
18 Years and older
Enrollment
600 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2030
U.S. locations
1
States / cities
Worcester, Massachusetts
Source: ClinicalTrials.gov public record
Updated Oct 22, 2025 · Synced Oct 14, 2026, 12:43 PM EDT
Completed No phase listed Observational

Curative Versus Disease-Modifying Therapies in Children With Severe Sickle Cell Disease

NCT01369160
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
Quality of Life measures
Behavioral
Lead sponsor
Emory University
Other
Eligibility
3 Years to 23 Years
Enrollment
33 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2014
U.S. locations
2
States / cities
Atlanta, Georgia
Source: ClinicalTrials.gov public record
Updated May 28, 2014 · Synced Oct 14, 2026, 12:43 PM EDT
Terminated Phase 2Phase 3 Interventional Results available

T-Cell Depletion and Stem Cell Transplant for Immune Deficiencies and Histiocytic Disorders

NCT00176826
View directory record Official record
Conditions
Hemophagocytic Lymphohistiocytosis, X-Linked Lymphoproliferative Disorders, Chediak-Higashi Syndrome, Griscelli Syndrome, Immunologic Diseases, Langerhans-Cell Histiocytosis, Hematologic Diseases
Interventions
Stem Cell Transplant, Myeloablative conditioning regimen
Procedure · Drug
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
Up to 55 Years
Enrollment
22 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2000 – 2015
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Jan 23, 2018 · Synced Oct 14, 2026, 12:43 PM EDT