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ClinicalTrials.gov public records Last synced Sep 22, 2026, 1:58 AM EDT

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Showing 25–48 of 24 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Inherited Bleeding Disorders Clear all
Active, not recruiting No phase listed Observational

An Observational Study of Participants With Type 3 Von Willebrand Disease on Prophylactic Standard-of-Care Treatment

NCT06883240
View directory record Official record
Conditions
Von Willebrand Disease, Type 3
Interventions
Von Willebrand Factor Concentrates, Von Willebrand Factor Concentrates and Factor VIII Concentrates, Factor VIII Concentrates, Recombinant Activated Factor VII, Activated Prothrombin Complex Concentrate
Drug
Lead sponsor
Hoffmann-La Roche
Industry
Eligibility
2 Years and older
Enrollment
44 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2026
U.S. locations
3
States / cities
Sacramento, California • Minneapolis, Minnesota • St Louis, Missouri
Source: ClinicalTrials.gov public record
Updated Aug 9, 2026 · Synced Sep 22, 2026, 1:58 AM EDT
Completed Phase 3 Interventional Results available

A Study of PEGylated Recombinant Factor VIII (BAX855) in Previously Untreated Young Children With Severe Hemophilia A

NCT02615691
View directory record Official record
Conditions
Hemophilia A
Interventions
PEGylated Recombinant Factor VIII, ITI
Biological
Lead sponsor
Baxalta now part of Shire
Industry
Eligibility
Up to 5 Years
Enrollment
120 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2024
U.S. locations
20
States / cities
Phoenix, Arizona • Cupertino, California • Oakland, California + 17 more
Source: ClinicalTrials.gov public record
Updated Jul 27, 2025 · Synced Sep 22, 2026, 1:58 AM EDT
Recruiting Phase 3 Interventional

A Clinical Study to Evaluate the Effects of NXT007 Compared to Factor VIII Prophylaxis in Participants With Hemophilia A

NCT07416526
View directory record Official record
Conditions
Hemophilia A
Interventions
NXT007, Human Coagulation Factor VIII
Combination Product · Drug
Lead sponsor
Hoffmann-La Roche
Industry
Eligibility
12 Years and older
Enrollment
126 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2031
U.S. locations
2
States / cities
Aurora, Colorado • Iowa City, Iowa
Source: ClinicalTrials.gov public record
Updated Sep 8, 2026 · Synced Sep 22, 2026, 1:58 AM EDT
Terminated Phase 3 Interventional Results available

Study of Recombinant Factor IX Product, IB1001, in Previously Treated Pediatric Subjects With Hemophilia B

NCT01271868
View directory record Official record
Conditions
Hemophilia B
Interventions
IB1001
Biological
Lead sponsor
Medexus Pharma, Inc.
Industry
Eligibility
Up to 12 Years
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2016
U.S. locations
3
States / cities
Chicago, Illinois • Indianapolis, Indiana • Houston, Texas
Source: ClinicalTrials.gov public record
Updated Mar 14, 2021 · Synced Sep 22, 2026, 1:58 AM EDT
Completed Phase 3 Interventional

Personalized Information or Basic Information in Helping Patients Make Decisions About Participating in a Clinical Trial

NCT00750009
View directory record Official record
Conditions
Brain and Central Nervous System Tumors, Chronic Myeloproliferative Disorders, Leukemia, Lymphoma, Lymphoproliferative Disorder, Multiple Myeloma and Plasma Cell Neoplasm, Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Neoplasms, Unspecified Adult Solid Tumor, Protocol Specific
Interventions
educational intervention
Other
Lead sponsor
Case Comprehensive Cancer Center
Other
Eligibility
18 Years to 120 Years
Enrollment
583 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2012
U.S. locations
4
States / cities
Detroit, Michigan • Cleveland, Ohio • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jul 26, 2020 · Synced Sep 22, 2026, 1:58 AM EDT
Completed Phase 3 Interventional Results available

Long-Term Safety and Efficacy of rFVIIIFc in the Prevention and Treatment of Bleeding Episodes in Previously Treated Participants With Hemophilia A

NCT01454739
View directory record Official record
Conditions
Hemophilia A
Interventions
rFVIIIFc
Drug
Lead sponsor
Bioverativ Therapeutics Inc.
Industry
Eligibility
0 Years and older · Male only
Enrollment
240 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2017
U.S. locations
22
States / cities
Los Angeles, California • Orange, California • Sacramento, California + 17 more
Source: ClinicalTrials.gov public record
Updated Dec 18, 2020 · Synced Sep 22, 2026, 1:58 AM EDT
Completed Phase 3 Interventional

A Research Study Investigating Mim8 in Adults and Adolescents With Haemophilia A With or Without Inhibitors

NCT05053139
View directory record Official record
Conditions
Haemophilia A, Haemophilia A With Inhibitors
Interventions
NNC0365-3769 (Mim8)
Drug
Lead sponsor
Novo Nordisk A/S
Industry
Eligibility
12 Years and older
Enrollment
281 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2024
U.S. locations
34
States / cities
Phoenix, Arizona • Los Angeles, California • Aurora, Colorado + 28 more
Source: ClinicalTrials.gov public record
Updated Dec 7, 2025 · Synced Sep 22, 2026, 1:58 AM EDT
Active, not recruiting Phase 3 Interventional Results available

A Study to Evaluate the Efficacy and Safety of Factor IX Gene Therapy With PF-06838435 in Adult Males With Moderately Severe to Severe Hemophilia B

NCT03861273
View directory record Official record
Conditions
Hemophilia B
Interventions
PF-06838435/ fidanacogene elaparvovec
Biological
Lead sponsor
Pfizer
Industry
Eligibility
18 Years to 65 Years · Male only
Enrollment
51 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2031
U.S. locations
17
States / cities
San Francisco, California • Aurora, Colorado • Indianapolis, Indiana + 3 more
Source: ClinicalTrials.gov public record
Updated May 31, 2026 · Synced Sep 22, 2026, 1:58 AM EDT
Completed Phase 2 Interventional Results available

IL-11 in Women With Von Willebrand Disease and Refractory Menorrhagia

NCT00524342
View directory record Official record
Conditions
Von Willebrand Disease
Interventions
Oprelvekin, Interleukin 11, IL-11
Drug
Lead sponsor
Margaret Ragni
Other
Eligibility
18 Years to 45 Years · Female only
Enrollment
7 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2010
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jan 30, 2018 · Synced Sep 22, 2026, 1:58 AM EDT
Completed Phase 1Phase 2 Interventional Results available

Dose-finding Study of SPK-8016 Gene Therapy in Patients With Hemophilia A to Support Evaluation in Individuals With FVIII Inhibitors

NCT03734588
View directory record Official record
Conditions
Adeno-Associated Virus (AAV), Blood Coagulation Disorder, Blood Coagulation Disorders, Inherited, Coagulation Protein Disorders, Factor VIII (FVIII), Factor VIII (FVIII) Deficiency, Factor VIII (FVIII) Gene, Factor VIII (FVIII) Protein, Genetic Diseases, Inborn, Genetic Diseases, X-Linked, Gene Therapy, Gene Transfer, Hematologic Diseases, Hemorrhagic Disorders, Recombinant, Vector, Inhibitors
Interventions
SPK-8016
Genetic
Lead sponsor
Spark Therapeutics, Inc.
Industry
Eligibility
18 Years and older · Male only
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2023
U.S. locations
11
States / cities
Los Angeles, California • Peoria, Illinois • Ann Arbor, Michigan + 7 more
Source: ClinicalTrials.gov public record
Updated Feb 22, 2024 · Synced Sep 22, 2026, 1:58 AM EDT
Completed No phase listed Observational

Real World Use of Emicizumab in Infants and Children Ages 0-3 Years With Hemophilia A

NCT05248594
View directory record Official record
Conditions
Hemophilia A
Interventions
HEMLIBRA
Drug
Lead sponsor
Montefiore Medical Center
Other
Eligibility
Up to 36 Months
Enrollment
71 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2025
U.S. locations
10
States / cities
Hartford, Connecticut • New Haven, Connecticut • Lebanon, New Hampshire + 7 more
Source: ClinicalTrials.gov public record
Updated Sep 7, 2025 · Synced Sep 22, 2026, 1:58 AM EDT
Recruiting No phase listed Observational

Vasculitis Pregnancy Registry

NCT02593565
View directory record Official record
Conditions
Vasculitis, Behcet's Disease, CNS Vasculitis, Cryoglobulinemic Vasculitis, Eosinophilic Granulomatosis With Polyangiitis (EGPA), Churg-Strauss Syndrome (CSS), Granulomatosis With Polyangiitis (GPA), Wegener's Granulomatosis, IgA Vasculitis, Henoch-Schoenlein Purpura (HSP), Microscopic Polyangiitis (MPA), Polyarteritis Nodosa (PAN), Takayasu Arteritis (TAK), Urticarial Vasculitis, Systemic Vasculitis
Interventions
Online questionnaires
Other
Lead sponsor
University of Pennsylvania
Other
Eligibility
18 Years to 50 Years · Female only
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2027
U.S. locations
1
States / cities
Tampa, Florida
Source: ClinicalTrials.gov public record
Updated Jan 22, 2026 · Synced Sep 22, 2026, 1:58 AM EDT
Recruiting No phase listed Observational

ATHN Transcends: A Natural History Study of Non-Neoplastic Hematologic Disorders

NCT04398628
View directory record Official record
Conditions
Hematologic Disorder, Bleeding Disorder, Connective Tissue Disorder, Hemophilia, Thrombosis, Von Willebrand Diseases, Thrombophilia, Rare Bleeding Disorder, Platelet Disorder, Factor IX Deficiency, Factor VIII Deficiency, Thalassemia, Sickle Cell Disease
Interventions
Not listed
Lead sponsor
American Thrombosis and Hemostasis Network
Network
Eligibility
Not listed
Enrollment
3,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2035
U.S. locations
76
States / cities
Phoenix, Arizona • Little Rock, Arkansas • Los Angeles, California + 60 more
Source: ClinicalTrials.gov public record
Updated Aug 16, 2026 · Synced Sep 22, 2026, 1:58 AM EDT
Completed Phase 4 Interventional

Stress Ulcer Prophylaxis Versus Placebo in Critically Ill Infants With Congenital Heart Disease

NCT03667703
View directory record Official record
Conditions
Congenital Heart Disease, Upper Gastrointestinal Bleeding, Stress Ulcer, Infection
Interventions
Famotidine, Placebo
Drug
Lead sponsor
Boston Children's Hospital
Other
Eligibility
1 Minute to 12 Months
Enrollment
70 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2022
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Sep 20, 2022 · Synced Sep 22, 2026, 1:58 AM EDT
Completed Phase 2Phase 3 Interventional Results available

Combination Chemotherapy and Donor Stem Cell Transplant in Treating Patients With Aplastic Anemia or Hematologic Cancer

NCT00003816
View directory record Official record
Conditions
Chronic Myeloproliferative Disorders, Leukemia, Lymphoma, Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Diseases, Nonmalignant Neoplasm, Unspecified Adult Solid Tumor, Protocol Specific, Unspecified Childhood Solid Tumor, Protocol Specific
Interventions
anti-thymocyte globulin, busulfan, carboplatin, cyclophosphamide, etoposide, fludarabine phosphate, melphalan, thiotepa, total-body irradiation
Biological · Drug · Radiation
Lead sponsor
Roswell Park Cancer Institute
Other
Eligibility
4 Years to 70 Years
Enrollment
361 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1998 – 2019
U.S. locations
1
States / cities
Buffalo, New York
Source: ClinicalTrials.gov public record
Updated Aug 12, 2021 · Synced Sep 22, 2026, 1:58 AM EDT
Withdrawn Phase 3 Interventional

Study of Recombinant Factor IX Product, IB1001, in Previously Treated Subjects With Hemophilia B

NCT02048111
View directory record Official record
Conditions
Hemophilia B
Interventions
IB1001
Biological
Lead sponsor
Cangene Corporation
Industry
Eligibility
12 Years and older
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2015
U.S. locations
1
States / cities
Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated Aug 22, 2016 · Synced Sep 22, 2026, 1:58 AM EDT
Terminated Not applicable Interventional Results available

Trial of RiaSTAP Versus Cryoprecipitate to Lower Operative Transfusions

NCT02540434
View directory record Official record
Conditions
Coagulopathy
Interventions
ROTEM, Cryoprecipitate, RiaSTAP
Device · Other · Drug
Lead sponsor
Weill Medical College of Cornell University
Other
Eligibility
18 Years to 100 Years
Enrollment
19 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2017
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jan 14, 2019 · Synced Sep 22, 2026, 1:58 AM EDT
Active, not recruiting Phase 3 Interventional

A Research Study Looking at Long-term Treatment With Mim8 in People With Haemophilia A

NCT05685238
View directory record Official record
Conditions
Haemophilia A, Haemophilia A With Inhibitors
Interventions
Mim8
Drug
Lead sponsor
Novo Nordisk A/S
Industry
Eligibility
Not listed
Enrollment
451 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2028
U.S. locations
15
States / cities
Los Angeles, California • Aurora, Colorado • Miami, Florida + 11 more
Source: ClinicalTrials.gov public record
Updated Sep 8, 2026 · Synced Sep 22, 2026, 1:58 AM EDT
Completed No phase listed Observational

Registry Study of COAGADEX® Patients With Moderate or Severe Hereditary Factor X Deficiency Undergoing Major Surgery

NCT03161626
View directory record Official record
Conditions
Factor 10 Deficiency
Interventions
Coagadex
Drug
Lead sponsor
Bio Products Laboratory
Other
Eligibility
12 Years and older
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2021
U.S. locations
3
States / cities
Gainesville, Florida • New Orleans, Louisiana • Kalamazoo, Michigan
Source: ClinicalTrials.gov public record
Updated Nov 11, 2021 · Synced Sep 22, 2026, 1:58 AM EDT
Completed No phase listed Observational

Socialization of Adult Men With Congenital Hemophilia A or B

NCT01510418
View directory record Official record
Conditions
Hemophilia A, Hemophilia B
Interventions
Not listed
Lead sponsor
Dana-Farber Cancer Institute
Other
Eligibility
21 Years and older
Enrollment
27 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2013
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Aug 2, 2016 · Synced Sep 22, 2026, 1:58 AM EDT
Recruiting Phase 2 Interventional

Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT

NCT01962415
View directory record Official record
Conditions
Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes, Inherited Metabolic Disorders (IMD), Hereditary Anemias, Inflammatory Conditions, Systemic Juvenile Idiopathic Arthritis (sJIA), Juvenile Rheumatoid Arthritis (JRA)
Interventions
Hydroxyurea, Alemtuzumab, Fludarabine, Melphalan, Thiotepa
Drug
Lead sponsor
Paul Szabolcs
Other
Eligibility
2 Months to 55 Years
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2027
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Dec 14, 2025 · Synced Sep 22, 2026, 1:58 AM EDT
Completed Phase 2 Interventional Results available

Timolol Gel for Epistaxis in Hereditary Hemorrhagic Telangiectasia

NCT04139018
View directory record Official record
Conditions
Hereditary Hemorrhagic Telangiectasia
Interventions
Timolol Gel, Placebo Gel
Drug
Lead sponsor
Washington University School of Medicine
Other
Eligibility
20 Years and older
Enrollment
27 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2020
U.S. locations
1
States / cities
St Louis, Missouri
Source: ClinicalTrials.gov public record
Updated Aug 23, 2021 · Synced Sep 22, 2026, 1:58 AM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Pharmacokinetics, Pharmacodynamics, and Safety Profile of Understudied Drugs Administered to Children Per Standard of Care (POPS)

NCT04278404
View directory record Official record
Conditions
Coronavirus Infection (COVID-19), Pulmonary Arterial Hypertension, Urinary Tract Infections in Children, Hypertension, Pain, Hyperphosphatemia, Primary Hyperaldosteronism, Edema, Hypokalemia, Heart Failure, Hemophilia, Menorrhagia, Insomnia, Pneumonia, Skin Infection, Arrythmia, Asthma in Children, Bronchopulmonary Dysplasia, Adrenal Insufficiency, Fibrinolysis; Hemorrhage, Attention Deficit Hyperactivity Disorder, Multisystem Inflammatory Syndrome in Children (MIS-C), Kawasaki Disease, Coagulation Disorder, Down Syndrome
Interventions
The POP02 study is collecting bodily fluid samples (i.e., whole blood, effluent samples) of children prescribed the following drugs of interest per standard of care:
Drug
Lead sponsor
Duke University
Other
Eligibility
0 Years to 20 Years
Enrollment
5,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2020 – 2026
U.S. locations
49
States / cities
Phoenix, Arizona • Little Rock, Arkansas • Los Angeles, California + 40 more
Source: ClinicalTrials.gov public record
Updated Aug 20, 2026 · Synced Sep 22, 2026, 1:58 AM EDT
Recruiting Phase 3 Interventional

Phase 3 Study on the Efficacy and Safety of Human Plasma Derived Antithrombin (Atenativ) in Heparin-Resistant Patients Scheduled to Undergo Cardiac Surgery Necessitating Cardiopulmonary Bypass

NCT06096116
View directory record Official record
Conditions
Acquired Antithrombin Deficiency
Interventions
Human plasma derived antithrombin, Placebo
Drug
Lead sponsor
Octapharma
Industry
Eligibility
18 Years to 85 Years
Enrollment
120 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2028
U.S. locations
9
States / cities
Stanford, California • Miami, Florida • Boston, Massachusetts + 6 more
Source: ClinicalTrials.gov public record
Updated May 11, 2026 · Synced Sep 22, 2026, 1:58 AM EDT