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ClinicalTrials.gov public records Last synced Oct 18, 2026, 2:11 AM EDT

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Showing 1–24 of 322 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Hemophilia A, Congenital Clear all
Active, not recruiting Phase 1Phase 2 Interventional

Open-Label Single Ascending Dose of Adeno-associated Virus Serotype 8 Factor IX Gene Therapy in Adults With Hemophilia B

NCT01687608
View directory record Official record
Conditions
Hemophilia B
Interventions
AskBio009
Biological
Lead sponsor
Baxalta now part of Shire
Industry
Eligibility
18 Years to 75 Years · Male only
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2030
U.S. locations
15
States / cities
Los Angeles, California • Sacramento, California • San Diego, California + 11 more
Source: ClinicalTrials.gov public record
Updated Mar 20, 2026 · Synced Oct 18, 2026, 2:11 AM EDT
Completed Phase 1 Interventional

Pharmacokinetics and Safety of a Single Intravenous Infusion of BAY 79-4980

NCT00629837
View directory record Official record
Conditions
Hemophilia A
Interventions
Recomb. Factor VIII (Kogenate FS Liposome, BAY79-4980), Recombinant Factor VIII (Kogenate FS, BAY14-2222)
Biological
Lead sponsor
Bayer
Industry
Eligibility
12 Years to 60 Years · Male only
Enrollment
27 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2006
U.S. locations
2
States / cities
Davis, California • Orange, California
Source: ClinicalTrials.gov public record
Updated Nov 18, 2014 · Synced Oct 18, 2026, 2:11 AM EDT
Terminated Phase 2 Interventional

A Study of SerpinPC in Participants with Hemophilia B (HemB) with Inhibitors

NCT05789537
View directory record Official record
Conditions
Hemophilia B with Inhibitor, Hemophilia B
Interventions
SerpinPC
Drug
Lead sponsor
ApcinteX Ltd
Industry
Eligibility
12 Years to 65 Years · Male only
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2025
U.S. locations
3
States / cities
Aurora, Colorado • Tampa, Florida • Greenville, North Carolina
Source: ClinicalTrials.gov public record
Updated Mar 11, 2025 · Synced Oct 18, 2026, 2:11 AM EDT
Not listed Not applicable Interventional

Comparison of Ideal vs. Actual Weight Base Factor Dosing

NCT03286153
View directory record Official record
Conditions
Hemophilia A
Interventions
Ideal Body Weight First, Actual Body Weight First
Other
Lead sponsor
Bloodworks
Other
Eligibility
12 Years and older · Male only
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2018
U.S. locations
4
States / cities
Portland, Oregon • Seattle, Washington • Spokane, Washington
Source: ClinicalTrials.gov public record
Updated Sep 18, 2017 · Synced Oct 18, 2026, 2:11 AM EDT
Completed No phase listed Observational

ATHN 2: Factor Switching Study

NCT02546622
View directory record Official record
Conditions
Hemophilia
Interventions
Factor VIII Replacement Products for Hemophilia which were FDA approved after January 1, 2013, Factor IX Replacement Products for Hemophilia which were FDA approved after January 1, 2013
Biological
Lead sponsor
American Thrombosis and Hemostasis Network
Network
Eligibility
Not listed
Enrollment
310 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2020
U.S. locations
27
States / cities
San Diego, California • Aurora, Colorado • New Haven, Connecticut + 23 more
Source: ClinicalTrials.gov public record
Updated Dec 9, 2021 · Synced Oct 18, 2026, 2:11 AM EDT
Completed Phase 4 Interventional

Study Evaluating ReFacto® in Hemophilia A Undergoing Major Surgery

NCT00092976
View directory record Official record
Conditions
Hemophilia A
Interventions
ReFacto
Drug
Lead sponsor
Wyeth is now a wholly owned subsidiary of Pfizer
Industry
Eligibility
6 Years and older · Male only
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2003 – 2007
U.S. locations
4
States / cities
Los Angeles, California • New Brunswick, New Jersey • Cincinnati, Ohio + 1 more
Source: ClinicalTrials.gov public record
Updated Dec 28, 2007 · Synced Oct 18, 2026, 2:11 AM EDT
Terminated Not applicable Interventional

Osteoporosis and MRI Study in Hemophilia

NCT01460147
View directory record Official record
Conditions
Bone Mineral Density, Hemophilia A, Hemophilia B
Interventions
DXA scan + MRI
Other
Lead sponsor
Baxalta now part of Shire
Industry
Eligibility
25 Years and older · Male only
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2012
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Apr 22, 2021 · Synced Oct 18, 2026, 2:11 AM EDT
Completed No phase listed Observational

Females With Severe or Moderate Hemophilia A or B: an International Multi-center Study

NCT00936312
View directory record Official record
Conditions
Hemophilia A, Hemophilia B
Interventions
Not listed
Lead sponsor
Weill Medical College of Cornell University
Other
Eligibility
Female only
Enrollment
200 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2011
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Dec 10, 2012 · Synced Oct 18, 2026, 2:11 AM EDT
Completed Phase 2 Interventional Results available

A Gene Therapy Study for Hemophilia B

NCT02484092
View directory record Official record
Conditions
Hemophilia B
Interventions
SPK-9001
Biological
Lead sponsor
Pfizer
Industry
Eligibility
18 Years and older · Male only
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2019
U.S. locations
9
States / cities
Sacramento, California • Jackson, Mississippi • Madison, Mississippi + 2 more
Source: ClinicalTrials.gov public record
Updated Jun 16, 2020 · Synced Oct 18, 2026, 2:11 AM EDT
Terminated Phase 3 Interventional Results available

Study of Modified Recombinant Factor VIII (OBI-1) in Subjects With Congenital Hemophilia A

NCT01434511
View directory record Official record
Conditions
Hemophilia A
Interventions
OBI-1
Biological
Lead sponsor
Baxalta now part of Shire
Industry
Eligibility
6 Years and older
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2013
U.S. locations
1
States / cities
Indianapolis, Indiana
Source: ClinicalTrials.gov public record
Updated May 14, 2021 · Synced Oct 18, 2026, 2:11 AM EDT
Completed Phase 3 Interventional Results available

Recombinant Factor VIIa BI (rFVIIa BI) Treatment of Acute Bleeding Episodes Per an On-demand Regimen

NCT01757405
View directory record Official record
Conditions
Hemophilia A, Hemophilia B
Interventions
Recombinant Factor VIIa BI (rFVIIa BI)
Biological
Lead sponsor
Baxalta now part of Shire
Industry
Eligibility
12 Years to 65 Years · Male only
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2014
U.S. locations
1
States / cities
Tampa, Florida
Source: ClinicalTrials.gov public record
Updated May 11, 2021 · Synced Oct 18, 2026, 2:11 AM EDT
Completed Phase 3 Interventional Results available

Study of the Efficacy and Safety PF-06741086 in Adult and Teenage Participants With Severe Hemophilia A or Moderately Severe to Severe Hemophilia B

NCT03938792
View directory record Official record
Conditions
Hemophilia A, Hemophilia B
Interventions
PF-06741086
Drug
Lead sponsor
Pfizer
Industry
Eligibility
12 Years to 74 Years · Male only
Enrollment
188 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2025
U.S. locations
4
States / cities
Tampa, Florida • Iowa City, Iowa • New Hyde Park, New York + 1 more
Source: ClinicalTrials.gov public record
Updated Aug 14, 2026 · Synced Oct 18, 2026, 2:11 AM EDT
Active, not recruiting Phase 1 Interventional

Gene Therapy Trial for Platelet Derived Factor VIII Production in Hemophilia A

NCT03818763
View directory record Official record
Conditions
Hemophilia A
Interventions
Auto CD34+PBSC, transduced with a lentiviral vector encoding the B domain deleted from of human coagulation factor VIII
Biological
Lead sponsor
Medical College of Wisconsin
Other
Eligibility
18 Years and older · Male only
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2033
U.S. locations
1
States / cities
Milwaukee, Wisconsin
Source: ClinicalTrials.gov public record
Updated Jul 13, 2026 · Synced Oct 18, 2026, 2:11 AM EDT
Completed Phase 3 Interventional Results available

A Study of Fitusiran (ALN-AT3SC) in Severe Hemophilia A and B Patients With Inhibitors

NCT03417102
View directory record Official record
Conditions
Hemophilia A, Hemophilia B
Interventions
fitusiran, Bypassing agents
Drug
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
12 Years and older · Male only
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2021
U.S. locations
12
States / cities
Phoenix, Arizona • Los Angeles, California • Orange, California + 9 more
Source: ClinicalTrials.gov public record
Updated Mar 28, 2022 · Synced Oct 18, 2026, 2:11 AM EDT
Completed Phase 3 Interventional Results available

Evaluating the Haemostatic Effect of NNC 0129-0000-1003 During Surgical Procedures in Subjects With Haemophilia A.

NCT01489111
View directory record Official record
Conditions
Congenital Bleeding Disorder, Haemophilia A
Interventions
turoctocog alfa pegol
Drug
Lead sponsor
Novo Nordisk A/S
Industry
Eligibility
12 Years and older · Male only
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2018
U.S. locations
19
States / cities
Phoenix, Arizona • Long Beach, California • Sacramento, California + 16 more
Source: ClinicalTrials.gov public record
Updated Aug 10, 2020 · Synced Oct 18, 2026, 2:11 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 23, 2024 · Synced Oct 18, 2026, 2:11 AM EDT
Terminated No phase listed Observational

Hemophilia Non-Interventional Study Prior to SerpinPC Intervention (PRESent-5)

NCT05605678
View directory record Official record
Conditions
Hemophilia a, Hemophilia B
Interventions
No Intervention
Other
Lead sponsor
ApcinteX Ltd
Industry
Eligibility
12 Years to 65 Years · Male only
Enrollment
108 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2024
U.S. locations
2
States / cities
Ann Arbor, Michigan • Greenville, South Carolina
Source: ClinicalTrials.gov public record
Updated Feb 7, 2025 · Synced Oct 18, 2026, 2:11 AM EDT
Completed Phase 3 Interventional Results available

A Study of Fitusiran (ALN-AT3SC) in Severe Hemophilia A and B Patients Without Inhibitors

NCT03417245
View directory record Official record
Conditions
Hemophilia A, Hemophilia B
Interventions
fitusiran, factor concentrates
Drug
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
12 Years and older · Male only
Enrollment
120 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2021
U.S. locations
8
States / cities
Little Rock, Arkansas • Gainesville, Florida • Tampa, Florida + 5 more
Source: ClinicalTrials.gov public record
Updated Mar 28, 2022 · Synced Oct 18, 2026, 2:11 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Biomarkers Serum Collection Methodology Pilot Study

NCT01318707
View directory record Official record
Conditions
Hemophilia A
Interventions
Not listed
Lead sponsor
Georgetown University
Other
Eligibility
18 Years to 75 Years
Enrollment
20 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2010 – 2011
U.S. locations
1
States / cities
Washington D.C., District of Columbia
Source: ClinicalTrials.gov public record
Updated Jul 24, 2012 · Synced Oct 18, 2026, 2:11 AM EDT
Completed Phase 3 Interventional Results available

A Multinational, Open-Label, Non-Controlled Trial on Safety, Efficacy and Pharmacokinetics of NNC 0129-0000-1003 in Previously Treated Paediatric Patients With Severe Haemophilia A

NCT01731600
View directory record Official record
Conditions
Congenital Bleeding Disorder, Haemophilia A
Interventions
turoctocog alfa pegol
Drug
Lead sponsor
Novo Nordisk A/S
Industry
Eligibility
0 Years to 11 Years · Male only
Enrollment
68 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2018
U.S. locations
24
States / cities
Phoenix, Arizona • Long Beach, California • Orange, California + 20 more
Source: ClinicalTrials.gov public record
Updated Nov 23, 2020 · Synced Oct 18, 2026, 2:11 AM EDT
Completed Phase 1 Interventional Results available

Pharmacokinetics of rFVIIIFc at Two Vial Strengths

NCT02083965
View directory record Official record
Conditions
Severe Hemophilia A
Interventions
rFVIIIFc
Biological
Lead sponsor
Bioverativ Therapeutics Inc.
Industry
Eligibility
12 Years and older · Male only
Enrollment
19 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2015
U.S. locations
3
States / cities
Los Angeles, California • Salt Lake City, Utah • Seattle, Washington
Source: ClinicalTrials.gov public record
Updated Dec 19, 2020 · Synced Oct 18, 2026, 2:11 AM EDT
Withdrawn Phase 3 Interventional

Hemophilia Inhibitor Clinical Trials (INHIBIT) Platform

NCT02196207
View directory record Official record
Conditions
Severe Hemophilia A
Interventions
Eloctate Prophylaxis, Emicizumab Prophylaxis, Eloctate ITI plus Emicizumab, Eloctate ITI
Drug
Lead sponsor
Margaret Ragni
Other
Eligibility
4 Months to 99 Years · Male only
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2027
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Aug 20, 2019 · Synced Oct 18, 2026, 2:11 AM EDT
Active, not recruiting Phase 3 Interventional Results available

Study to Evaluate the Efficacy and Safety of PF-07055480 / Giroctocogene Fitelparvovec Gene Therapy in Moderately Severe to Severe Hemophilia A Adults

NCT04370054
View directory record Official record
Conditions
Hemophilia A
Interventions
PF-07055480 (giroctocogene fitelparovec): Recombinant AAV2/6 Human Factor VIII Gene Therapy
Biological
Lead sponsor
Pfizer
Industry
Eligibility
18 Years to 64 Years · Male only
Enrollment
77 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2026
U.S. locations
19
States / cities
Mountain View, California • Oakland, California • Palo Alto, California + 5 more
Source: ClinicalTrials.gov public record
Updated Aug 19, 2026 · Synced Oct 18, 2026, 2:11 AM EDT
Completed Phase 2 Interventional

Efficacy of NovoSeven® in Bleeding Prophylaxis in Hemophilia

NCT00108758
View directory record Official record
Conditions
Congenital Bleeding Disorder, Haemophilia A With Inhibitors, Haemophilia B With Inhibitors
Interventions
activated recombinant human factor VII
Drug
Lead sponsor
Novo Nordisk A/S
Industry
Eligibility
2 Years and older · Male only
Enrollment
23 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2005
U.S. locations
13
States / cities
Berkeley, California • Los Angeles, California • Chicago, Illinois + 9 more
Source: ClinicalTrials.gov public record
Updated Jan 12, 2017 · Synced Oct 18, 2026, 2:11 AM EDT