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ClinicalTrials.gov public records Last synced Oct 5, 2026, 5:53 PM EDT

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Showing 1–24 of 45 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Severe Hemophilia B Clear all
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 5, 2026, 5:53 PM EDT
Terminated No phase listed Observational

The Hemophilia Ultrasound Project

NCT02807753
View directory record Official record
Conditions
Hemophilia A, Hemophilia B
Interventions
Ankle and Knee Ultrasound Joint Assessment, Ankle and Knee Magnetic Resonance Imaging
Other
Lead sponsor
University of Miami
Other
Eligibility
Up to 30 Months
Enrollment
18 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2020
U.S. locations
4
States / cities
Gainesville, Florida • Miami, Florida • Lexington, Kentucky + 1 more
Source: ClinicalTrials.gov public record
Updated Jan 22, 2021 · Synced Oct 5, 2026, 5:53 PM EDT
Terminated Phase 1 Interventional Results available

Ascending Dose Study of Genome Editing by Zinc Finger Nuclease Therapeutic SB-FIX in Subjects With Severe Hemophilia B

NCT02695160
View directory record Official record
Conditions
Hemophilia B
Interventions
SB-FIX
Biological
Lead sponsor
Sangamo Therapeutics
Industry
Eligibility
18 Years and older · Male only
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2021
U.S. locations
1
States / cities
Washington D.C., District of Columbia
Source: ClinicalTrials.gov public record
Updated Jul 19, 2024 · Synced Oct 5, 2026, 5:53 PM EDT
Completed No phase listed Observational

Global Epidemiologic Study of Preexisting Immunity to AAV in Adults With Severe Hemophilia

NCT03185897
View directory record Official record
Conditions
Hemophilia A, Hemophilia B
Interventions
Non-treatment, seroprevalence
Other
Lead sponsor
Baxalta now part of Shire
Industry
Eligibility
18 Years to 75 Years · Male only
Enrollment
242 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2021
U.S. locations
7
States / cities
Los Angeles, California • Aurora, Colorado • East Lansing, Michigan + 4 more
Source: ClinicalTrials.gov public record
Updated Oct 25, 2021 · Synced Oct 5, 2026, 5:53 PM EDT
Terminated Phase 1Phase 2 Interventional

Safety of a New Type of Treatment Called Gene Transfer for the Treatment of Severe Hemophilia B

NCT00076557
View directory record Official record
Conditions
Hemophilia B
Interventions
Adeno-Associated Viral with Human Factor IX
Genetic
Lead sponsor
Avigen
Industry
Eligibility
18 Years and older · Male only
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2004
U.S. locations
3
States / cities
Palo Alto, California • Philadelphia, Pennsylvania • Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Apr 4, 2007 · Synced Oct 5, 2026, 5:53 PM EDT
Terminated Phase 1Phase 2 Interventional

BeCoMe-9: A Clinical Study of BE-101 for the Treatment of Adults With Moderately Severe or Severe Hemophilia B

NCT06611436
View directory record Official record
Conditions
Hemophilia B, Hemophilia B, Moderately Severe or Severe
Interventions
BE-101
Drug
Lead sponsor
Be Biopharma
Industry
Eligibility
18 Years and older · Male only
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
5
States / cities
Davis, California • Washington D.C., District of Columbia • Ann Arbor, Michigan + 2 more
Source: ClinicalTrials.gov public record
Updated Jun 15, 2026 · Synced Oct 5, 2026, 5:53 PM EDT
Completed Phase 3 Interventional Results available

Study to Determine the Safety and Efficacy of rFIXFc in Previously Untreated Males With Severe Hemophilia B

NCT02234310
View directory record Official record
Conditions
Hemophilia B
Interventions
rFIXFc
Biological
Lead sponsor
Bioverativ, a Sanofi company
Industry
Eligibility
Up to 17 Years · Male only
Enrollment
33 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2019
U.S. locations
11
States / cities
Sacramento, California • Washington D.C., District of Columbia • Atlanta, Georgia + 8 more
Source: ClinicalTrials.gov public record
Updated Mar 25, 2022 · Synced Oct 5, 2026, 5:53 PM EDT
Recruiting Phase 3 Interventional

A Clinical Trial of Study Medicine (Marstacimab) in Pediatric Patients With Hemophilia A or Hemophilia B

NCT05611801
View directory record Official record
Conditions
Hemophilia A, Hemophilia B
Interventions
marstacimab
Drug
Lead sponsor
Pfizer
Industry
Eligibility
1 Year to 17 Years · Male only
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2028
U.S. locations
1
States / cities
Salt Lake City, Utah
Source: ClinicalTrials.gov public record
Updated Sep 23, 2026 · Synced Oct 5, 2026, 5:53 PM 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 5, 2026, 5:53 PM EDT
Completed Phase 1Phase 2 Interventional Results available

An Open-label Extension Study of an Investigational Drug, Fitusiran, in Patients With Moderate or Severe Hemophilia A or B

NCT02554773
View directory record Official record
Conditions
Hemophilia A, Hemophilia B
Interventions
Fitusiran (SAR439774)
Drug
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
18 Years and older · Male only
Enrollment
34 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2023
U.S. locations
2
States / cities
Ann Arbor, Michigan • Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jun 5, 2024 · Synced Oct 5, 2026, 5:53 PM EDT
Completed Phase 3 Interventional Results available

Study of Recombinant Factor IX Fc Fusion Protein (rFIXFc) in Participants With Hemophilia B

NCT01027364
View directory record Official record
Conditions
Severe Hemophilia B
Interventions
Factor IX (rFIXFc), rFIX
Drug
Lead sponsor
Bioverativ Therapeutics Inc.
Industry
Eligibility
12 Years and older · Male only
Enrollment
123 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2012
U.S. locations
9
States / cities
Phoenix, Arizona • Sacramento, California • Aurora, Colorado + 6 more
Source: ClinicalTrials.gov public record
Updated Dec 19, 2020 · Synced Oct 5, 2026, 5:53 PM EDT
Completed Phase 2 Interventional Results available

Dose Confirmation Trial of AAV5-hFIXco-Padua

NCT03489291
View directory record Official record
Conditions
Hemophilia B
Interventions
AAV5-hFIXco-Padua (AMT-061)
Genetic
Lead sponsor
CSL Behring
Industry
Eligibility
18 Years and older · Male only
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2023
U.S. locations
4
States / cities
Phoenix, Arizona • Sacramento, California • San Diego, California + 1 more
Source: ClinicalTrials.gov public record
Updated Jun 28, 2024 · Synced Oct 5, 2026, 5:53 PM EDT
Recruiting Phase 3 Interventional

Open-Label Extension Study of Marstacimab in Hemophilia Participants With or Without Inhibitors

NCT05145127
View directory record Official record
Conditions
Hemophilia A, Hemophilia B
Interventions
PF-06741086
Drug
Lead sponsor
Pfizer
Industry
Eligibility
1 Year to 74 Years · Male only
Enrollment
245 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2030
U.S. locations
4
States / cities
Iowa City, Iowa • New Hyde Park, New York • Philadelphia, Pennsylvania + 1 more
Source: ClinicalTrials.gov public record
Updated Jul 1, 2026 · Synced Oct 5, 2026, 5:53 PM EDT
Terminated Phase 3 Interventional Results available

Safety and Efficacy of FVIIa to Prevent Excessive Bleeding in Hemophilia Patients With Inhibitors Undergoing Major Surgery

NCT05695391
View directory record Official record
Conditions
Hemophilia
Interventions
Coagulation Factor VIIa (Recombinant)
Biological
Lead sponsor
Laboratoire français de Fractionnement et de Biotechnologies
Industry
Eligibility
12 Years to 65 Years · Male only
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2025
U.S. locations
5
States / cities
Los Angeles, California • Atlanta, Georgia • New Orleans, Louisiana + 2 more
Source: ClinicalTrials.gov public record
Updated Jul 31, 2026 · Synced Oct 5, 2026, 5:53 PM EDT
Completed Phase 2 Interventional Results available

PF-06741086 Long-term Treatment in Severe Hemophilia

NCT03363321
View directory record Official record
Conditions
Hemophilia A or B
Interventions
PF-06741086
Biological
Lead sponsor
Pfizer
Industry
Eligibility
12 Years to 74 Years · Male only
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2020
U.S. locations
1
States / cities
Aurora, Colorado
Source: ClinicalTrials.gov public record
Updated Jul 27, 2021 · Synced Oct 5, 2026, 5:53 PM EDT
Completed No phase listed Observational

Study to Describe the Allergic Reactions to Factor IX in Patients With Hemophilia B

NCT00195221
View directory record Official record
Conditions
Hemophilia B, Allergic Reactions
Interventions
Not listed
Lead sponsor
Wyeth is now a wholly owned subsidiary of Pfizer
Industry
Eligibility
Not listed
Enrollment
166 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2007
U.S. locations
15
States / cities
Phoenix, Arizona • Orange, California • Atlanta, Georgia + 12 more
Source: ClinicalTrials.gov public record
Updated Dec 5, 2007 · Synced Oct 5, 2026, 5:53 PM EDT
Completed Phase 1Phase 2 Interventional Results available

A Study of BAX 888 in Male Adults With Severe Hemophilia A

NCT03370172
View directory record Official record
Conditions
Hemophilia A
Interventions
BAX 888
Drug
Lead sponsor
Baxalta now part of Shire
Industry
Eligibility
18 Years to 75 Years · Male only
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2024
U.S. locations
8
States / cities
Phoenix, Arizona • Los Angeles, California • Sacramento, California + 5 more
Source: ClinicalTrials.gov public record
Updated Sep 11, 2025 · Synced Oct 5, 2026, 5:53 PM EDT
Completed No phase listed Observational

Females With Severe or Moderate Hemophilia A or B: A Multi-Center Study

NCT00936845
View directory record Official record
Conditions
Hemophilia A, Hemophilia B
Interventions
Not listed
Lead sponsor
Weill Medical College of Cornell University
Other
Eligibility
1 Month and older · Female only
Enrollment
22 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2010
U.S. locations
18
States / cities
Duarte, California • Aurora, Colorado • Washington D.C., District of Columbia + 15 more
Source: ClinicalTrials.gov public record
Updated Dec 10, 2012 · Synced Oct 5, 2026, 5:53 PM EDT
Completed Phase 1Phase 2 Interventional

Study to Test the Safety and How Well Patients With Severe Hemophilia A Respond to Treatment With BAY 2599023 (DTX 201), a Drug Therapy That Delivers a Healthy Version of the Defective Factor VIII Gene Into the Nucleus of Liver Cells Using an Altered, Non-infectious Virus (AAV) as a "Shuttle"

NCT03588299
View directory record Official record
Conditions
Hemophilia A
Interventions
BAY2599023 (DTX201)
Drug
Lead sponsor
Bayer
Industry
Eligibility
18 Years and older · Male only
Enrollment
11 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2026
U.S. locations
3
States / cities
Little Rock, Arkansas • Ann Arbor, Michigan • Madison, Wisconsin
Source: ClinicalTrials.gov public record
Updated Sep 23, 2026 · Synced Oct 5, 2026, 5:53 PM EDT
Completed Phase 3 Interventional Results available

Study Evaluating B-Domain Deleted Recombinant Factor VIII (BDDrFVIII, ReFacto AF) in Patients With Hemophilia A Undergoing Elective Major Surgery

NCT00243659
View directory record Official record
Conditions
Hemophilia A
Interventions
ReFacto AF
Biological
Lead sponsor
Wyeth is now a wholly owned subsidiary of Pfizer
Industry
Eligibility
12 Years and older · Male only
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2008
U.S. locations
8
States / cities
La Jolla, California • Detroit, Michigan • Chapel Hill, North Carolina + 4 more
Source: ClinicalTrials.gov public record
Updated Jun 3, 2011 · Synced Oct 5, 2026, 5:53 PM EDT
Terminated Phase 2 Interventional

The Efficacy and Safety of SerpinPC in Participants with Severe Hemophilia a or Moderately Severe to Severe Hemophilia B

NCT05789524
View directory record Official record
Conditions
Hemophilia a, Hemophilia B
Interventions
SerpinPC
Drug
Lead sponsor
ApcinteX Ltd
Industry
Eligibility
12 Years to 65 Years · Male only
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2025
U.S. locations
10
States / cities
Aurora, Colorado • Tampa, Florida • Indianapolis, Indiana + 7 more
Source: ClinicalTrials.gov public record
Updated Mar 11, 2025 · Synced Oct 5, 2026, 5:53 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 5, 2026, 5:53 PM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

A Phase 1 Study of an Investigational Drug, ALN-AT3SC, in Healthy Volunteers and Hemophilia A or B Patients

NCT02035605
View directory record Official record
Conditions
Hemophilia A, Hemophilia B
Interventions
ALN-AT3SC, Sterile Normal Saline (0.9% NaCl)
Drug
Lead sponsor
Sanofi
Industry
Eligibility
18 Years to 65 Years · Male only
Enrollment
51 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2014 – 2017
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Dec 19, 2020 · Synced Oct 5, 2026, 5:53 PM EDT
Completed Phase 3 Interventional Results available

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

NCT01425723
View directory record Official record
Conditions
Severe Hemophilia B
Interventions
rFIXFc
Biological
Lead sponsor
Bioverativ Therapeutics Inc.
Industry
Eligibility
Male only
Enrollment
120 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2017
U.S. locations
10
States / cities
Phoenix, Arizona • Sacramento, California • Aurora, Colorado + 7 more
Source: ClinicalTrials.gov public record
Updated Dec 19, 2020 · Synced Oct 5, 2026, 5:53 PM EDT