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ClinicalTrials.gov public records Last synced Oct 8, 2026, 2:10 AM EDT

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Showing 25–48 of 122 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Severe Haemophilia A Clear all
Completed Phase 3 Interventional Results available

A Trial to Compare Prophylaxis Therapy to On-demand Therapy With a New Full Length Recombinant FVIII in Patients With Severe Hemophilia A

NCT01233258
View directory record Official record
Conditions
Hemophilia A
Interventions
rFVIII (BAY81-8973) on demand, rFVIII (BAY81-8973) prophylaxis low-dose, rFVIII (BAY81-8973) prophylaxis high-dose
Biological
Lead sponsor
Bayer
Industry
Eligibility
12 Years to 65 Years · Male only
Enrollment
80 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2012
U.S. locations
2
States / cities
Dayton, Ohio • Houston, Texas
Source: ClinicalTrials.gov public record
Updated Nov 28, 2016 · Synced Oct 8, 2026, 2:10 AM EDT
Completed Phase 1 Interventional

Trial to Evaluate the Pharmacokinetics and Safety Profile of BAY94-9027 Following Single and Multiple Dose Administration

NCT01184820
View directory record Official record
Conditions
Hemophilia A
Interventions
BAY94-9027 + Recombinant Factor VIII (Kogenate FS, BAY14-2222))
Biological
Lead sponsor
Bayer
Industry
Eligibility
18 Years to 65 Years · Male only
Enrollment
14 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2011
U.S. locations
4
States / cities
Davis, California • Boston, Massachusetts • Minneapolis, Minnesota + 1 more
Source: ClinicalTrials.gov public record
Updated Sep 7, 2018 · Synced Oct 8, 2026, 2:10 AM EDT
Recruiting Phase 1Phase 2 Interventional

A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of NXT007 in Persons With Severe or Moderate Hemophilia A

NCT05987449
View directory record Official record
Conditions
Hemophilia A
Interventions
NXT007
Drug
Lead sponsor
Hoffmann-La Roche
Industry
Eligibility
2 Years to 59 Years · Male only
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2033
U.S. locations
4
States / cities
Sacramento, California • Washington D.C., District of Columbia • Indianapolis, Indiana + 1 more
Source: ClinicalTrials.gov public record
Updated Sep 11, 2026 · Synced Oct 8, 2026, 2:10 AM EDT
Completed Not applicable Interventional

Rescue Immunotolerance Study in Induction of Immune Tolerance (ITI)-Experienced Patients (RES.I.S.T. Experienced)

NCT01051076
View directory record Official record
Conditions
Severe Hemophilia A
Interventions
VWF/FVIII concentrates
Drug
Lead sponsor
City of Hope Medical Center
Other
Eligibility
Male only
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2020
U.S. locations
1
States / cities
Duarte, California
Source: ClinicalTrials.gov public record
Updated Aug 23, 2021 · Synced Oct 8, 2026, 2:10 AM EDT
Terminated Phase 1 Interventional

Hemophilia B Gene Therapy With AAV8 Vector

NCT01620801
View directory record Official record
Conditions
Hemophilia B
Interventions
AAV8-hFIX19
Biological
Lead sponsor
Spark Therapeutics, Inc.
Industry
Eligibility
18 Years and older · Male only
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2016
U.S. locations
2
States / cities
Philadelphia, Pennsylvania • Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Mar 12, 2019 · Synced Oct 8, 2026, 2:10 AM 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 8, 2026, 2:10 AM EDT
Recruiting Phase 1 Interventional

Emicizumab for Severe Von Willebrand Disease (VWD) and VWD/Hemophilia A

NCT05500807
View directory record Official record
Conditions
Von Willebrand Disease, Type 3, Concomitant VWD and Hemophilia
Interventions
Emicizumab
Drug
Lead sponsor
Bleeding and Clotting Disorders Institute Peoria, Illinois
Other
Eligibility
0 Years to 90 Years
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2028
U.S. locations
12
States / cities
Orange, California • Redwood City, California • Coral Gables, Florida + 9 more
Source: ClinicalTrials.gov public record
Updated Apr 3, 2026 · Synced Oct 8, 2026, 2:10 AM EDT
Completed No phase listed Observational

Long-Term Safety, Tolerability, and Efficacy of DTX101 (AAVrh10FIX) in Adults With Moderate/Severe to Severe Hemophilia B

NCT02971969
View directory record Official record
Conditions
Hemophilia B
Interventions
Not listed
Lead sponsor
Ultragenyx Pharmaceutical Inc
Industry
Eligibility
18 Years and older · Male only
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2021
U.S. locations
3
States / cities
Little Rock, Arkansas • Gainesville, Florida • Ann Arbor, Michigan
Source: ClinicalTrials.gov public record
Updated Jan 6, 2022 · Synced Oct 8, 2026, 2:10 AM EDT
Recruiting Phase 1 Interventional

A Study to Learn About How Changing Therapy From Emicizumab to Marstacimab Affects People With the Severe Hemophilia A.

NCT06703606
View directory record Official record
Conditions
Severe Hemophilia A
Interventions
MARSTACIMAB
Drug
Lead sponsor
Pfizer
Industry
Eligibility
12 Years to 74 Years · Male only
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
3
States / cities
Los Angeles, California • Greenville, North Carolina
Source: ClinicalTrials.gov public record
Updated Jun 3, 2026 · Synced Oct 8, 2026, 2:10 AM EDT
Completed Phase 3 Interventional Results available

Study Comparing On-Demand Treatment With Two Prophylaxis Regimens Of BeneFIX In Patients With Severe Hemophilia B

NCT00364182
View directory record Official record
Conditions
Hemophilia B
Interventions
Recombinant Coagulation Factor IX (BeneFIX)
Drug
Lead sponsor
Pfizer
Industry
Eligibility
6 Years to 65 Years · Male only
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2010
U.S. locations
5
States / cities
Phoenix, Arizona • Chicago, Illinois • New Brunswick, New Jersey + 1 more
Source: ClinicalTrials.gov public record
Updated Oct 4, 2011 · Synced Oct 8, 2026, 2:10 AM EDT
Completed Phase 2 Interventional Results available

Rituximab to Treat Severe Hemophilia A

NCT00331006
View directory record Official record
Conditions
Hemophilia A
Interventions
Rituximab
Drug
Lead sponsor
Carelon Research
Other
Eligibility
18 Months and older
Enrollment
23 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2012
U.S. locations
13
States / cities
Orange, California • Atlanta, Georgia • Chicago, Illinois + 10 more
Source: ClinicalTrials.gov public record
Updated Jun 11, 2013 · Synced Oct 8, 2026, 2:10 AM EDT
Completed No phase listed Observational

Safety and Efficacy of Turoctocog Alfa During Long-Term Treatment of Severe and Moderately Severe Haemophilia A

NCT02035384
View directory record Official record
Conditions
Congenital Bleeding Disorder, Haemophilia A
Interventions
turoctocog alfa
Drug
Lead sponsor
Novo Nordisk A/S
Industry
Eligibility
Male only
Enrollment
69 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2020
U.S. locations
6
States / cities
Mobile, Alabama • Torrance, California • Peoria, Illinois + 3 more
Source: ClinicalTrials.gov public record
Updated Apr 27, 2020 · Synced Oct 8, 2026, 2:10 AM EDT
Completed Phase 3 Interventional Results available

A Study to Evaluate Prospective Efficacy and Safety Data of Current FIX Prophylaxis Replacement Therapy in Adult Hemophilia B Subjects (FIX:C≤2%) or Current FVIII Prophylaxis Replacement Therapy in Adult Hemophilia A Subjects (FVIII:C≤1%)

NCT03587116
View directory record Official record
Conditions
Hemophilia B, Hemophilia A
Interventions
Standard of Care FIX Replacement therapy, Standard of Care FVIII Replacement therapy
Drug
Lead sponsor
Pfizer
Industry
Eligibility
18 Years to 64 Years · Male only
Enrollment
212 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2024
U.S. locations
13
States / cities
Palo Alto, California • San Francisco, California • Stanford, California + 7 more
Source: ClinicalTrials.gov public record
Updated Nov 6, 2025 · Synced Oct 8, 2026, 2:10 AM EDT
Completed Phase 4 Interventional Results available

A Study to Evaluate Efficacy of rFVIIIFc for Immune Tolerance Induction (ITI) in Severe Hemophilia A Participants With Inhibitors Undergoing the First ITI Treatment (verITI-8 Study)

NCT03093480
View directory record Official record
Conditions
Hemophilia A With Inhibitors
Interventions
rFVIIIFc
Biological
Lead sponsor
Bioverativ, a Sanofi company
Industry
Eligibility
Male only
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2021
U.S. locations
12
States / cities
Orange, California • Aurora, Colorado • Washington D.C., District of Columbia + 9 more
Source: ClinicalTrials.gov public record
Updated Mar 28, 2022 · Synced Oct 8, 2026, 2:10 AM 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 8, 2026, 2:10 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 23, 2016 · Synced Oct 8, 2026, 2:10 AM EDT
Active, not recruiting Phase 3 Interventional Results available

A Study to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Subcutaneous Emicizumab in Participants From Birth to 12 Months of Age With Hemophilia A Without Inhibitors

NCT04431726
View directory record Official record
Conditions
Severe Hemophilia A
Interventions
Emicizumab
Drug
Lead sponsor
Hoffmann-La Roche
Industry
Eligibility
0 Months to 12 Months
Enrollment
55 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2030
U.S. locations
6
States / cities
Phoenix, Arizona • Los Angeles, California • Aurora, Colorado + 3 more
Source: ClinicalTrials.gov public record
Updated Oct 1, 2026 · Synced Oct 8, 2026, 2:10 AM EDT
Terminated Phase 1Phase 2 Interventional Results available

Safety & Efficacy of Encapsulated Allogeneic FVIII Cell Therapy in Haemophilia A

NCT04541628
View directory record Official record
Conditions
Hemophilia A
Interventions
SIG-001
Combination Product
Lead sponsor
Eli Lilly and Company
Industry
Eligibility
18 Years and older · Male only
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2023
U.S. locations
3
States / cities
Indianapolis, Indiana • Boston, Massachusetts • Seattle, Washington
Source: ClinicalTrials.gov public record
Updated Sep 4, 2024 · Synced Oct 8, 2026, 2:10 AM EDT
Completed Phase 3 Interventional Results available

An Open Label Study to Determine the Safety and Efficacy of Replacement Factor VIII Protein (Known as rFVIIIFc) in Previously Untreated Males With Severe Hemophilia A

NCT02234323
View directory record Official record
Conditions
Hemophilia A
Interventions
rFVIIIFc
Biological
Lead sponsor
Bioverativ, a Sanofi company
Industry
Eligibility
Up to 5 Years · Male only
Enrollment
108 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2019
U.S. locations
20
States / cities
Little Rock, Arkansas • Duarte, California • Sacramento, California + 16 more
Source: ClinicalTrials.gov public record
Updated Apr 11, 2022 · Synced Oct 8, 2026, 2:10 AM EDT
Completed Phase 3 Interventional Results available

Safety, Efficacy and PK of BIVV001 in Pediatric Patients With Hemophilia A

NCT04759131
View directory record Official record
Conditions
Hemophilia A
Interventions
efanesoctocog alfa (BIVV001)
Drug
Lead sponsor
Bioverativ, a Sanofi company
Industry
Eligibility
Up to 11 Years · Male only
Enrollment
74 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2023
U.S. locations
10
States / cities
Los Angeles, California • Gainesville, Florida • Atlanta, Georgia + 7 more
Source: ClinicalTrials.gov public record
Updated Sep 11, 2025 · Synced Oct 8, 2026, 2:10 AM EDT
Withdrawn Phase 3 Interventional

Study of a Gene Therapy Treatment for Hemophilia A

NCT06297486
View directory record Official record
Conditions
Hemophilia A
Interventions
SPK-8011
Genetic
Lead sponsor
Spark Therapeutics, Inc.
Industry
Eligibility
18 Years and older · Male only
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2035
U.S. locations
27
States / cities
Phoenix, Arizona • Loma Linda, California • Los Angeles, California + 23 more
Source: ClinicalTrials.gov public record
Updated Dec 13, 2024 · Synced Oct 8, 2026, 2:10 AM EDT
Completed Phase 1Phase 2 Interventional Results available

A Safety, Tolerability, and Pharmacokinetics Study of a Single Intravenous Injection of Recombinant Coagulation Factor VIII Fc - Von Willebrand Factor - XTEN Fusion Protein (rFVIIIFc-VWF-XTEN) (BIVV001) in Previously Treated Adults With Severe Hemophilia A (EXTEN-A)

NCT03205163
View directory record Official record
Conditions
Hemophilia A
Interventions
Advate (Low Dose), Advate (High Dose), BIVV001 (Low Dose), BIVV001 (High Dose)
Biological
Lead sponsor
Bioverativ, a Sanofi company
Industry
Eligibility
18 Years to 65 Years · Male only
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2018
U.S. locations
7
States / cities
Los Angeles, California • Indianapolis, Indiana • Iowa City, Iowa + 4 more
Source: ClinicalTrials.gov public record
Updated Apr 19, 2022 · Synced Oct 8, 2026, 2:10 AM EDT
Completed No phase listed Observational

Platelet Function in Patients With Hemophilia A

NCT02093065
View directory record Official record
Conditions
Hemophilia A
Interventions
Not listed
Lead sponsor
Boston Children's Hospital
Other
Eligibility
2 Years to 18 Years · Male only
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2020
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Jul 14, 2020 · Synced Oct 8, 2026, 2:10 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 8, 2026, 2:10 AM EDT