Haemophilia trials in United States

In United States 42 Also abroad 128

42 trials · From ClinicalTrials.gov, checked 11 November 2026. Ask the study team whether a trial fits you.

Phase 4Taking people
Efanesoctocog Alfa Prophylaxis in Patients With Hemophilia A With Synovial Hypertrophy
Where
Columbus, Indianapolis, Iowa City, Los Angeles, Portland, Sacramento, Seattle
Age
12 years and over
People
35 worldwide
Run by
Sanofi
See details and how to joinNCT06941870
Phase 1/2Taking people
A Gene Therapy Study of SPK-8011QQ in Adults With Severe or Moderately Severe Hemophilia A
Where
Newark, New Haven, New York, Orange, San Francisco, Vallejo
Age
18 years and over
People
5
Run by
Hoffmann-La Roche
See details and how to joinNCT07226206
Phase 3Taking people
A Study to Assess the Safety of Marstacimab and How Well it Works in People With Mild to Moderate Hemophilia A or B Without Inhibitors
Where
Flowood
Age
12 years to 74 years
People
95
Run by
Pfizer
See details and how to joinNCT07923981
Phase 1/2Taking people
A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of NXT007 in Persons With Severe or Moderate Hemophilia A
Where
Indianapolis, Iowa City, Sacramento, Washington D.C.
Age
2 years to 59 years
People
60 worldwide
Run by
Hoffmann-La Roche
See details and how to joinNCT05987449 · EU 2023-503906-35-00
Phase 3Taking people
A Clinical Study to Evaluate the Effects of NXT007 Compared to Emicizumab Prophylaxis in People With Hemophilia A
Where
Ann Arbor, Aurora, Indianapolis, Iowa City, Miami, Minneapolis, Orange, San Antonio, Seattle, Tampa
Age
12 years and over
People
360 worldwide
Run by
Hoffmann-La Roche
See details and how to joinNCT07416604 · EU 2025-522435-33-00
Phase 3Taking people
A Research Study Investigating Denecimig (Mim8) in Adults With Acquired Haemophilia A
Where
Burlington, Chapel Hill, Charlottesville, Dallas, Durham, Hershey, Houston, Iowa City, Metairie, Minneapolis, Oklahoma City, Philadelphia, Richmond, Rochester, Salt Lake City, San Antonio, Seattle, St Louis
Age
18 years and over
People
84 worldwide
Run by
Novo Nordisk A/S
See details and how to joinNCT07830732 · EU 2026-525529-19-00
Phase 3Taking people
A Study to Investigate the Efficacy and Safety of Fitusiran Prophylaxis in Male Participants Aged 1 to Less Than 12 Years With Hemophilia A or B
Where
Ann Arbor, Dallas, Hackensack, Las Vegas, Los Angeles, Orange, Torrance
Age
1 year to 11 years
People
85 worldwide
Run by
Sanofi
See details and how to joinNCT07285460 · EU 2025-521858-42-00
Phase 3Taking people
Open-Label Extension Study of Marstacimab in Hemophilia Participants With or Without Inhibitors
Where
Iowa City, New Hyde Park, Philadelphia, Seattle
Age
1 year to 74 years
People
245 worldwide
Run by
Pfizer
See details and how to joinNCT05145127 · EU 2022-500470-33-00
No phaseTaking people
An Observational Study to Learn More About How Well Damoctocog Alfa Pegol Works in Previously Treated Children With Hemophilia A
Where
Loma Linda, New Brunswick
Age
7 years to 12 years
People
40 worldwide
Run by
Bayer
See details and how to joinNCT07088458
Phase 3Taking people
A Clinical Study to Evaluate the Effects of NXT007 Compared to Factor VIII Prophylaxis in Participants With Hemophilia A
Where
Aurora, Iowa City
Age
12 years and over
People
138 worldwide
Run by
Hoffmann-La Roche
See details and how to joinNCT07416526 · EU 2025-522434-32-00
No phaseNot yet taking people in United States
A Study to Describe Patient REal-world FitusiRan ExpeRience
Where
Indianapolis
Age
12 years and over
People
100
Run by
Sanofi
See details and how to joinNCT07895381
No phaseNot yet taking people in United States
Liver Biopsy Following Gene Therapy For Hemophilia
Where
Memphis
Age
18 years to 80 years
People
8
Run by
St. Jude Children's Research Hospital
See details and how to joinNCT05932914
Phase 1/2Taking people
A Study to Investigate the Safety and Effectiveness of a Coagulation Factor IX Gene Insertion Therapy (REGV131-LNP1265) in Pediatric, Adolescent and Adult Participants With Hemophilia B
Where
Ann Arbor, Aurora, Columbus, Dallas, Gainesville, Houston, Indianapolis, Los Angeles, Nashville, New Haven, New Orleans, Philadelphia, Phoenix, Sacramento, San Francisco
Age
2 years and over
People
130 worldwide
Run by
Regeneron Pharmaceuticals
See details and how to joinNCT06379789 · EU 2023-507260-40-00
Phase 3Taking people
Phase 3, Open-label, Single-dose Study of CSL222 in Adolescent Male Subjects (≥ 12 to < 18 Years of Age) With Severe or Moderately Severe Hemophilia B
Where
Ann Arbor, Atlanta, Gainesville, Memphis, Orange, San Diego
Age
138 months to 206 months
People
20 worldwide
Run by
CSL Behring
See details and how to joinNCT07080905 · EU 2023-505805-18-00
Phase 1Taking people
A Study to Learn About How Changing Therapy From Emicizumab to Marstacimab Affects People With the Severe Hemophilia A.
Where
Greenville, La Jolla, Los Angeles, San Diego
Age
12 years to 74 years
People
15 worldwide
Run by
Pfizer
See details and how to joinNCT06703606
Phase 3Taking people
A Clinical Trial of Study Medicine (Marstacimab) in Pediatric Patients With Hemophilia A or Hemophilia B
Where
Salt Lake City
Age
1 year to 17 years
People
100 worldwide
Run by
Pfizer
See details and how to joinNCT05611801 · EU 2022-500495-65-00
Phase 3Taking people
Safety and Effectiveness of Giroctocogene Fitelparvovec or Fidanacogene Elaparvovec in Patients With Hemophilia A or B Respectively
Where
Indianapolis, Madison, New York, Philadelphia, Rancho Cordova, Sacramento, San Francisco, Seattle, Tampa
Age
18 years and over
People
173 worldwide
Run by
Pfizer
See details and how to joinNCT05568719
No phaseTaking people
A Prospective Assessment of Bone Health in Patients With Severe Hemophilia A on Factor VIII vs Factor Mimetic Prophylaxis (Efa Emi Bone Health Study)
Where
Little Rock
Age
30 years to 50 years
People
50
Run by
Arkansas Children's Hospital Research Institute
See details and how to joinNCT07582276
No phaseTaking people
TG-INSIGHT With Joint POCUS, Hemostatic Potential in Patients With Severe Hemophilia A on Novel Replacement and Substitution FVIII Therapies
Where
Dallas
Age
6 months and over
People
60
Run by
University of Texas Southwestern Medical Center
See details and how to joinNCT07692217
Phase 4Taking people
Treatment of Hemophilia A Patients With FVIII Inhibitors
Where
Atlanta
People
120 worldwide
Run by
Emory University
See details and how to joinNCT04023019 · EU 2024-516741-39-00
Phase 4Taking people
A Study to Test a Medicine (Fitusiran) for Preventing Bleeds in People With Severe Hemophilia Who Previously Received Preventive Treatment With Emicizumab
Where
Flowood, Los Angeles
Age
12 years and over
People
20 worldwide
Run by
Sanofi
See details and how to joinNCT06145373 · EU 2022-502414-84-00
Phase 1Taking people
A Research Study Looking at How Different Doses of Study Medicine (Inno8) Work in the Body of People With Haemophilia A
Where
Aurora, Hershey, Indianapolis, Iowa City, Philadelphia
Age
18 years to 64 years
People
30 worldwide
Run by
Novo Nordisk A/S
See details and how to joinNCT07220564 · EU 2025-520490-38-00
Phase 3Taking people
Efficacy and Safety of CSL222 (Etranacogene Dezaparvovec) Gene Therapy in Adults With Hemophilia B With Pretreatment Adeno-associated Virus Serotype 5 (AAV5) Neutralizing Antibodies (Nabs)
Where
Ann Arbor, Houston, Pittsburgh, San Diego
Age
18 years and over
People
35 worldwide
Run by
CSL Behring
See details and how to joinNCT06003387 · EU 2023-509590-23-00
No phaseTaking people
Pharmacokinetics, Pharmacodynamics, and Safety Profile of Understudied Drugs Administered to Children Per Standard of Care (POPS)
Where
Albuquerque, Aurora, Austin, Baltimore, Boston, Chapel Hill, Charleston, Chicago, Cincinnati, Columbia, Dallas, Durham, Hershey, Honolulu, Houston, Indianapolis, Iowa City, Jackson, Jacksonville, Kansas City, Lebanon, Lexington, Little Rock, Los Angeles, Louisville, Madison, Milwaukee, Minneapolis, Morgantown, Nashville, New Orleans, Oklahoma City, Omaha, Palo Alto, Phoenix, Portland, Providence, Salt Lake City, Seattle, Sioux Falls, Wilmington
Age
0 years to 20 years
People
5,000 worldwide
Run by
Duke University
See details and how to joinNCT04278404
No phaseTaking people
ATHN Transcends: A Natural History Study of Non-Neoplastic Hematologic Disorders
Where
Akron, Albuquerque, Atlanta, Austin, Baltimore, Boston, Buffalo, Chapel Hill, Charlotte, Chicago, Cincinnati, Cleveland, Columbus, Dallas, Dayton, Detroit, Gainesville, Green Bay, Greenville, Hartford, Houston, Hyde Park, Indianapolis, Iowa City, Kansas City, Las Vegas, Little Rock, Los Angeles, Memphis, Miami, Milwaukee, Nashville, Newark, New Haven, New Orleans, New York, Norfolk, Oakland, Orlando, Peoria, Philadelphia, Phoenix, Pittsburgh, Providence, Reno, Rochester, Sacramento, San Antonio, San Bernardino, San Diego, San Francisco, Savannah, Scarborough, Seattle, St Louis, St. Petersburg, Tampa, The Bronx, Toledo, Washington D.C., Wilmington, Winston-Salem
People
3,000
Run by
American Thrombosis and Hemostasis Network
See details and how to joinNCT04398628