RecruitingPhase 3NCT07416604
Blood
Hemophilia A
Also known as Factor VIII deficiency, classic hemophilia, F8 deficiency
Hemophilia A is the most common severe inherited coagulation disorder, caused by deficiency of clotting factor VIII. Severe disease presents with spontaneous bleeding into joints and muscles; moderate and mild forms bleed with injury or sur
ORPHA:98878 ↗Gene F8Prevalence 1-9 per 100,000 (Orphanet)Onset Neonatal, Infancy, ChildhoodGenetic (X-linked recessive)
82
studies recruiting now
as of 7 Sept 2026
933
studies registered in total
as of 7 Sept 2026
21
countries with a recruiting site
as of 7 Sept 2026
26 Aug 2026
most recent study posted
among recruiting studies
Recruiting trials
RecruitingPhase 1 / Phase 2NCT07787091
A First-in Human Study of ARD001 in Healthy Volunteers and People With Hemophilia A
RecruitingPhase 3NCT04563520
SAFE Study: Safety of aPCC Following Emicizumab Prophylaxis
RecruitingNCT04278404
Pharmacokinetics, Pharmacodynamics, and Safety Profile of Understudied Drugs Administered to Children Per Standard of Care (POPS)
RecruitingNCT04398628
ATHN Transcends: A Natural History Study of Non-Neoplastic Hematologic Disorders
Showing the 5 most recently updated recruiting studies, as recorded 7 Sept 2026. Live status on each study page.
See all 82 recruiting studiesWhere recruiting studies are running
Countries with at least one recruiting site among the studies above, 7 Sept 2026. Tap a country to search trials there.
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About Hemophilia A
Hemophilia A is the most common severe inherited coagulation disorder, caused by deficiency of clotting factor VIII. Severe disease presents with spontaneous bleeding into joints and muscles; moderate and mild forms bleed with injury or surgery. Emicizumab (Hemlibra) and gene therapy (valoctocogene roxaparvovec) have dramatically changed treatment. Inhibitor development (antibodies against factor replacement) is a major complication driving clinical trial interest.
Common clinical features
Reduced factor VIII activityBleeding with minor or no traumaJoint hemorrhageArthralgiaSpontaneous hematomasOral cavity bleedingProlonged bleeding timeThromboembolism
From Orphanet’s phenotype annotations (CC BY 4.0). Not a complete list.
Treatments being studied
20 approved treatments and 15 in clinical development, from Open Targets (CC BY 4.0). Not medical advice.
Approved: Damoctocog Alfa Pegol (Jivi)Approved: Susoctocog Alfa (Obizur)Approved: Marstacimab (Hympavzi)Approved: Turoctocog Alfa Pegol (Esperoct)Approved: Efmoroctocog Alfa (Elocta)Approved: Valoctocogene Roxaparvovec (Roctavian)Approved: Concizumab (Alhemo)Approved: Emicizumab (Hemlibra)Approved: Moroctocog Alfa (Refacto af)Approved: Human Coagulation Factor Viii (Hemofil m)Approved: Eptacog Beta (Activated) (Cevenfacta)Approved: Von Willebrand Factor Human (Von willebrand factor human component of voncento)Approved: Simoctocog Alfa (Nuwiq)Approved: Turoctocog Alfa (Novoeight)Approved: Antihemophilic Factor, Pegylated (Mw 20000) Human Sequence Recombinant (Adynovi)Approved: Tranexamic Acid (Cyklo-f heavy period relief)Approved: Efanesoctocog Alfa (Altuviiio)Approved: Eptacog Alfa (Activated) (Novoseven)Approved: Octocog Alfa (Advate)Approved: Lonoctocog Alfa (Afstyla)
Phase 3Denecimig
Phase 3Giroctocogene Fitelparvovec
Phase 3Fitusiran
Phase 3Anti-Inhibitor Coagulant Complex
Phase 3Vatreptacog Alfa (Activated)
Phase 3Omfiloctocog Alfa
Phase 2/3Thrombin
Phase 2Ataluren (Translarna)
+ 7 more in development
Before you apply
Things trial teams commonly ask about for Hemophilia A. Not eligibility rules; those are set by each study.
- Severity (severe below 1%, moderate 1-5%, mild 5-40% factor VIII activity) is the primary eligibility criterion
- Inhibitor status (presence or absence of anti-factor antibodies) defines major trial categories
- Prior gene therapy exposure may exclude from new gene therapy trials - confirm before applying
Related conditions
Information, not medical advice. Trial listings are shown as recorded on ClinicalTrials.gov; whether any study is right for you is a decision for you and your clinicians, and eligibility is decided by each research team. Disease information from Orphanet (CC BY 4.0).