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Blood & Immune

Hemophilia A Clinical Trials and Treatments

Also called Factor VIII deficiency, classical hemophilia, X-linked hemophilia, Hemophilia Type A, Haemophilia A

Hemophilia A is an X-linked recessive inherited bleeding disorder resulting from deficiency or dysfunction of clotting factor VIII. Pathophysiology involves reduced or abnormal factor VIII protein function, which is critical for the intrinsic tenase complex in the coagulation cascade.

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About Hemophilia A

Hemophilia A is an X-linked recessive inherited bleeding disorder resulting from deficiency or dysfunction of clotting factor VIII. Pathophysiology involves reduced or abnormal factor VIII protein function, which is critical for the intrinsic tenase complex in the coagulation cascade.

The condition manifests with easy bruising, spontaneous bleeding into joints (hemarthrosis) and muscles (intramuscular hematomas), and spontaneous bleeding in severe forms. Clinical severity correlates directly with factor VIII levels: severe disease (<1% activity) causes spontaneous bleeding and hemarthrosis without provocation; moderate disease (1-5% activity) causes bleeding with minor trauma and significant joint problems; mild disease (5-40% activity) causes bleeding only with significant trauma or surgery. Hemarthrosis leads to progressive arthropathy (arthritis, joint destruction) if untreated, with knees, elbows, and ankles most commonly affected. Intracranial hemorrhage and other serious bleeds represent major causes of morbidity and mortality.

Diagnosis is confirmed by factor VIII activity assay showing reduced levels. Most hemophilia A cases requiring regular treatment tend to be severe or moderate; mild cases may remain undiagnosed until a surgical or traumatic challenge reveals the bleeding tendency.

Common Symptoms of Hemophilia A

Recognizing the signs of Hemophilia A early can lead to faster diagnosis and better outcomes. Symptoms may vary in severity from person to person. If you or a loved one are experiencing any of the following, consider speaking with a specialist.

  • Easy bruising and bleeding
  • Hemarthrosis (joint bleeds) causing pain and swelling
  • Muscle hematomas
  • Spontaneous bleeding in severe forms
  • Bleeding after trauma or surgery
  • Oral and GI bleeding

Who Hemophilia A Affects

Affects males predominantly due to X-linked inheritance; approximately 1 in 4,000-5,000 males worldwide carry the condition. Very rare in females, occurring only with homozygosity, compound heterozygosity, or skewed X-inactivation (lyonization).

Hemizygous males have severe disease; heterozygous females occasionally manifest mild-moderate bleeding from unfavorable lyonization. All ethnicities and racial groups affected with similar prevalence. No geographic variation in incidence. Approximately 30% of cases represent de novo mutations. Family history essential for identification of carriers.

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FDA-Approved Treatments for Hemophilia A

There are currently 4 FDA-approved medications for Hemophilia A. These therapies represent the current standard of care and may be used alongside or compared against investigational treatments in active clinical trials. Compare all 4 side by side, with dosing, trial results and warnings from the labels.

concizumab
Novo Nordisk
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emicizumab
Roche / Genentech
Official site
fitusiran
Sanofi / Alnylam
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Source: openFDA drug labeling data. This list may not include all treatments. Always consult your doctor.

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Help Paying for Hemophilia A Treatment

Charity funds and drugmaker programs for Hemophilia A, checked at the source. Pick your insurance to see what fits.

Your insurance
Charity funds
  • From a charity · Hemophilia Federation of America
    Helping Hands Program fund
    Open

    Pays for: Emergency living expenses ($250 prepaid card, once per year; referral through HTC or member organization; does not cover medical bills, copays or premiums), up to $250 per year.

    The foundation says: “The Helping Hands program is now open on a limited basis for 2026. Due to funding constraints, a maximum of 20 requests will be approved…”
  • From a charity · TotalAssist (formerly PAN Foundation)
    Hemophilia fund
    Open

    Pays for: Out-of-pocket costs for approved medications, up to $12,500 per year. Requires health insurance (any kind).

  • From a charity · The Assistance Fund
    Hemophilia fund
    Waitlist

    Pays for: Copays, coinsurance, deductibles and other health-related expenses.

    The foundation says: “WAITLIST — Accepting Waitlist Patients. TAF is currently accepting requests to join the enrollment waitlist for this program. Waitlists a…”
Status as each foundation showed it on September 28, 2026.

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Side Effect Explorer

Real-world side effect reports from the FDA Adverse Event Reporting System (FAERS). Includes both FDA-approved drugs and investigational therapies from active clinical trials. Click any drug to see what patients reported.

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Genetic Testing

Genetic testing can confirm a diagnosis, guide treatment decisions, and identify family members who may be at risk.

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Trusted Hemophilia A Resources

Reputable organizations and medical references for learning more about Hemophilia A, including disease registries, foundation resources, and clinical guidelines.

FDA decision ahead
The FDA is due to decide on Denecimig (Mim8) (Novo Nordisk) for Hemophilia A, with or without inhibitors in Q3 2026. Under-the-skin injection to prevent bleeding episodes in adults and children with hemophilia A, including people with inhibitors (antibodies that stop standard factor VIII replacement from working). It mimics the job of factor VIII, the clotting protein people with hemophilia A lack, and can be given once a month, every two weeks or weekly from a single-use prefilled pen. Novo submitted the application in September 2025 and, in its August 4, 2026 half-year report, listed a US decision as expected in Q3 2026 without an exact date. That quarter ended on September 30, 2026 with no announcement, so as of October 1 the decision is overdue against Novo's guidance; no PDUFA date was ever disclosed and Novo's next scheduled update is its nine-month report on November 4, 2026. In the pivotal FRONTIER 2 trial it significantly reduced bleeding rates compared with prior factor prophylaxis or on-demand treatment. Europe's CHMP recommended approval under the name Frehemgo on September 17, 2026.
See all upcoming rare disease FDA decisions →

Active Clinical Trials for Hemophilia A

Use this Hemophilia A clinical trial finder to see the 16 studies recruiting patients and 4 opening soon in the United States and worldwide, with eligibility criteria in plain English. These studies play a critical role in advancing care for blood & immune conditions and may offer access to treatments not yet widely available. Each trial below is sourced directly from ClinicalTrials.gov, with eligibility criteria translated into plain English to help patients and caregivers evaluate whether a study may be a fit.

TrialsSite mapPipeline timeline

Note: Trial recruitment statuses on ClinicalTrials.gov may not immediately reflect recent FDA decisions, sponsor announcements, or enrollment changes. Always confirm a trial's current status directly with the study coordinator before making plans.

116 active trials worldwide
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RECRUITINGPHASE3Recently updatedNCT05568719

Safety and Effectiveness of Giroctocogene Fitelparvovec or Fidanacogene Elaparvovec in Patients With Hemophilia A or B Respectively

Intervention: Testing of hepatic AAV Vector integration

Sponsor: Pfizer

A study to learn about the long-term safety and efficacy of giroctocogene fitelparvovec or fidanacogene elaparvovec in patients with hemophilia A or hemophilia B respectively, who have received treatment through prior participation in a Pfizer-sponsored clinical trial. Data colle...

Ages 18 Years+39 locations
Started Dec 2022Updated yesterdayEst. Feb 2040 (~13y 4m)
RECRUITINGRecently updatedNCT07692217

TG-INSIGHT With Joint POCUS, Hemostatic Potential in Patients With Severe Hemophilia A on Novel Replacement and Substitution FVIII Therapies

Intervention: Half-life factor VIII based replacement therapy, Non-FVIII based replacement therapy

Sponsor: University of Texas Southwestern Medical Center · Sanofi

This is an observational research study to find out if there is a difference in the way children with moderate or severe hemophilia A, treated on two different types of factor replacement, form a clot and also evaluate if they develop tiny bleeds within the joint and subsequently...

Ages 6 Months+1 location
Started Jul 2026Updated 4 days agoEst. May 2033 (~6y 8m)
RECRUITINGRecently updatedNCT04023019

Treatment of Hemophilia A Patients With FVIII Inhibitors

Intervention: Nuwiq, Octanate, Wilate, Emicizumab, Recombinant factor VIIa (rFVIIa), Activated prothrombin complex concentrate (aPCC)

Sponsor: Emory University · Octapharma

This is a non-interventional, multicenter, observational, international study in male persons with haemophilia A who have developed inhibitors to any replacement coagulation factor VIII (FVIII) product. The purpose of the study is to capture different approaches in the management...

Ages not specified2 locations
Started Mar 2020Updated 1 week agoEst. Dec 2028 (~2y 2m)
RECRUITINGPHASE3Recently updatedNCT07416526

A Clinical Study to Evaluate the Effects of NXT007 Compared to Factor VIII Prophylaxis in Participants With Hemophilia A

Intervention: NXT007, Human Coagulation Factor VIII

Sponsor: Hoffmann-La Roche · Chugai Pharmaceutical

The purpose of this study is to evaluate the efficacy, safety, pharmacokinetics and pharmacodynamics of NXT007 prophylaxis compared with Factor VIII (FVIII) prophylaxis in participants with severe or moderate congenital hemophilia A without FVIII inhibitors. The study will includ...

Ages 12 Years+32 locations
Started May 2026Updated 1 week agoEst. Sep 2027 (~11 months)
RECRUITINGPHASE3Recently updatedNCT05611801

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

Intervention: marstacimab

Sponsor: Pfizer

The purpose of this clinical trial is to learn about the safety and effects of the study medicine (called marstacimab) for the potential treatment of hemophilia in pediatric patients.

Ages 1 Year – 17 Years63 locations
Started Dec 2022Updated 1 week agoEst. Sep 2028 (~1y 11m)
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Active trial locations95 cities in the US
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Trial Pipeline

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Data from ClinicalTrials.gov, U.S. National Library of Medicine.
Always talk to your doctor before considering a clinical trial.

Patient Communities

Connect with other Hemophilia A patients, caregivers, and advocacy groups across Facebook groups, Reddit communities, and YouTube channels. These patient communities offer peer support, shared experiences, caregiver resources, and real-time discussion about Hemophilia A treatments, clinical trial participation, and day-to-day disease management.

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Related Blood & Immune Conditions

Other rare diseases in the blood & immune category. Patients with Hemophilia A may find relevant research, shared treatment pathways, or overlapping clinical trials among these related conditions.

Hemophilia A News and Analysis

Trial Friend articles about Hemophilia A, newest first

Companies Developing Hemophilia A Treatments

12 pharmaceutical companies have Hemophilia A in their rare disease portfolio

Frequently Asked Questions About Hemophilia A