Condition

Hemophilia clinical trials and research

• 16Clinical trials
• 43NIH research projects
• 15Countries

Newest Hemophilia clinical trials

All recruiting trials (16)

• Personalized Hemophilia Care Through Telemedicine and Adherence Monitoring

  This study tests a telemedicine-based program to closely monitor bleeding, ensure patients stick to their treatment plan, and personalize care for adults with hemophilia who use a smartphone.

  Recruiting Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico · Milan · NCT07867834

• Checks how well the Figure-of-Eight Walk and Weight-Bearing Lunge tests measure movement in adults with hemophilia

  This observational study tests two movement tests to see if they reliably reflect mobility in adults with hemophilia who have a history of joint bleeding.

  Recruiting Yuzuncu Yil University · Van · NCT07851922

• Multimodal Telerehabilitation vs Face-to-Face Care for Adults with Hemophilia and Chronic Pain

  This trial tests whether a 6-week telerehabilitation program combining pain education and therapeutic exercise works as well as in-person care for adults with hemophilia and chronic pain.

  Recruiting University of Chile · Santiago, Santiago Metropolitan · NCT07682519

• PCC treatment for hemophilia A patients with inhibitors

  This trial tests whether giving prothrombin complex concentrate (PCC) during bleeding episodes can stop bleeds in people aged 12–65 with hemophilia A who have inhibitors.

  Recruiting Phase 4 Institute of Hematology & Blood Diseases Hospital, China · Tianjin, Tianjin Municipality · NCT07406139

• Fitusiran prophylaxis for boys aged 1 to under 12 with severe hemophilia A or B

  This study tests whether fitusiran given as prophylaxis can reduce bleeding in boys aged 1 to under 12 with severe hemophilia A or B, with or without inhibitors.

  Recruiting at 37 sites Phase 3 Sanofi · Los Angeles, California and 36 other locations · NCT07285460

• High-altitude stem cell and bone marrow transplant registry on the Qinghai-Tibet Plateau

  This project will collect clinical data and blood/bone marrow samples from people having hematopoietic stem cell transplants on the Qinghai‑Tibet Plateau to see how high altitude affects transplant outcomes and recovery.

  Recruiting Affiliated Hospital of Qinghai University · Xining, Qinghai · NCT07205523

• Monitoring anti‑TFPI antibodies in adult men with severe hemophilia

  This project tests blood-based lab methods to monitor anti-TFPI antibodies in adult men with severe hemophilia who are on prophylaxis.

  Recruiting Hospices Civils de Lyon · Bron and 1 other locations · NCT07101926

• Extended‑ vs short‑half‑life clotting factor prophylaxis and its impact on treatment burden and psychosocial wellbeing in adults with hemophilia

  This project will see if adults with hemophilia who use extended‑half‑life versus short‑half‑life prophylaxis feel a lower treatment burden and have better self‑efficacy, adherence, sleep, and health‑control beliefs.

  Recruiting Investigación en Hemofilia y Fisioterapia · Oviedo, Principality of Asturias · NCT07099313

• Marstacimab experiences in adolescents and adults with hemophilia

  This registry will collect experiences from adolescents and adults with hemophilia who are receiving marstacimab to see if they prefer subcutaneous injections and to track treatment burden and bleeding rates.

  Recruiting at 2 sites Institute of Hematology & Blood Diseases Hospital, China · Xiamen, Fujian and 1 other locations · NCT06992076

• ATHNdataset registry for people with bleeding and clotting disorders

  This registry collects health, treatment, and patient-reported information from people with bleeding and clotting disorders seen at ATHN-affiliated centers to help improve care and outcomes.

  Recruiting American Thrombosis and Hemostasis Network · Hickory, North Carolina · NCT06820515

• Embolization treatment for patients with hereditary bleeding disorders

  This study is testing whether a new treatment using tiny particles can help people with hereditary bleeding disorders and joint pain feel better and improve their quality of life.

  Recruiting University of Sao Paulo General Hospital · São Paulo · NCT05629130

• Long-term study of blood diseases in patients in China

  This study is trying to gather detailed information from patients in China with blood diseases like leukemia and multiple myeloma to better understand how these conditions affect their lives and treatment options.

  Recruiting Institute of Hematology & Blood Diseases Hospital, China · Tianjin, Tianjin Municipality · NCT04645199

• Long-term observation of non-cancer blood disorders

  This study is tracking people with blood disorders like hemophilia and thrombosis for at least 15 years to see how their conditions change over time and gather useful information for doctors and researchers.

  Recruiting at 74 sites American Thrombosis and Hemostasis Network · Phoenix, Arizona and 75 other locations · NCT04398628

• Understanding how drugs work in children

  This study is trying to find the best and safest doses of certain medications for children and young adults under 21 who are being treated for conditions like COVID-19 and pulmonary arterial hypertension.

  Recruiting at 5 sites Duke University · Phoenix, Arizona and 51 other locations · NCT04278404

• Collecting patient experiences and outcomes in hemophilia

  This study is trying to collect and compare experiences and outcomes from people with hemophilia across different countries to better understand their needs and challenges over time.

  Recruiting McMaster University · Hamilton, Ontario · NCT03549858

• Evaluating haemostatic methods for hemophilia patients with inhibitors

  This study is testing new bleeding management methods for people with hemophilia who have developed inhibitors to see if they work better than traditional treatments.

  Recruiting Karolinska Institutet · Solna, Stockholm County · NCT02453542

NIH-funded research (newest 40 of 43)

Early-stage science funded by the US National Institutes of Health. Most of these projects don't enroll patients, but they show what is being worked on.

• cGMP production of FVIII-making placental cells to help people with Hemophilia A

  A new, patient-friendly therapy aims to make FVIII-producing placental cells in the lab to help adults with Hemophilia A, potentially offering a longer-lasting and more affordable treatment.

  NIH research Wake Forest University Health Sciences · Winston-Salem, United States · NIH-11443970

• Enhancing immune regulation in gene therapy for hemophilia (new approaches to safer treatment)

  A patient-focused overview of a long-running NIH grant exploring how to make gene therapy for hemophilia safer by teaching the body’s immune system to tolerate the therapeutic protein.

  NIH research Indiana University Indianapolis · Indianapolis, United States · NIH-11416511

• A safer, longer-lasting gene delivery approach for Hemophilia A using improved nonviral vectors

  Researchers are testing a new type of nonviral gene delivery that could provide longer expression of a therapeutic gene for adults with Hemophilia A.

  NIH research M34 INC · Hillsborough, United States · NIH-11408326

• Non-viral gene therapy approach to permanently fix hemophilia B

  A new gene therapy approach aims to permanently correct hemophilia B without using viruses, potentially reducing bleeding events for people with this condition.

  NIH research Hydrogene Therapeutics INC · Houston, United States · NIH-11406850

• Hemophilia Point-of-Care Test Gets Regulatory Ready

  A new point-of-care test for hemophilia A measures FVIII and emicizumab to help doctors manage bleeding risk quickly, with plans to obtain regulatory clearance.

  NIH research DNA Medicine Institute · Cambridge, UNITED STATES · NIH-11405365

• Hemophilia A: studying why some people develop inhibitors to FVIII across families

  This NIH grant funds a multi-year program to follow families affected by severe hemophilia A to understand why some individuals develop antibodies against factor VIII.

  NIH research University of Washington · Seattle, United States · NIH-11405075

• Intraosseous delivery of gene therapy vectors for hemophilia A

  A gene therapy approach using vectors delivered into bone to help people with hemophilia A reduce or eliminate the need for frequent FVIII treatments.

  NIH research Seattle Children's Hospital · Seattle, United States · NIH-11389762

• Understanding why some hemophilia patients of Mexican descent develop antibodies to factor VIII

  Researchers are studying why people with severe hemophilia A who are of Mexican descent are more likely to develop inhibitors to standard factor VIII therapy, using genetic and environmental clues from a large patient group.

  NIH research University of Texas Rio Grande Valley · Edinburg, United States · NIH-11387778

• Targeting FVIII production to eye-opening blood vessels in hemophilia A

  Aims to improve treatment for people with hemophilia A by directing how the body makes clotting factor VIII, using a targeted gene-delivery approach.

  NIH research Children's Hosp of Philadelphia · Philadelphia, United States · NIH-11382316

• Platelet-targeted FVIII Gene Therapy for Hemophilia A

  A gene therapy approach aims to make platelets carry FVIII to help people with hemophilia A, even if they have inhibitors against FVIII.

  NIH research Medical College of Wisconsin · Milwaukee, United States · NIH-11377676

• Intergenerational Hemophilia A Program to Understand Factor VIII Immune Responses

  This program follows mothers who carry hemophilia A and their babies with severe hemophilia to learn how immune reactions to factor VIII develop in early life.

  NIH research University of Washington · Seattle, United States · NIH-11376434

• Hemophilia: a bedside monitor and app to track FVIII and emicizumab

  A new point-of-care device and app help people with hemophilia A quickly measure FVIII activity and emicizumab levels at home or in clinics.

  NIH research DNA Medicine Institute · Cambridge, UNITED STATES · NIH-11367348

• Gene editing for hemophilia A using targeted lipid nanoparticles

  A patient-friendly approach uses tiny lipid bubbles to deliver gene editing tools that fix the FVIII gene in the liver, aiming to reduce or eliminate the need for frequent FVIII infusions.

  NIH research Seattle Children's Hospital · Seattle, United States · NIH-11332298

• Safer gene therapy options for Hemophilia A

  Developing liver-directed gene therapy that is safer and keeps factor VIII working longer for people with Hemophilia A.

  NIH research Indiana University Indianapolis · Indianapolis, United States · NIH-11325354

• Understanding why some people with hemophilia develop harmful antibodies to replacement therapies

  Researchers are studying why some patients with hemophilia develop immune antibodies that block factor therapies and cause problems, with a focus on predicting who might be affected.

  NIH research Emory University · Atlanta, United States · NIH-11309332

• Boosting factor VIII production for people with hemophilia A

  This project tests small medicines that help certain faulty factor VIII genes produce more working protein for people with hemophilia A.

  NIH research Cleveland Clinic Lerner Com-Cwru · Cleveland, United States · NIH-11289337

• Liver-directed gene delivery to restore clotting factor in hemophilia B and other inherited liver diseases

  This research uses a liver-targeted viral gene delivery approach to help adults with hemophilia B produce the missing clotting protein and could be applied to other inherited liver disorders.

  NIH research Stanford University · Stanford, United States · NIH-11257294

• Targeted IL-2 therapy to reduce inhibitor antibodies in hemophilia

  This project uses a new IL-2–based immunocytokine (F5111) to boost regulatory immune cells and lower antibodies that block clotting factor treatment in people with hemophilia.

  NIH research Indiana University Indianapolis · Indianapolis, United States · NIH-11252575

• Improved AAV3 gene delivery for hemophilia

  This work develops better AAV3-based gene delivery to help people with hemophilia make missing clotting factors using lower doses and fewer side effects.

  NIH research University of Florida · Gainesville, United States · NIH-11248395

• Making gene therapy for hemophilia safer by improving immune control

  This project aims to reduce harmful immune reactions so gene therapy for people with hemophilia works better and lasts longer.

  NIH research Indiana University Indianapolis · Indianapolis, United States · NIH-11243917

• Creating a universal gene therapy for hemophilia A and B

  This study is testing a new gene therapy for people with hemophilia A and B to help their bodies make the clotting factors they need, aiming to provide a longer-lasting solution than the usual treatments, and it’s designed for both patients who have developed antibodies against these treatments and those who haven’t.

  NIH research Geneventiv Therapeutics, INC. · Raleigh, United States · NIH-11181888

• Why people with Hemophilia A of Mexican descent develop antibodies to factor VIII

  This project uses genetic and immune testing to learn why people with Hemophilia A of Mexican ancestry more often develop antibodies that block therapeutic factor VIII.

  NIH research University of Texas Rio Grande Valley · Edinburg, United States · NIH-11180308

• Improving Gene Therapy for Hemophilia A

  This project aims to make gene therapy safer and more effective for people with hemophilia A, potentially even before birth.

  NIH research Wake Forest University Health Sciences · Winston-Salem, United States · NIH-11169708

• Using gene editing and lipid nanoparticles to treat hemophilia A

  This study is testing a new way to treat hemophilia A by using tiny particles to deliver gene-editing tools that could fix the gene causing the bleeding disorder, aiming to offer a more lasting solution than the usual treatments.

  NIH research Seattle Children's Hospital · Seattle, United States · NIH-11163538

• A New Way to Deliver Factor VIII for Hemophilia A

  This research explores a new gene therapy approach for hemophilia A that uses platelets to deliver Factor VIII, especially for patients who develop antibodies against current treatments.

  NIH research Medical College of Wisconsin · Milwaukee, United States · NIH-11128549

• Creating a universal gene therapy for hemophilia A and B

  This study is testing a new gene therapy that could help people with hemophilia A and B by providing a long-lasting treatment, so they won’t need to get regular infusions of clotting factors anymore, especially for those who have trouble with traditional treatments.

  NIH research Geneventiv Therapeutics, INC. · Raleigh, United States · NIH-11073122

• Creating new treatments for hemophilia A by improving factor VIII production

  This study is looking at new ways to help people with hemophilia A by testing small drugs that can boost a key protein needed for blood clotting and exploring gene therapy options to provide longer-lasting solutions, making treatments easier and more affordable for patients.

  NIH research Cleveland Clinic Lerner Com-Cwru · Cleveland, United States · NIH-11047704

• New treatment to help hemophilia patients manage immune responses

  This study is looking at a new treatment that uses a special protein to help the immune system work better for people with hemophilia who have trouble with their clotting factor therapy, aiming to make their treatment easier and more effective.

  NIH research Indiana University Indianapolis · Indianapolis, United States · NIH-11041250

• Improving gene therapy for hemophilia using modified AAV3 vectors.

  This study is working on improving gene therapy for hemophilia A and B by creating better delivery systems that help get the right genes into liver cells, which could lead to safer and more effective treatments for people with these bleeding disorders.

  NIH research University of Florida · Gainesville, United States · NIH-11024765

• Improving gene therapy for hemophilia A

  This study is looking at ways to make gene therapy for hemophilia A safer and more effective by using a special virus to deliver the missing gene to the liver, so that patients can have a better and longer-lasting treatment.

  NIH research Indiana University Indianapolis · Indianapolis, United States · NIH-11023034

• Smart implants for delivering therapeutic proteins to treat hemophilia A

  This study is exploring a new type of implant that can safely deliver helpful proteins to people with hemophilia A, aiming to provide a long-lasting and effective treatment while keeping the body’s immune system from attacking it.

  NIH research Massachusetts Institute of Technology · Cambridge, United States · NIH-11014418

• Understanding joint bleeding in people with hemophilia

  This study looks at how bleeding in the joints affects how people with hemophilia move, using special technology to measure their movements, so we can help them make safer choices for staying active and reduce the chances of future bleeding.

  NIH research University of Colorado Denver · Aurora, UNITED STATES · NIH-11005040

• New strategies to prevent bleeding in people with hemophilia A

  This study is looking for better ways to help kids with hemophilia A avoid bleeding problems by exploring different treatment options, including new medicines and gene therapy, so they can find the safest and most effective choices for their needs.

  NIH research Yale University · New Haven, United States · NIH-10949401

• Monitoring blood clotting in hemophilia using sound technology

  This study is exploring a new way to check how well your blood clots using a tiny drop of blood, which could make it easier and more accurate for people with hemophilia to manage their treatment.

  NIH research Levisonics, INC. · New Orleans, United States · NIH-10922338

• Investigating immune responses in severe hemophilia A across generations

  This study is looking for mothers and their babies with severe hemophilia A to learn how babies' immune systems respond to a treatment called Factor VIII during their first two years of life, so we can better understand and help manage this condition.

  NIH research University of Washington · Seattle, United States · NIH-10916298

• Investigating bleeding risks in women who are carriers of Hemophilia A

  This study is looking at why some women who carry Hemophilia A experience unusual bleeding, even when their blood tests seem normal, and it aims to create a tool to help predict their bleeding risk so they can get better care.

  NIH research Pennsylvania State Univ Hershey Med Ctr · Hershey, United States · NIH-10907656

• Investigating genetic factors in hemophilia patients of Mexican descent

  This study is looking into why some people with severe hemophilia A, especially those of Mexican descent, develop antibodies that make their treatment less effective, and it aims to gather information and samples from these patients to help find better ways to treat them.

  NIH research University of Texas Rio Grande Valley · Edinburg, United States · NIH-10891149

• Investigating gene therapy for hemophilia A using liver-targeted AAV vectors

  This study is looking at a new gene therapy for hemophilia A that uses a special virus to help the liver produce the missing clotting factor, and it’s testing different ways to give this treatment safely to babies before they are born or shortly after.

  NIH research Wake Forest University Health Sciences · Winston-Salem, United States · NIH-10889145

• Gene therapy to induce immune tolerance in dogs with hemophilia A

  This study is testing a new gene therapy for dogs with hemophilia A who have developed issues with their treatment, aiming to help their bodies accept a key protein they need for blood clotting, which could make their care easier and less costly.

  NIH research Children's Hosp of Philadelphia · Philadelphia, United States · NIH-10851822

• Creating a new treatment to prevent complications in Hemophilia A

  This study is testing a new treatment for people with Hemophilia A that aims to help their bodies accept Factor VIII without creating harmful antibodies, making it safer and more effective than current options.

  NIH research State University of New York at Buffalo · Amherst, United States · NIH-10815822

Related topics

Quick answers

How many Hemophilia clinical trials are recruiting right now?

16, as of November 12, 2026. We update this list each time we refresh our data from ClinicalTrials.gov.

Where are these trials running?

In 15 countries. The most sites are in China (5 trials), United States (4 trials), Canada (3 trials), Turkey (Türkiye) (2 trials), Spain (2 trials). Each study page lists its sites and which ones are recruiting.

What phases are they in?

Phase 3: 1, Phase 4: 1. Trials with no phase listed are usually device, behavioural or observational studies.

How do I join one?

Open a study, check the "Is this trial a fit for you?" section, then contact the study team listed on the page. They will ask a few screening questions. Asking is free and does not commit you to anything. Talk it over with your doctor first.

Find the studies that fit you

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