Condition

Hemophilia A clinical trials and research

• 35Clinical trials
• 27NIH research projects
• 43Countries

Newest Hemophilia A clinical trials

All recruiting trials (35)

• First‑in‑human test of ARD001 in healthy adults and people with hemophilia A

  This study tests ARD001 to see if it is safe and tolerable in healthy adults and in people with hemophilia A, using a single dose (Phase 1) and potentially repeated doses (Phase 2) if results look good.

  Recruiting Phase 1/2 Arditus Pte Ltd · Singapore · NCT07787091

• Hemophilia A: comparing long-acting FVIII replacement vs non-FVIII therapy in kids

  This study tests whether children with moderate to severe Hemophilia A on long-acting FVIII or non-FVIII therapies form clots differently and develop small joint bleeds or early joint changes.

  Recruiting University of Texas Southwestern Medical Center · Dallas, Texas · NCT07692217

• SR604: a new treatment tested in adults with Hemophilia A/B or congenital Factor VII deficiency

  This study tests a single and multiple doses of SR604 to see if it is safe and effective for adults with Hemophilia A or B, with or without inhibitors, and for those with congenital Factor VII deficiency.

  Recruiting at 7 sites Phase 1/2 Shanghai RAAS Blood Products Co., Ltd. · Changsha and 8 other locations · NCT07644832

• Bone health in severe hemophilia A: emicizumab vs efanesoctocog alfa

  This study tests whether bone density and bone health markers differ in adults with severe hemophilia A who use emicizumab versus efanesoctocog alfa for prophylaxis.

  Recruiting Arkansas Children's Hospital Research Institute · Little Rock, Arkansas · NCT07582276

• Joint health, balance, and quality of life in adults with hemophilia A

  See how joint damage and postural balance relate to quality of life in adults with severe hemophilia A.

  Recruiting Yuzuncu Yil University · Van · NCT07523399

• NXT007 versus emicizumab to prevent bleeds in people with Hemophilia A

  This will test whether NXT007 prevents bleeding better than emicizumab in people aged 12 and older with Hemophilia A, including those with or without factor VIII inhibitors.

  Recruiting at 63 sites Phase 3 Hoffmann-La Roche · Orange, California and 62 other locations · NCT07416604

• Comparing NXT007 injections with Factor VIII prophylaxis for people with moderate-to-severe hemophilia A

  This will see if regular NXT007 injections prevent bleeds better than standard Factor VIII prophylaxis in people aged 12 and older with moderate-to-severe hemophilia A who do not have FVIII inhibitors.

  Recruiting at 36 sites Phase 3 Hoffmann-La Roche · Aurora, Colorado and 35 other locations · NCT07416526

• Mother-and-child hemophilia A program

  See if following pregnant people with a severe hemophilia A genotype and their children from pregnancy through early childhood can identify factors linked to maternal bleeding and the child’s development of anti‑FVIII antibodies.

  Recruiting University of Washington · Seattle, Washington · NCT07414511

• Long-term anticoagulation for adults with severe hemophilia A

  This project will see if adults with severe hemophilia A who are on modern factor VIII prophylaxis can be safely treated with long-term blood thinners.

  Recruiting University Hospital, Strasbourg, France · Strasbourg · NCT07314983

• SPK-8011QQ gene therapy for adults with severe or moderately severe hemophilia A

  This trial will test a one-time gene therapy called SPK-8011QQ in adult men with severe or moderately severe hemophilia A to see if it is safe and well tolerated.

  Recruiting at 6 sites Phase 1/2 Hoffmann-La Roche · Orange, California and 5 other locations · NCT07226206

• Damoctocog alfa pegol in previously treated children with Hemophilia A

  This is a watching-the-treatment study to see how damoctocog alfa pegol works in kids aged 7 to under 12 who have Hemophilia A and have been treated before.

  Recruiting Bayer · Loma Linda, California and 11 other locations · NCT07088458

• Evaluating GS1191-0445 Injection for Hemophilia A Treatment

  This study is testing a new gene therapy injection for hemophilia A to see if it can safely help men aged 18 to 65 by improving their blood clotting.

  Recruiting at 2 sites Phase 3 Gritgen Therapeutics Co., Ltd. · Hefei, Anhui and 12 other locations · NCT06833983

• 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

• Manual therapy for ankle pain in hemophilia patients

  This study tests if a hands-on treatment can help people with hemophilia and ankle pain feel better and move more easily.

  Recruiting Investigación en Hemofilia y Fisioterapia · Murcia · NCT06816056

• Is synovial proliferation in haemophilia joints active or inactive?

  This project will test whether ultrasound, MRI and elastography can tell if subclinical synovial proliferation in people with severe haemophilia is currently active so clinicians can treat earlier.

  Recruiting UMC Utrecht · Utrecht · NCT06809972

• Observational study of blood clotting and bleeding disorders in Chinese patients

  This study looks at blood clotting and bleeding problems in Chinese patients to see how common they are and what factors might affect them.

  Recruiting Peking University People's Hospital · Beijing, Beijing Municipality and 4 other locations · NCT06727669

• Evaluating the safety and effects of SR604 in healthy individuals and those with Hemophilia A or B

  This study is testing a new drug called SR604 to see if it's safe and effective for healthy people and those with Hemophilia A or B.

  Recruiting at 8 sites Phase 1 Equilibra Bioscience LLC · Glendale, California and 9 other locations · NCT06349473

• Evaluating NGGT003 for treating Hemophilia A

  This study is testing a new treatment called NGGT003 to see if it can safely help men with hemophilia A by allowing their bodies to produce a missing protein.

  Recruiting Early Phase 1 Institute of Hematology & Blood Diseases Hospital, China · Tianjin, Tianjin Municipality · NCT06238908

• Observing the safety of damoctocog-alfa-pegol in Korean patients with hemophilia A

  This study is looking at how safe the hemophilia A treatment damoctocog alfa pegol is for Korean patients aged 12 and older who are already using it as part of their regular care.

  Recruiting Bayer · Multiple Locations · NCT06222697

• Non-invasive prenatal diagnosis for single gene disorders

  This study is testing a new way to safely diagnose single-gene disorders in unborn babies using a simple blood test from the mother, starting as early as 9 weeks into the pregnancy.

  Recruiting Assistance Publique - Hôpitaux de Paris · Paris · NCT06147414

• Testing fitusiran to prevent bleeds in severe hemophilia A patients switching from emicizumab

  This study is testing if fitusiran can help prevent bleeding in men with severe hemophilia A who are switching from another treatment called emicizumab.

  Recruiting at 3 sites Phase 4 Sanofi · Los Angeles, California and 2 other locations · NCT06145373

• Gene therapy for treating Hemophilia A

  This study is testing a new gene therapy to see if it can safely boost factor VIII levels in people with severe Hemophilia A.

  Recruiting at 10 sites Phase 2/3 Shanghai Xinzhi BioMed Co., Ltd. · Beijing, Beijing Municipality and 9 other locations · NCT06111638

• Evaluating NXT007 for treating severe or moderate Hemophilia A

  This study is testing a new treatment called NXT007 to see if it can safely help boys and men with severe or moderate Hemophilia A manage their condition better.

  Recruiting at 12 sites Phase 1/2 Hoffmann-La Roche · Sacramento, California and 13 other locations · NCT05987449

• Using Nuwiq to manage bleeding in women and girls with Hemophilia A during surgery

  This study is testing if Nuwiq can help prevent bleeding during major surgeries for women and girls aged 12 and older with mild to moderate Hemophilia A.

  Recruiting at 13 sites Phase 4 Octapharma · San Antonio, Texas and 15 other locations · NCT05936580

• Testing marstacimab for treating hemophilia in children

  This study is testing a new medication called marstacimab to see if it can help children with severe hemophilia control their bleeding and improve their health.

  Recruiting at 51 sites Phase 3 Pfizer · Salt Lake City, Utah and 62 other locations · NCT05611801

• Evaluating the safety and effectiveness of gene therapies for Hemophilia A and B

  This study is testing two gene therapies for Hemophilia A and B to see how safe and effective they are for patients who have already taken part in previous trials.

  Recruiting at 36 sites Phase 3 Pfizer · Rancho Cordova, California and 38 other locations · NCT05568719

• Gene therapy for treating Hemophilia A

  This study is testing a new gene therapy to see if a single injection can help men with Hemophilia A increase their factor VIII levels and reduce bleeding.

  Recruiting Early Phase 1 Institute of Hematology & Blood Diseases Hospital, China · Tianjin, Tianjin Municipality · NCT05454774

• Long-term treatment with marstacimab for hemophilia patients

  This study is testing a long-term treatment called marstacimab to see if it can safely help people with severe hemophilia A or B manage their condition better.

  Recruiting at 68 sites Phase 3 Pfizer · Iowa City, Iowa and 77 other locations · NCT05145127

• Comparing Factor VIII and Emicizumab for Preventing Bleeding in Hemophilia A Patients Playing Sports

  This study tests whether Factor VIII or Emicizumab is better at preventing bleeding in boys aged 6 to 19 with Hemophilia A while they play sports.

  Recruiting at 10 sites Wayne State University · Little Rock, Arkansas and 9 other locations · NCT05022459

• Gene therapy for hemophilia A in adults

  This study is testing a new gene therapy to see if it can safely help adults with hemophilia A who have very low levels of a crucial clotting factor.

  Recruiting Institute of Hematology & Blood Diseases Hospital, China · Tianjin, Tianjin Municipality · NCT04728841

• Gene therapy for patients with Hemophilia A

  This study is testing a new gene therapy for adult men with severe hemophilia A to see if it can help increase their factor VIII levels and reduce bleeding episodes, making treatment easier than current options.

  Recruiting Phase 1/2 ASC Therapeutics · Little Rock, Arkansas · NCT04676048

• 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

• Observing treatment approaches for Hemophilia A patients with inhibitors

  This study looks at different treatment methods for men with Hemophilia A who have developed inhibitors to see how well they work and how safe they are over five years.

  Recruiting at 2 sites Emory University · Atlanta, Georgia and 1 other locations · NCT04023019

• International registry for patients with hemophilia and bleeding disorders

  This study is collecting information from patients with hemophilia and bleeding disorders around the world to help improve treatment and understanding of these conditions.

  Recruiting World Federation of Hemophilia · Montreal, Quebec · NCT03327779

• Database for pediatric hemophilia treatment and outcomes

  This study is collecting information on how children with hemophilia A and B are treated and how it affects their lives to help improve their care and outcomes.

  Recruiting Goethe University · Frankfurt am Main, Hesse · NCT02912143

NIH-funded research (27)

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

• 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

• 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

• 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

• 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

• 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

• 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

• 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

• 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

• 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

• 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 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

• Improving gene therapy for hemophilia A by targeting liver cells for factor VIII production

  This study is testing a new gene therapy for people with hemophilia A to help their bodies make more of the factor VIII protein they need to stop bleeding, using a different method that targets specific liver cells, which could lead to better treatment results.

  NIH research Children's Hosp of Philadelphia · Philadelphia, United States · NIH-10675697

• Testing a new gene therapy to improve blood clotting in patients with severe hemophilia A.

  This study is testing a new gene therapy for people with severe hemophilia A to see if it can help their bodies make a key protein that helps stop bleeding, which could mean fewer trips for treatments and less hassle with current therapies.

  NIH research Medical College of Wisconsin · Milwaukee, United States · NIH-10599276

• Using ultrasound to deliver genes for treating hemophilia A

  This study is exploring a new way to help people with hemophilia A by using ultrasound to safely deliver genes that can help their bodies make the missing factor VIII protein, which could lead to better and more affordable treatments.

  NIH research Seattle Children's Hospital · Seattle, United States · NIH-10599134

Related topics

Quick answers

How many Hemophilia A clinical trials are recruiting right now?

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

Where are these trials running?

In 43 countries. The most sites are in United States (18 trials), China (12 trials), Spain (9 trials), Canada (8 trials), Germany (7 trials). Each study page lists its sites and which ones are recruiting.

What phases are they in?

Phase 3: 6, Phase 1/2: 5, Early Phase 1: 2, Phase 4: 2. 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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