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A Once-a-Week shot could transform hemophilia Care—Even for those with inhibitors

NCT ID NCT03938792

Disease control Sponsor: Pfizer Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed This study
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Aug 14, 2026 · Last updated Aug 14, 2026

Summary

This trial is testing an investigational drug called PF-06741086, given as a once-weekly injection under the skin, to see if it can reduce the number of bleeding episodes in people with severe hemophilia A or moderately severe to severe hemophilia B. The study includes both people who have inhibitors (antibodies that block standard factor replacement therapy) and those who do not. The goal is to compare this new approach to standard treatment and see if it offers better bleeding control, more convenience, and fewer bleeds.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
PF-06741086, an investigational drug given as a weekly injection under the skin
What this could lead to
If successful, this could offer a more convenient and effective way to prevent bleeding episodes in people with hemophilia, including those who have developed inhibitors to standard treatments.
What could go wrong
This is a phase 3 trial, but it's still experimental. It may not work for everyone, and there are risks of side effects like injection site reactions, allergic reactions, or blood clotting issues.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

188 people

The number who actually took part.

Started

Mar 2020

Finished

Apr 2025

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

12 to 74 years

Sex

Male participants only

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria * Participants with a diagnosis of severe hemophilia A or moderately severe to severe hemophilia B with a minimum weight of 35 kg at screening. * Participant or legally authorized representative, or participant's caregiver capable of giving signed informed consent (or minor assent, when applicable). Participants who are enrolled into the Non-Inhibitor Cohort must also meet the following criteria: * No detectable or documented history of inhibitors * Participants on FVIII/FIX routine prophylaxis who have demonstrated at least 80% compliance with scheduled prophylaxis regimen during 6 months prior to enrollment and are willing to continue to receive routine prophylaxis treatment with FVIII/FIX replacement during the Observational Phase. * Participants with on-demand treatment regimen with ≥6 acute bleeding episodes (spontaneous or traumatic) that required coagulation factor infusion during the 6 months period prior to enrollment and willing to continue to receive on demand treatment during the Observational Phase. Participants who are enrolled into the Inhibitor Cohort must also meet the following criteria: * Documentation of current high titer inhibitor (≥5 BU/mL) or current low titer inhibitor (\<5 BU/mL) refractory to FVIII or FIX replacement and with FVIII or FIX recovery \<60% of expected within previous 6 months prior to enrolment into the Observational Phase * Hemophilia A participants with on-demand treatment regimen with ≥6 bleeding episodes or hemophilia B participants with ≥4 bleeding episodes (spontaneous or traumatic) necessitating treatment with bypass factor during the 6 months prior to Enrollment into Observational Phase and willing to continue to receive on-demand treatment during the Observational Phase. * Participants who have documented inhibitors while on factor-replacement therapy but who do not meet the quantitative inhibitor criteria described in the prior bullet at the time of Screening (eg, participant with a previously documented high-titer inhibitor (≥5 BU/mL) and whose condition precludes re-challenge with FVIII or FIX replacement) may be considered for eligibility on a case-by-case basis with prior agreement from the Pfizer Medical Monitor * Participants who meet the bleeding criteria noted above and who are on routine prophylaxis (defined as treatment by IV injection of bypass factor to prevent bleeding) and have demonstrated at least 80% compliance with scheduled prophylaxis regimen during the 6 months prior to enrollment, may be considered for eligibility on a case-by-case basis with discussion and agreement from the Pfizer medical monitor. Exclusion Criteria * Previous or current treatment for and/or history of coronary artery diseases, venous or arterial thrombosis or ischemic disease * Known planned surgical procedure during the planned study period. * Known hemostatic defect other than hemophilia A or B. * Abnormal renal or hepatic function * Current unstable liver or biliary disease * Abnormal hematologic parameters * Other acute or chronic medical or psychiatric condition or laboratory abnormality that may increase the risk associated with study participation or investigational product administration or may interfere with the interpretation of study results and, in the judgment of the investigator, * Current routine prophylaxis with bypassing agent or non-coagulation non-factor- replacement therapy, or any previous treatment with a gene therapy product for treatment of hemophilia (participants treated with prophylaxis using bypassing agents or who had prior treatment with non-factor products may be considered on a case-by-case basis). * Regular, concomitant therapy with immunomodulatory drugs \- Ongoing or planned use of immune tolerance induction during the Observational Phase or Active Treatment Phase, or prophylaxis with FVIII or FIX replacement at any time after initiation of treatment with study intervention during the Active Treatment Phase * Previous exposure to PF 06741086 during participation in studies B7841002 and B7841003. * Participation in other studies involving investigational drug(s) or investigational vaccines within 30 days (or as determined by local requirements) or 5 half-lives prior to study entry and/or during study participation. * CD4 cell count ≤200/uL if human immunodeficiency virus (HIV)-positive * Screening ECG that demonstrates clinically relevant abnormalities that may affect participant safety or interpretation of study results. * Individuals with hypersensitivity or an allergic reaction to hamster protein or other components of the study intervention. * Investigator site staff members directly involved in the conduct of the study and their family members, site staff members otherwise supervised by the investigator, or participants who are Pfizer employees, including their family members, directly involved in the conduct of the study.

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Conditions

The condition(s) this trial relates to.

hemophilia A hemophilia B Hemorrhage severe hemophilia A

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Hemophilia A Hemophilia B

Contacts and locations

Locations

  • Acibadem Adana Hospital

    Adana, 01130, Turkey (Türkiye)

  • Akdeniz University Medical Faculty

    Antalya, 07060, Turkey (Türkiye)

  • Beijing Children's Hospital, Capital Medical University

    Beijing, 100045, China

  • Centro Multidisciplinario para el Desarrollo Especializado de la Inv. Clínica en Yucatán, S.C.P.

    Mérida, Yucatán, 97130, Mexico

  • ChangHua Christian Hospital

    Changhua, Changhua County, 500, Taiwan

  • Charlotte Maxeke Johannesburg Academic Hospital

    Johannesburg, Gauteng, 2193, South Africa

  • Christian Medical College

    Vellore, Tamil Nadu, 632004, India

  • Clinical Center Kragujevac

    Kragujevac, 34000, Serbia

  • Clinical Center Nis

    Niš, 18000, Serbia

  • Clinical Center of Serbia

    Belgrade, 11000, Serbia

  • Dr. Behcet Uz Child Diseases Surgery Education and Research Hospital

    Izmir, 35210, Turkey (Türkiye)

  • Ege University Medical Faculty

    Izmir, 35040, Turkey (Türkiye)

  • Erciyes University Medical Faculty

    Kayseri, 38039, Turkey (Türkiye)

  • FGBOU VO "Samara State Medical University" of MoH of Russia

    Samara, 443079, Russia

  • Fondazione IRCCS Cà Granda Ospedale Maggiore Policlinico

    Milan, Milan, 20122, Italy

  • Gazi University Health Research and Practice Center Gazi Hospital

    Ankara, 06500, Turkey (Türkiye)

  • Gaziantep University Sahinbey Research and Training Hospital

    Gaziantep, 27310, Turkey (Türkiye)

  • Hacettepe University Medical Faculty

    Ankara, 06230, Turkey (Türkiye)

  • Hiroshima University Hospital

    Hiroshima, 734-8551, Japan

  • Hospital Universitario "Dr Jose Eleuterio Gonzalez"

    Monterrey, Nuevo León, 64460, Mexico

  • Hospital Universitario A Coruna

    A Coruña, 15006, Spain

  • Hospital Universitario La Paz

    Madrid, 28046, Spain

  • Hospital Universitario Miguel Servet

    Zaragoza, 50009, Spain

  • Hospital Universitario Vall d´Hebron

    Barcelona, 08035, Spain

  • Hospital Universitario de Salamanca

    Salamanca, 37007, Spain

  • Hôpital Necker Enfants Malades

    Paris, 75015, France

  • Institute for Mother and Child healthcare "Dr Vukan Cupic"

    Belgrade, 11000, Serbia

  • Institute of Hematology, Chinese Academy of Medical Sciences

    Tianjin, Tianjin Municipality, 300020, China

  • Istanbul University Oncology Institute

    Istanbul, 34093, Turkey (Türkiye)

  • Jiangxi Provincial People's Hospital

    Nanchang, Jiangxi, 306113, China

  • Karadeniz Technical University Medical Faculty

    Trabzon, 61080, Turkey (Türkiye)

  • King Abdulaziz University Hospital

    Jeddah, 21589, Saudi Arabia

  • King Faisal Specialist Hospital & Research Center

    Riyadh, Saudi Arabia

  • Klinicki bolnicki centar Zagreb

    Zagreb, 10000, Croatia

  • Kyung Hee University Hospital At Gangdong

    Seoul, 05278, South Korea

  • Kyungpook National University Hospital

    Daegu, 41944, South Korea

  • McMaster Children's Hospital

    Hamilton, Ontario, L8S 4K1, Canada

  • McMaster University

    Hamilton, Ontario, L8S 4K1, Canada

  • McMaster University Medical Centre - Hamilton Health Sciences

    Hamilton, Ontario, L8N 3Z5, Canada

  • Nagoya University Hospital - Transfusion Medicine

    Nagoya, Aichi-ken, 466-8560, Japan

  • Nanfang Hospital, Southern Medical University

    Guangzhou, Guangdong, 510515, China

  • National Specialized Hospital for the Active Treatment of Hematological Diseases - EAD, Sofia

    Sofia, 1756, Bulgaria

  • Nirmal Hospital Pvt, Ltd

    Surat, Gujarat, 395002, India

  • Northwell Health HTC

    New Hyde Park, New York, 11040, United States

  • Ondokuz Mayıs University Medical Faculty

    Samsun, 55280, Turkey (Türkiye)

  • Prince of Wales Hospital

    Hong Kong, Hong Kong

  • Queen Mary Hospital

    Hong Kong, Hong Kong

  • Sahyadri Clinical Research and Development Center

    Pune, Maharashtra, 411004, India

  • Sahyadri Super Speciality Hospital

    Pune, Maharashtra, 411004, India

  • Saitama Medical University Hospital

    Iruma-gun, Saitama, 350-0495, Japan

  • Sapporo Tokushukai Hospital

    Sapporo, Hokkaido, 004-0041, Japan

  • Severance Hospital, Yonsei University Health System

    Seoul, 03722, South Korea

  • Sultan Qaboos University Hospital

    Muscat, 123, Oman

  • Taichung Veterans General Hospital

    Taichung, 40705, Taiwan

  • The Affiliated Hospital of Guizhou Medical University

    Guiyang, Guizhou, 550004, China

  • The Hospital For Sick Children

    Toronto, Ontario, M5G 1X8, Canada

  • UMHAT "Prof.Dr. Stoyan Kirkovich"

    Stara Zagora, 6003, Bulgaria

  • USF Health Morsani Center For Advanced Healthcare

    Tampa, Florida, 33612, United States

  • University of Iowa

    Iowa City, Iowa, 52242, United States

  • Università degli Studi di Perugia, Azienda Ospedaliera di Perugia, Ospedale Santa Maria della

    Perugia, 06156, Italy

  • Università degli Studi di Roma "Sapienza"-Policlinico Umberto I

    Roma, RM, 00161, Italy

  • Washington Institute for Coagulation

    Seattle, Washington, 98101, United States

More trials for these conditions

Other studies related to the condition(s) this trial covers.