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

NCT ID NCT03938792

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.

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Conditions

The condition(s) this trial relates to.

As listed by the trial registrant

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

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

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