Study of the Efficacy and Safety PF-06741086 in Adult and Teenage Participants With Severe Hemophilia A or Moderately Severe to Severe Hemophilia B

NCT ID: NCT03938792

Last Updated: 2025-06-10

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE3
• Total Enrollment: 189 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2020-03-09
• Study Completion Date: 2025-04-29

Brief Summary

Treatment with PF-06741086 is anticipated to demonstrate a clinically relevant advantage and/or a major contribution to patient care in comparison to current methods of treatment for hemophilia A or B because it works differently than factor replacement products and will work in the presence of inhibitors. The potential for once weekly (QW) subcutaneous (SC) administration provides for treatment options in the absence of reliable vascular access, increased convenience and may enable better compliance. Combined, these qualities should result in a reduction of bleeding episodes.

Conditions

Hemophilia A; Hemophilia B

Study Design

• Allocation Method: NA
• Intervention Model: CROSSOVER
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

PF-06741086

Participants will be assigned to treatment with PF-06741086 after a 6 month Observation Phase on their current hemophilia regimen.

Group Type: EXPERIMENTAL

PF-06741086

Intervention Type: DRUG

300 milligrams(mg) subcutaneous (sc) loading dose followed by 150 mg sq once weekly (qw). 300 mg sc qw is prescribed for participants who meet dose escalation criteria.

Interventions

PF-06741086

300 milligrams(mg) subcutaneous (sc) loading dose followed by 150 mg sq once weekly (qw). 300 mg sc qw is prescribed for participants who meet dose escalation criteria.

Intervention Type: DRUG

Eligibility Criteria

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.

• Minimum Eligible Age: 12 Years
• Maximum Eligible Age: 74 Years
• Eligible Sex: MALE
• Accepts Healthy Volunteers: No

Sponsors

Pfizer

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Pfizer CT.gov Call Center

Role: STUDY_DIRECTOR

Pfizer

Locations

USF Health Morsani Center For Advanced Healthcare

Tampa, Florida, United States

University of Iowa

Iowa City, Iowa, United States

Northwell Health HTC

New Hyde Park, New York, United States

Washington Institute for Coagulation

Seattle, Washington, United States

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

Sofia, , Bulgaria

UMHAT "Prof.Dr. Stoyan Kirkovich"

Stara Zagora, , Bulgaria

McMaster University Medical Centre - Hamilton Health Sciences

Hamilton, Ontario, Canada

McMaster Children's Hospital

Hamilton, Ontario, Canada

McMaster University

Hamilton, Ontario, Canada

The Hospital For Sick Children

Toronto, Ontario, Canada

Nanfang Hospital, Southern Medical University

Guangzhou, Guangdong, China

The Affiliated Hospital of Guizhou Medical University

Guiyang, Guizhou, China

Jiangxi Provincial People's Hospital

Nanchang, Jiangxi, China

Institute of Hematology, Chinese Academy of Medical Sciences

Tianjin, Tianjin Municipality, China

Beijing Children's Hospital, Capital Medical University

Beijing, , China

Klinicki bolnicki centar Zagreb

Zagreb, , Croatia

Hôpital Necker Enfants Malades

Paris, , France

Prince of Wales Hospital

Hong Kong, , Hong Kong

Queen Mary Hospital

Hong Kong, , Hong Kong

Nirmal Hospital Pvt, Ltd

Surat, Gujarat, India

Sahyadri Clinical Research and Development Center

Pune, Maharashtra, India

Sahyadri Super Speciality Hospital

Pune, Maharashtra, India

Christian Medical College

Vellore, Tamil Nadu, India

Fondazione IRCCS Cà Granda Ospedale Maggiore Policlinico

Milan, Milan, Italy

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

Perugia, PERUGIA, Italy

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

Roma, RM, Italy

Nagoya University Hospital - Transfusion Medicine

Nagoya, Aichi-ken, Japan

Hiroshima University Hospital

Hiroshima, Hiroshima, Japan

Sapporo Tokushukai Hospital

Sapporo, Hokkaido, Japan

Saitama Medical University Hospital

Iruma-gun, Saitama, Japan

Hospital Universitario "Dr Jose Eleuterio Gonzalez"

Monterrey, Nuevo León, Mexico

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

Mérida, Yucatán, Mexico

Sultan Qaboos University Hospital

Muscat, , Oman

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

Samara, , Russia

King Abdulaziz University Hospital

Jeddah, , Saudi Arabia

King Faisal Specialist Hospital & Research Center

Riyadh, , Saudi Arabia

Clinical Center of Serbia

Belgrade, , Serbia

Institute for Mother and Child healthcare "Dr Vukan Cupic"

Belgrade, , Serbia

Clinical Center Kragujevac

Kragujevac, , Serbia

Clinical Center Nis

Niš, , Serbia

Charlotte Maxeke Johannesburg Academic Hospital

Johannesburg, Gauteng, South Africa

Kyungpook National University Hospital

Daegu, , South Korea

Severance Hospital, Yonsei University Health System

Seoul, , South Korea

Kyung Hee University Hospital At Gangdong

Seoul, , South Korea

Hospital Universitario A Coruna

A Coruña, A CORUNA, Spain

Hospital Universitario Vall d´Hebron

Barcelona, BARCELONA, Spain

Hospital Universitario La Paz

Madrid, Madrid, Spain

Hospital Universitario de Salamanca

Salamanca, SALAMANCA, Spain

Hospital Universitario Miguel Servet

Zaragoza, ZARAGOZA, Spain

ChangHua Christian Hospital

Changhua, CHANGHUA COUNTY, Taiwan

Taichung Veterans General Hospital

Taichung, , Taiwan

Acibadem Adana Hospital

Adana, , Turkey (Türkiye)

Hacettepe University Medical Faculty

Ankara, , Turkey (Türkiye)

Gazi University Health Research and Practice Center Gazi Hospital

Ankara, , Turkey (Türkiye)

Akdeniz University Medical Faculty

Antalya, , Turkey (Türkiye)

Gaziantep University Sahinbey Research and Training Hospital

Gaziantep, , Turkey (Türkiye)

Istanbul University Oncology Institute

Istanbul, , Turkey (Türkiye)

Ege University Medical Faculty

Izmir, , Turkey (Türkiye)

Dr. Behcet Uz Child Diseases Surgery Education and Research Hospital

Izmir, , Turkey (Türkiye)

Erciyes University Medical Faculty

Kayseri, , Turkey (Türkiye)

Ondokuz Mayıs University Medical Faculty

Samsun, , Turkey (Türkiye)

Karadeniz Technical University Medical Faculty

Trabzon, , Turkey (Türkiye)

Countries

United States; Bulgaria; Canada; China; Croatia; France; Hong Kong; India; Italy; Japan; Mexico; Oman; Russia; Saudi Arabia; Serbia; South Africa; South Korea; Spain; Taiwan; Turkey (Türkiye)

References

Matino D, Palladino A, Taylor CT, Hwang E, Raje S, Nayak S, McDonald R, Acharya SS, Mahlangu J, Jimenez-Yuste V, Choraria N, Yang R, Li CK, Al-Khabori M, Wali Y, Morales Adrian J, Park YS, Zulfikar OB, Teeter J. Marstacimab prophylaxis in hemophilia A/B without inhibitors: results from the phase 3 BASIS trial. Blood. 2025 Oct 2;146(14):1654-1663. doi: 10.1182/blood.2024027468.

Reference Type: DERIVED

PMID: 40608864 (View on PubMed)

Related Links

https://pmiform.com/clinical-trial-info-request?StudyID=B7841005

To obtain contact information for a study center near you, click here.

Other Identifiers

2018-003660-31

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

B7841005

Identifier Type: -

Identifier Source: org_study_id