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

Phase 3

Active, not recruiting

• Conditions: Hemophilia B; Hemophilia A
• Interventions: Drug: PF-06741086

• Registration Number: NCT03938792
• Lead Sponsor: Pfizer

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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2019-05-02
• Study First Submitted QC: 2019-05-02
• Study First Posted: 2019-05-06
• Study Start: 2020-03-09
• Status Verified: 2024-10
• Last Update Submitted: 2024-10-08
• Last Update Posted: 2024-10-10
• Primary Completion: 2025-06-16
• Study Completion: 2025-06-16

Recruitment & Eligibility

• Status: ACTIVE_NOT_RECRUITING
• Sex: Male
• Target Recruitment: 186

Inclusion Criteria

Not provided

Read More

Exclusion Criteria

Not provided

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: CROSSOVER

Arm && Interventions

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

Primary Outcome Measures

Name	Time	Method
Incidence of clinically significant laboratory value abnormalities	From Screening through Observation and Active Treatment (approximately 18 months)
Incidence of severe hypersensitivity and anaphylactic reactions	From Screening through Observational and Active Treatment (approximately 18 months)
Incidence of adverse events and serious adverse events	From screening through Observation and Active treatment (approximately 18 months)
Number of participants with clinically significant changes from baseline in vital signs	From Baseline through Observation and Active Treatment (approximately 18 months)
Incidence and severity of thromboticangiopathy	Throughout Active Treatment Phase (12 months)
Incidence of intravascular coagulation/consumption coagulopathy	Throughout Active Treatment Phase (12 months)
Number of participants with clinically significant changes from baseline in physical exam	From Baseline through Observation and Active Treatment (approximately 18 months)
Annualized bleeding rate (ABR) of treated bleeding events	Through Observational Phase (6months) and Active Treatment Phase (12 months) for total of approximately 18 months	Derived for each subject for each treatment period by using the following formula: ABR = number of bleeds requiring treatments/ (days on treatment period / 365.25)
Incidence and severity of thrombotic events	Through Observational Phase (6 months) and Active Treatment Phase (12 months) for total of approximately 18 months
Incidence of anti drug antibody [ADA] against PF-06741086	Throughout Active Treatment Phase (12 months)
Incidence of clinically significant persistent neutralizing antibody [NAb] against PF-06741086	Throughout Active Treatment Phase (12 months)
Incidence and severity of injection site reaction	Throughout Active Treatment Phase (12 months)

Secondary Outcome Measures

Name	Time	Method
Incidence of joint bleeds	Through Observational Phase (6 months) and Active Treatment Phase (12 months) for total of approximately 18 months
Incidence of spontaneous bleeds	Through Observational Phase (6 months) and Active Treatment Phase (12 months) for total of approximately 18 months
Incidence of target joint bleeds	Through Observational Phase (6 months) and Active Treatment Phase (12 months) for total of approximately 18 months
Incidence of total bleeds (treated and untreated)	Through Observational and Active Treatment Phases (18 Months)
Change from baseline in the Hemophilia Joint Health Score (HJHS)	Through Observational Phase (6 months) and Active Treatment Phase (12 months) for total of approximately 18 months
Change from baseline in (Haemophilia Adult Quality of Life Questionnaire (Haem-A-QoL)	Through Observational Phase (6 months) and Active Treatment Phase (12 months) for total of approximately 18 months
Change from baseline in Haemophilia Quality of Life Questionnaire for Children (Haemo-QoL)	Through Observational Phase (6 months) and Active Treatment Phase (12 months) for total of approximately 18 months
Change from baseline in Hemophilia Adult Activities List (HAL)	Through Observational Phase (6 months) and Active Treatment Phase (12 months) for total of approximately 18 months
Change from baseline in Hemophilia Pediatric Activities List (PedHAL)	Through Observational Phase (6 months) and Active Treatment Phase (12 months) for total of approximately 18 months
Patient Global Impression of Change - Hemophilia (PGIC-H)	Through Observational Phase (6 months) and Active Treatment Phase (12 months) for total of approximately 18 months
Change from baseline in EuroQol 5 Dimensions 5 Level (EQ-5D-5L)	Through Observational Phase (6 months) and Active Treatment Phase (12 months) for total of approximately 18 months

Trial Locations

Locations (64)

Washington Institute for Coagulation; 🇺🇸 Seattle, Washington, United States

Institute of Hematology, Chinese Academy of Medical Sciences; 🇨🇳 Tianjin, Tianjin, China

USF Health Morsani Center For Advanced Healthcare; 🇺🇸 Tampa, Florida, United States

University of Iowa; 🇺🇸 Iowa City, Iowa, United States

National Specialized Hospital for the Active Treatment of Hematological Diseases - EAD, Sofia; 🇧🇬 Sofia, Bulgaria

Northwell Health HTC; 🇺🇸 New Hyde Park, New York, United States

McMaster Children's Hospital; 🇨🇦 Hamilton, Ontario, Canada

McMaster University Medical Centre - Hamilton Health Sciences; 🇨🇦 Hamilton, Ontario, Canada

UMHAT "Prof.Dr. Stoyan Kirkovich"; 🇧🇬 Stara Zagora, Bulgaria

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

Beijing Children's Hospital, Capital Medical University; 🇨🇳 Beijing, China

Hôpital Necker Enfants Malades; 🇫🇷 Paris, France

Klinicki bolnicki centar Zagreb; 🇭🇷 Zagreb, Croatia

Prince of Wales Hospital; 🇭🇰 Hong Kong, Hong Kong

Fondazione IRCCS Cà Granda Ospedale Maggiore Policlinico; 🇮🇹 Milano, Milan, Italy

Università degli Studi di Roma "Sapienza"-Policlinico Umberto I; 🇮🇹 Roma, RM, Italy

Nagoya University Hospital - Transfusion Medicine; 🇯🇵 Nagoya, Aichi, Japan

Sapporo Tokushukai Hospital; 🇯🇵 Sapporo, Hokkaido, Japan

Saitama Medical University Hospital; 🇯🇵 Iruma-gun, Saitama, Japan

Ogikubo Hospital; 🇯🇵 Suginami-ku, Tokyo, Japan

Hiroshima University Hospital; 🇯🇵 Hiroshima, Japan

Kyungpook National University Hospital; 🇰🇷 Daegu, Korea, Republic of

Severance Hospital, Yonsei University Health System; 🇰🇷 Seoul, Korea, Republic of

Kyung Hee University Hospital At Gangdong; 🇰🇷 Seoul, Korea, Republic of

Hospital Universitario "Dr Jose Eleuterio Gonzalez"; 🇲🇽 Monterrey, Nuevo LEON, Mexico

Charlotte Maxeke Johannesburg Academic Hospital; 🇿🇦 Johannesburg, Gauteng, South Africa

Clinical Center of Serbia; 🇷🇸 Belgrade, Serbia

Clinical Center Kragujevac; 🇷🇸 Kragujevac, Serbia

Hospital Universitario A Coruna; 🇪🇸 A Coruna, Spain

Clinical Center Nis; 🇷🇸 Nis, Serbia

Hospital Universitario La Paz; 🇪🇸 Madrid, Spain

Hospital Universitario Vall d´Hebron; 🇪🇸 Barcelona, Spain

Hospital Universitario de Salamanca; 🇪🇸 Salamanca, Spain

Hospital Universitario Miguel Servet; 🇪🇸 Zaragoza, Spain

Akdeniz University Medical Faculty; 🇹🇷 Antalya, Turkey

Acibadem Adana Hospital; 🇹🇷 Adana, Turkey

Hacettepe University Medical Faculty; 🇹🇷 Ankara, Turkey

Istanbul University Oncology Institute; 🇹🇷 Istanbul, Turkey

Gaziantep University Sahinbey Research and Training Hospital; 🇹🇷 Gaziantep, Turkey

Dr. Behcet Uz Child Diseases Surgery Education and Research Hospital; 🇹🇷 Izmir, Turkey

Ege University Medical Faculty; 🇹🇷 Izmir, Turkey

Erciyes University Medical Faculty; 🇹🇷 Kayseri, Turkey

Ondokuz Mayıs University Medical Faculty; 🇹🇷 Samsun, Turkey

Karadeniz Technical University Medical Faculty; 🇹🇷 Trabzon, Turkey

Centro Multidisciplinario para el Desarrollo Especializado de la Inv. Clínica en Yucatán, S.C.P.; 🇲🇽 Mérida, Yucatán, Mexico

Nirmal Hospital Pvt, Ltd; 🇮🇳 Surat, Gujarat, India

Sahyadri Clinical Research and Development Center; 🇮🇳 Pune, Maharashtra, India

Sahyadri Super Speciality Hospital; 🇮🇳 Pune, Maharashtra, India

Sultan Qaboos University Hospital; 🇴🇲 Muscat, Oman

ChangHua Christian Hospital; 🇨🇳 Changhua City, Changhua County, Taiwan

Queen Mary Hospital; 🇭🇰 Hong Kong, Hong Kong

FGBOU VO "Samara State Medical University" of MoH of Russia; 🇷🇺 Samara, Russian Federation

Taichung Veterans General Hospital; 🇨🇳 Taichung, Taiwan

King Faisal Specialist Hospital & Research Center; 🇸🇦 Riyadh, Saudi Arabia

The Hospital For Sick Children; 🇨🇦 Toronto, Ontario, Canada

Christian Medical College; 🇮🇳 Vellore, Tamil NADU, India

McMaster University; 🇨🇦 Hamilton, Ontario, Canada

Tokyo Metropolitan Children's Medical Center; 🇯🇵 Fuchu, Tokyo, Japan

King Abdulaziz University Hospital; 🇸🇦 Jeddah, Saudi Arabia

Institute for Mother and Child healthcare "Dr Vukan Cupic"; 🇷🇸 Belgrade, Serbia

Gazi University Health Research and Practice Center Gazi Hospital; 🇹🇷 Ankara, Turkey

Università degli Studi di Perugia, Azienda Ospedaliera di Perugia, Ospedale Santa Maria della; 🇮🇹 Perugia, Italy