Study of the Efficacy and Safety PF-06741086 in Adult and Teenage Participants With Severe Hemophilia A or Moderately Severe to Severe Hemophilia B
- 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
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- Male
- Target Recruitment
- 189
Not provided
Not provided
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 severe hypersensitivity and anaphylactic reactions From Screening through Observational 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) Number of participants with clinically significant changes from baseline in physical exam From Baseline through Observation and Active Treatment (approximately 18 months) Incidence of clinically significant laboratory value abnormalities From Screening through Observation 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)
- Secondary Outcome Measures
Name Time Method 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 Hemophilia Pediatric Activities List (PedHAL) Through Observational Phase (6 months) and Active Treatment Phase (12 months) for total of approximately 18 months 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 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 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 (62)
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
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