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.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
189
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.
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
Annualized bleeding rate (ABR) of treated bleeding events
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)
Time frame: Through Observational Phase (6months) and Active Treatment Phase (12 months) for total of approximately 18 months
Incidence and severity of thrombotic events
Time frame: 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
Time frame: Throughout Active Treatment Phase (12 months)
Incidence of clinically significant persistent neutralizing antibody [NAb] against PF-06741086
Time frame: Throughout Active Treatment Phase (12 months)
Incidence and severity of injection site reaction
Time frame: Throughout Active Treatment Phase (12 months)
Number of participants with clinically significant changes from baseline in physical exam
Time frame: From Baseline through Observation and Active Treatment (approximately 18 months)
Incidence of clinically significant laboratory value abnormalities
Time frame: From Screening through Observation and Active Treatment (approximately 18 months)
Incidence of severe hypersensitivity and anaphylactic reactions
Time frame: From Screening through Observational and Active Treatment (approximately 18 months)
Incidence of adverse events and serious adverse events
Time frame: From screening through Observation and Active treatment (approximately 18 months)
Number of participants with clinically significant changes from baseline in vital signs
Time frame: From Baseline through Observation and Active Treatment (approximately 18 months)
Incidence and severity of thromboticangiopathy
Time frame: Throughout Active Treatment Phase (12 months)
Incidence of intravascular coagulation/consumption coagulopathy
Time frame: Throughout Active Treatment Phase (12 months)
Incidence of joint bleeds
Time frame: Through Observational Phase (6 months) and Active Treatment Phase (12 months) for total of approximately 18 months
Incidence of spontaneous bleeds
Time frame: Through Observational Phase (6 months) and Active Treatment Phase (12 months) for total of approximately 18 months
Incidence of target joint bleeds
Time frame: Through Observational Phase (6 months) and Active Treatment Phase (12 months) for total of approximately 18 months
Incidence of total bleeds (treated and untreated)
Time frame: Through Observational and Active Treatment Phases (18 Months)
Change from baseline in the Hemophilia Joint Health Score (HJHS)
Time frame: 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)
Time frame: 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)
Time frame: 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)
Time frame: 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)
Time frame: Through Observational Phase (6 months) and Active Treatment Phase (12 months) for total of approximately 18 months
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
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
...and 52 more locations
Patient Global Impression of Change - Hemophilia (PGIC-H)
Time frame: 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)
Time frame: Through Observational Phase (6 months) and Active Treatment Phase (12 months) for total of approximately 18 months