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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/ct2/show/NCT03938792
Registration number
NCT03938792
Ethics application status
Date submitted
2/05/2019
Date registered
6/05/2019
Date last updated
27/10/2023
Titles & IDs
Public title
Study of the Efficacy and Safety PF-06741086 in Adult and Teenage Participants With Severe Hemophilia A or Moderately Severe to Severe Hemophilia B
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Scientific title
An Open-Label Study in Adolescent and Adult Severe (Coagulation Factor Activity <1%) Hemophilia A Participants With or Without Inhibitors or Moderately Severe to Severe Hemophilia B Participants (Coagulation Factor Activity =2%) With or Without Inhibitors Comparing Standard Treatment to PF-06741086 Prophylaxis
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Secondary ID [1]
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2018-003660-31
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Secondary ID [2]
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B7841005
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Universal Trial Number (UTN)
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Trial acronym
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Hemophilia A
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Hemophilia B
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Condition category
Condition code
Blood
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Clotting disorders
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Human Genetics and Inherited Disorders
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Other human genetics and inherited disorders
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Intervention/exposure
Study type
Interventional
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Description of intervention(s) / exposure
Treatment: Drugs - PF-06741086
Experimental: PF-06741086 - Participants will be assigned to treatment with PF-06741086 after a 6 month Observation Phase on their current hemophilia regimen.
Treatment: Drugs: 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.
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Intervention code [1]
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Treatment: Drugs
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Annualized bleeding rate (ABR) of treated bleeding events
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Assessment method [1]
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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)
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Timepoint [1]
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Through Observational Phase (6months) and Active Treatment Phase (12 months) for total of approximately 18 months
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Primary outcome [2]
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Incidence and severity of thrombotic events
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Assessment method [2]
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Timepoint [2]
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Through Observational Phase (6 months) and Active Treatment Phase (12 months) for total of approximately 18 months
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Primary outcome [3]
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Incidence of anti drug antibody [ADA] against PF-06741086
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Assessment method [3]
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Timepoint [3]
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Throughout Active Treatment Phase (12 months)
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Primary outcome [4]
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Incidence of clinically significant persistent neutralizing antibody [NAb] against PF-06741086
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Assessment method [4]
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0
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Timepoint [4]
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Throughout Active Treatment Phase (12 months)
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Primary outcome [5]
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Incidence and severity of injection site reaction
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Assessment method [5]
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0
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Timepoint [5]
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Throughout Active Treatment Phase (12 months)
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Primary outcome [6]
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Number of participants with clinically significant changes from baseline in physical exam
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Assessment method [6]
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Timepoint [6]
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From Baseline through Observation and Active Treatment (approximately 18 months)
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Primary outcome [7]
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Incidence of clinically significant laboratory value abnormalities
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Assessment method [7]
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Timepoint [7]
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From Screening through Observation and Active Treatment (approximately 18 months)
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Primary outcome [8]
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Incidence of severe hypersensitivity and anaphylactic reactions
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Assessment method [8]
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Timepoint [8]
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From Screening through Observational and Active Treatment (approximately 18 months)
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Primary outcome [9]
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Incidence of adverse events and serious adverse events
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Assessment method [9]
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0
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Timepoint [9]
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From screening through Observation and Active treatment (approximately 18 months)
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Primary outcome [10]
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Number of participants with clinically significant changes from baseline in vital signs
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Assessment method [10]
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Timepoint [10]
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From Baseline through Observation and Active Treatment (approximately 18 months)
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Primary outcome [11]
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Incidence and severity of thromboticangiopathy
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Assessment method [11]
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0
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Timepoint [11]
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Throughout Active Treatment Phase (12 months)
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Primary outcome [12]
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Incidence of intravascular coagulation/consumption coagulopathy
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Assessment method [12]
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Timepoint [12]
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Throughout Active Treatment Phase (12 months)
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Secondary outcome [1]
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Incidence of joint bleeds
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Assessment method [1]
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Timepoint [1]
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Through Observational Phase (6 months) and Active Treatment Phase (12 months) for total of approximately 18 months
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Secondary outcome [2]
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Incidence of spontaneous bleeds
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Assessment method [2]
0
0
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Timepoint [2]
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Through Observational Phase (6 months) and Active Treatment Phase (12 months) for total of approximately 18 months
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Secondary outcome [3]
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0
Incidence of target joint bleeds
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Assessment method [3]
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0
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Timepoint [3]
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Through Observational Phase (6 months) and Active Treatment Phase (12 months) for total of approximately 18 months
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Secondary outcome [4]
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Incidence of total bleeds (treated and untreated)
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Assessment method [4]
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0
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Timepoint [4]
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Through Observational and Active Treatment Phases (18 Months)
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Secondary outcome [5]
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Change from baseline in the Hemophilia Joint Health Score (HJHS)
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Assessment method [5]
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Timepoint [5]
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Through Observational Phase (6 months) and Active Treatment Phase (12 months) for total of approximately 18 months
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Secondary outcome [6]
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Change from baseline in (Haemophilia Adult Quality of Life Questionnaire (Haem-A-QoL)
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Assessment method [6]
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Timepoint [6]
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Through Observational Phase (6 months) and Active Treatment Phase (12 months) for total of approximately 18 months
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Secondary outcome [7]
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Change from baseline in Haemophilia Quality of Life Questionnaire for Children (Haemo-QoL)
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Assessment method [7]
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Timepoint [7]
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Through Observational Phase (6 months) and Active Treatment Phase (12 months) for total of approximately 18 months
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Secondary outcome [8]
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Change from baseline in Hemophilia Adult Activities List (HAL)
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Assessment method [8]
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Timepoint [8]
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Through Observational Phase (6 months) and Active Treatment Phase (12 months) for total of approximately 18 months
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Secondary outcome [9]
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Change from baseline in Hemophilia Pediatric Activities List (PedHAL)
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Assessment method [9]
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Timepoint [9]
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Through Observational Phase (6 months) and Active Treatment Phase (12 months) for total of approximately 18 months
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Secondary outcome [10]
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Patient Global Impression of Change - Hemophilia (PGIC-H)
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Assessment method [10]
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Timepoint [10]
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Through Observational Phase (6 months) and Active Treatment Phase (12 months) for total of approximately 18 months
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Secondary outcome [11]
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Change from baseline in EuroQol 5 Dimensions 5 Level (EQ-5D-5L)
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Assessment method [11]
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Timepoint [11]
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Through Observational Phase (6 months) and Active Treatment Phase (12 months) for total of approximately 18 months
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Eligibility
Key inclusion 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.
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Minimum age
12
Years
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Maximum age
74
Years
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Sex
Males
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Can healthy volunteers participate?
No
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Key exclusion criteria
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.
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Study design
Purpose of the study
Treatment
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Allocation to intervention
N/A
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Open (masking not used)
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Who is / are masked / blinded?
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Intervention assignment
Crossover
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Other design features
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Phase
Phase 3
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Recruiting
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
9/03/2020
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
27/09/2024
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Actual
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Sample size
Target
145
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Accrual to date
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Final
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Recruitment in Australia
Recruitment state(s)
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Recruitment outside Australia
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United States of America
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Florida
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United States of America
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Iowa
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United States of America
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New York
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United States of America
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State/province [4]
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Washington
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Country [5]
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Bulgaria
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Sofia
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Country [6]
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Bulgaria
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State/province [6]
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Stara Zagora
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Country [7]
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Canada
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State/province [7]
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Ontario
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China
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Guangdong
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China
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Guizhou
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China
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State/province [10]
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Jiangxi
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Country [11]
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China
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Tianjin
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China
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Beijing
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Croatia
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Zagreb
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France
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Paris
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Hong Kong
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Hong Kong
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India
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Gujarat
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India
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Maharashtra
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India
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Tamil NADU
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Italy
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Milan
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Italy
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RM
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Italy
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Perugia
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Japan
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Aichi
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Japan
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Hokkaido
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Japan
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Saitama
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Japan
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Tokyo
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Japan
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Hiroshima
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Korea, Republic of
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Daegu
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Korea, Republic of
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Seoul
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Mexico
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Nuevo LEON
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Mexico
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Yucatán
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Oman
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Muscat
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Russian Federation
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Samara
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Saudi Arabia
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Jeddah
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Saudi Arabia
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Riyadh
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Serbia
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Belgrade
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Serbia
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Kragujevac
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Serbia
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Nis
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South Africa
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Gauteng
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Spain
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A Coruna
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Spain
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Barcelona
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Spain
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Madrid
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Spain
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Salamanca
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Spain
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Zaragoza
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Taiwan
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Changhua County
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Taiwan
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Taichung
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Turkey
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Adana
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Turkey
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Ankara
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Turkey
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Antalya
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Turkey
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Gaziantep
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Turkey
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Istanbul
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Turkey
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Izmir
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Turkey
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Kayseri
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Turkey
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Samsun
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Turkey
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Trabzon
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Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
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Name
Pfizer
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Address
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Country
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Ethics approval
Ethics application status
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Summary
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.
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Trial website
https://clinicaltrials.gov/ct2/show/NCT03938792
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Trial related presentations / publications
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Public notes
This record is viewable in the ANZCTR as it had previously listed Australia and/or New Zealand as a recruitment site, however these sites have since been removed
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Contacts
Principal investigator
Name
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Pfizer CT.gov Call Center
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Address
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Pfizer
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Contact person for public queries
Name
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Pfizer CT.gov Call Center
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Address
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Phone
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1-800-718-1021
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Email
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[email protected]
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Contact person for scientific queries
Summary Results
For IPD and results data, please see
https://clinicaltrials.gov/ct2/show/NCT03938792
Download to PDF