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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/study/NCT05145127
Registration number
NCT05145127
Ethics application status
Date submitted
11/11/2021
Date registered
6/12/2021
Date last updated
25/03/2025
Titles & IDs
Public title
Open-Label Extension Study of Marstacimab in Hemophilia Participants With or Without Inhibitors
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Scientific title
AN OPEN-LABEL EXTENSION STUDY TO EVALUATE THE LONG-TERM SAFETY, TOLERABILITY, AND EFFICACY OF MARSTACIMAB PROPHYLAXIS IN 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
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Secondary ID [1]
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PHASE 3 ANTI-TFPI OLE
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Secondary ID [2]
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B7841007
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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 - For participants aged =12 years 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.
For participants aged =6 to \<12 years is marstacimab 150 mg SC for initial loading dose followed by 75 mg SC QW. 150 mg sc qw is prescribed for participants who meet dose escalation criteria.
Treatment: Drugs: PF-06741086
For participants aged =12 years 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.
For participants aged =6 to \<12 years is marstacimab 150 mg SC for initial loading dose followed by 75 mg SC QW. 150 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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Number of subject reporting Adverse Events
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Assessment method [1]
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Timepoint [1]
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Baseline up to 7 years
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Primary outcome [2]
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Number of subjects reporting Serious Adverse Events
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Assessment method [2]
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Timepoint [2]
0
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Baseline up to 7 years
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Primary outcome [3]
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Incidence and severity of thrombotic events
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Assessment method [3]
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Timepoint [3]
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Baseline up to 7 years
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Primary outcome [4]
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Incidence and severity of thrombotic microangiopathy
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Assessment method [4]
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Timepoint [4]
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Baseline up to 7 years
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Primary outcome [5]
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Number of subjects reporting Disseminated intravascular coagulalopathy/consumption coagulopathy
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Assessment method [5]
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Timepoint [5]
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Baseline up to 7 years
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Primary outcome [6]
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Incidence of clinically significant persistent NAb against marstacimab
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Assessment method [6]
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Timepoint [6]
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Baseline up to 7 years
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Primary outcome [7]
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Incidence and severity of injection site reaction
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Assessment method [7]
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Timepoint [7]
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Baseline up to 7 years
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Primary outcome [8]
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Clinically significant changes in vital signs from baseline
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Assessment method [8]
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Timepoint [8]
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Baseline up to 7 years
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Primary outcome [9]
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Incidence of clinically significant laboratory value abnormalities
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Assessment method [9]
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Timepoint [9]
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Baseline up to 7 years
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Primary outcome [10]
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Incidence of severe hypersensitivity and anaphylactic reactions
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Assessment method [10]
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Timepoint [10]
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Baseline up to 7 years
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Secondary outcome [1]
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Annualized rate of bleeding episodes
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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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Baseline up to 7 years
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Secondary outcome [2]
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Total coagulation factor product consumption
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Assessment method [2]
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Timepoint [2]
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Baseline up to 7 years
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Secondary outcome [3]
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Incidence of joint bleeds
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Assessment method [3]
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Timepoint [3]
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Baseline up to 7 years
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Secondary outcome [4]
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Incidence of spontaneous bleeds
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Assessment method [4]
0
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Timepoint [4]
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Baseline up to 7 years
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Secondary outcome [5]
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Incidence of target joint bleeds
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Assessment method [5]
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Timepoint [5]
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Baseline up to 7 years
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Secondary outcome [6]
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Incidence of total bleeds (treated and untreated)
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Assessment method [6]
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Timepoint [6]
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Baseline up to 7 year
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Secondary outcome [7]
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Change in joints measured by the HJHS
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Assessment method [7]
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Change in joints as measured by the HJHS for participants =4 years of age
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Timepoint [7]
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Baseline up to 7 years
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Secondary outcome [8]
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Change in number of target joints per subject from baseline
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Assessment method [8]
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Timepoint [8]
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Baseline up to 7 years
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Secondary outcome [9]
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Changes in Health Utilities Measure questionnaire data
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Assessment method [9]
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Timepoint [9]
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Baseline up to 7 years
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Secondary outcome [10]
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Changes in Haem-A-QoL questionnaire data for participants =17 years of age
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Assessment method [10]
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Timepoint [10]
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Baseline up to 7 years
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Secondary outcome [11]
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Changes in Haemo-QoL questionnaire data
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Assessment method [11]
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Haemo-QoL CII (Ages 8 to \<12 years), Haemo-QoL (Ages 12 to \<17),
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Timepoint [11]
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Baseline up to 7 years
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Secondary outcome [12]
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Total bypass product consumption
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Assessment method [12]
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Timepoint [12]
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Baseline up to 7 years
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Secondary outcome [13]
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Changes in EQ-5D questionnaire data
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Assessment method [13]
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EQ-5D-Y Proxy (Ages = 4 to = 6 years), EQ-5D-Y Self (Ages = 7 to = 11 years), EQ-5D-5L (Ages =12)
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Timepoint [13]
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Baseline up to 7 years
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Eligibility
Key inclusion criteria
* All participants will have a minimum body weight as defined by parent studies
* Participants who are willing and able to comply with all scheduled visits, treatment plan, laboratory tests, and other study procedures.
* Participants have successfully completed participation in parent studies, defined as did not require "Early Termination"
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Minimum age
1
Year
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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
* Previous or current treatment for or history of coronary artery disease, venous or arterial thrombosis (CTCAE Grade >3), or ischemic disease (except catheter-associated thrombosis)
* Abnormal renal function as defined by eGFR <30 mL.min/1.73 m(2)
* Known planned surgical procedure during the planned study period
* Unstable hepatic function as determined by the Investigator clinical assessment and review of the participant's most recent laboratory results, which would make the participant inappropriate for the study
* For participants known to be HIV+, worsening disease status as determined by the Investigator clinical assessment and review of participant's most recent laboratory results, to include recent locally available CD4 count (if available), which would make the participant inappropriate for the study
* Regular, concomitant therapy with immunomodulatory drugs (eg, IVIG, and routine systemic corticosteroids, rituximab)
* Ongoing or planned use of immune tolerance induction or prophylaxis with FVIII or FIX replacement during the study
* Participation in other study involving investigational drug(s) or investigational vaccine(s) within 30 days or 5 half-lives prior to or during study participation, with the exception of participation in parent studies
* Investigator site staff or Pfizer employees directly involved in the conduct of the study, site staff otherwise supervised by the Investigator, and their respective family members
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Study design
Purpose of the study
Treatment
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Allocation to intervention
Not applicable
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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
Single group
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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
17/11/2021
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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
31/07/2030
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Actual
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Sample size
Target
245
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Accrual to date
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Final
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Recruitment in Australia
Recruitment state(s)
VIC
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Recruitment hospital [1]
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Royal Children's Hospital - Melbourne
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Recruitment postcode(s) [1]
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3052 - Melbourne
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Recruitment outside Australia
Country [1]
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United States of America
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State/province [1]
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Iowa
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Country [2]
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United States of America
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State/province [2]
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New York
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Country [3]
0
0
United States of America
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State/province [3]
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Washington
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Country [4]
0
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Canada
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State/province [4]
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Alberta
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Country [5]
0
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Canada
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State/province [5]
0
0
Ontario
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Country [6]
0
0
China
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State/province [6]
0
0
Guangdong
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Country [7]
0
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China
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State/province [7]
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Guizhou
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Country [8]
0
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China
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State/province [8]
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Hubei
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Country [9]
0
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China
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State/province [9]
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Jiangxi
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Country [10]
0
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China
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State/province [10]
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0
Tianjin
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Country [11]
0
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China
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State/province [11]
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Beijing
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Country [12]
0
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Croatia
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State/province [12]
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Zagreb
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Country [13]
0
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France
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State/province [13]
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Paris
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Country [14]
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Hong Kong
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State/province [14]
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Hong Kong
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Country [15]
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India
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State/province [15]
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Gjuarat
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Country [16]
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India
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State/province [16]
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Maharashtra
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Country [17]
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India
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State/province [17]
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WEST Bengal
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Country [18]
0
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Israel
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State/province [18]
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Hamerkaz
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Country [19]
0
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Italy
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State/province [19]
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RM
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Country [20]
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Italy
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State/province [20]
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Milano
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Country [21]
0
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Japan
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State/province [21]
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Aichi
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Country [22]
0
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Japan
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State/province [22]
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Nagano
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Country [23]
0
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Japan
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State/province [23]
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Nara
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Country [24]
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Japan
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State/province [24]
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Saitama
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Country [25]
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Japan
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State/province [25]
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Hiroshima
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Country [26]
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Korea, Republic of
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Seoul-teukbyeolsi [seoul]
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Country [27]
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Korea, Republic of
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Seoul-teukbyeolsi[seoul]
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Country [28]
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Korea, Republic of
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State/province [28]
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Taegu-kwangyokshi
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Country [29]
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Mexico
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State/province [29]
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Yucatán
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Country [30]
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Oman
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State/province [30]
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Muscat
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Country [31]
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Serbia
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State/province [31]
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Belgrade
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Country [32]
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Serbia
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State/province [32]
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Nis
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Country [33]
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South Africa
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State/province [33]
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Gauteng
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Country [34]
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South Africa
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State/province [34]
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Benoni
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Country [35]
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South Africa
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State/province [35]
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Johannesburg
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Country [36]
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Spain
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State/province [36]
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A Coruna
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Country [37]
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Spain
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State/province [37]
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Barcelona
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Country [38]
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Spain
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State/province [38]
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Madrid
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Country [39]
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Spain
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State/province [39]
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Zaragoza
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Country [40]
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Taiwan
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State/province [40]
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Changhua County
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Country [41]
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Taiwan
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State/province [41]
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Taichung
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Country [42]
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Turkey
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State/province [42]
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Adana
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Country [43]
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Turkey
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State/province [43]
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Ankara
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Country [44]
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Turkey
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State/province [44]
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Gaziantep
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Country [45]
0
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Turkey
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State/province [45]
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Istanbul
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Country [46]
0
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Turkey
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State/province [46]
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Izmir
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Country [47]
0
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Turkey
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State/province [47]
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Kayseri
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Country [48]
0
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Turkey
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State/province [48]
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Samsun
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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
Study B7841007 is an open-label extension study to assess the long-term safety, tolerability, and efficacy of prophylaxis treatment with marstacimab in participants who did not require "Early Termination" from the Phase 3 Study B7841005 and from the Phase 3 Study B7841008. Study B7841005: approximately 145 adolescent and adult participants 12 to \<75 years of age with severe hemophilia A or moderately severe to severe hemophilia B (defined as FVIII activity \<1% or FIX activity =2%, respectively) with or without inhibitors are expected to be enrolled in Study B7841005 during which they will receive prophylaxis (defined as treatment by SC injection of marstacimab). Study B7841008: this is an ongoing Phase 3, open-label study in pediatric participants \<18 years of age with severe hemophilia A (FVIII Coagulation Factor Activity \<1%) or moderately severe to severe hemophilia B (FIX Coagulation Factor Activity =2%). A sequential approach will be used in enrolling at least 100 pediatric participants, at least 20 of which will be aged =12 to \<18 years and at least 80 participants will be aged =1 to \<12 years. At the start of study B7841008, the dosing and data available in adolescent and adult participants in Study B7841005 supported the initiation of B7841008 study in participants aged =12 to \<18 years. Subsequently, additional safety and efficacy data from adolescent participants in Study B7841005 became available for benefit/risk assessment in support of dosing participants aged =6 to \<12 years. Based on the positive benefit/risk assessment conducted by both internal Pfizer review and eDMC review, dosing of the =6 to \<12 years age group was initiated in June 2023 in B7841008 Study. Data from participants =6 years from B7841008 Study and Study B7841005 will support the dosing of participants aged =1 to \<6 years. All participants will be provided the prefilled pen (PFP) for administration of marstacimab in the study. Use of the prefilled syringe (PFS) will be permitted at the investigator's discretion for those participants who have difficulty with administration of the PFP. Additionally, participants will be provided the PFS for use in this study in countries where the PFS is anticipated to be the only presentation available commercially. An optional, open-label, single arm, substudy using the PFP was completed in the first 23 participants rolled over from Study B7841005 who agreed to participate in the substudy.
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Trial website
https://clinicaltrials.gov/study/NCT05145127
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Trial related presentations / publications
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Public notes
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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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Country
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Phone
0
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Fax
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Email
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Contact person for public queries
Name
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Pfizer CT.gov Call Center
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Address
0
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Country
0
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Phone
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1-800-718-1021
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Fax
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Email
0
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[email protected]
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Contact person for scientific queries
Data sharing statement
What supporting documents are/will be available?
No Supporting Document Provided
Results publications and other study-related documents
No documents have been uploaded by study researchers.
Results not provided in
https://clinicaltrials.gov/study/NCT05145127
Download to PDF