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Open-Label Extension Study of Marstacimab in Hemophilia Participants With or Without Inhibitors

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ClinicalTrials.gov Identifier: NCT05145127
Recruitment Status : Recruiting
First Posted : December 6, 2021
Last Update Posted : January 23, 2023
Sponsor:
Information provided by (Responsible Party):
Pfizer

Tracking Information
First Submitted Date  ICMJE November 11, 2021
First Posted Date  ICMJE December 6, 2021
Last Update Posted Date January 23, 2023
Actual Study Start Date  ICMJE November 17, 2021
Estimated Primary Completion Date July 31, 2030   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: December 13, 2021)
  • Number of subject reporting Adverse Events [ Time Frame: Baseline up to 7 years ]
  • Number of subjects reporting Serious Adverse Events [ Time Frame: Baseline up to 7 years ]
  • Incidence and severity of thrombotic events [ Time Frame: Baseline up to 7 years ]
  • Incidence and severity of thrombotic microangiopathy [ Time Frame: Baseline up to 7 years ]
  • Number of subjects reporting Disseminated intravascular coagulalopathy/consumption coagulopathy [ Time Frame: Baseline up to 7 years ]
  • Incidence of clinically significant persistent NAb against marstacimab [ Time Frame: Baseline up to 7 years ]
  • Incidence and severity of injection site reaction [ Time Frame: Baseline up to 7 years ]
  • Clinically significant changes in vital signs from baseline [ Time Frame: Baseline up to 7 years ]
  • Incidence of clinically significant laboratory value abnormalities [ Time Frame: Baseline up to 7 years ]
  • Incidence of severe hypersensitivity and anaphylactic reactions [ Time Frame: Baseline up to 7 years ]
Original Primary Outcome Measures  ICMJE
 (submitted: December 1, 2021)
  • Number of subject reporting Adverse Events [ Time Frame: Baseline up to 7 years ]
  • Number of subjects reporting Serious Adverse Events [ Time Frame: Baseline up to 7 years ]
  • Incidence and severity of thrombotic events [ Time Frame: Baseline up to 7 years ]
  • Incidence and severity of thrombotic microangiopathy [ Time Frame: Baseline up to 7 years ]
  • Number of subjects reporting Disseminated intravascular coagulopathy/consumption coagulopathy [ Time Frame: Baseline up to 7 years ]
  • Incidence of clinically significant persistent NAb against marstacimab [ Time Frame: Baseline up to 7 years ]
  • Incidence and severity of injection site reaction [ Time Frame: Baseline up to 7 years ]
  • Clinically significant changes in vital signs from baseline [ Time Frame: Baseline up to 7 years ]
  • Incidence of clinically significant laboratory value abnormalities [ Time Frame: Baseline up to 7 years ]
  • Incidence of severe hypersensitivity and anaphylactic reactions [ Time Frame: Baseline up to 7 years ]
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: January 19, 2023)
  • Annualized rate of bleeding episodes [ Time Frame: Baseline up to 7 years ]
    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)
  • Total coagulation factor product consumption [ Time Frame: Baseline up to 7 years ]
  • Incidence of joint bleeds [ Time Frame: Baseline up to 7 years ]
  • Incidence of spontaneous bleeds [ Time Frame: Baseline up to 7 years ]
  • Incidence of target joint bleeds [ Time Frame: Baseline up to 7 years ]
  • Incidence of total bleeds (treated and untreated) [ Time Frame: Baseline up to 7 year ]
  • Change in joints measured by the HJHS [ Time Frame: Baseline up to 7 years ]
  • Change in number of target joints per subject from baseline [ Time Frame: Baseline up to 7 years ]
  • Changes in Health Utilities Measure questionnaire data [ Time Frame: Baseline up to 7 years ]
  • Changes in Haem-A-QoL questionnaire data for participants ≥17 years of age [ Time Frame: Baseline up to 7 years ]
  • Changes in Haemo-QoL questionnaire data for participants 12 to <17 years of age [ Time Frame: Baseline up to 7 years ]
  • Total bypass product consumption [ Time Frame: Baseline up to 7 years ]
Original Secondary Outcome Measures  ICMJE
 (submitted: December 1, 2021)
  • Annualized rate of bleeding episodes [ Time Frame: Baseline up to 7 years ]
    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)
  • Total coagulation factor product consumption [ Time Frame: Baseline up to 7 years ]
  • Incidence of joint bleeds [ Time Frame: Baseline up to 7 years ]
  • Incidence of spontaneous bleeds [ Time Frame: Baseline up to 7 years ]
  • Incidence of target joint bleeds [ Time Frame: Baseline up to 7 years ]
  • Incidence of total bleeds (treated and untreated) [ Time Frame: Baseline up to 7 year ]
  • Percentage of participants with no bleeding episodes [ Time Frame: Baseline up to 7 years ]
  • Change in joints measured by the HJHS [ Time Frame: Baseline up to 7 years ]
  • Change in number of target joints per subject from baseline [ Time Frame: Baseline up to 7 years ]
  • Changes in Health Utilities Measure questionnaire data [ Time Frame: Baseline up to 7 years ]
  • Changes in Haem-A-QoL questionnaire data for participants ≥17 years of age [ Time Frame: Baseline up to 7 years ]
  • Changes in Haemo-QoL questionnaire data for participants 12 to <17 years of age [ Time Frame: Baseline up to 7 years ]
  • Total bypass product consumption [ Time Frame: Baseline up to 7 years ]
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Open-Label Extension Study of Marstacimab in Hemophilia Participants With or Without Inhibitors
Official Title  ICMJE 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
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. 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 this study during which they will receive prophylaxis (defined as treatment by SC injection of marstacimab). The dosing regimen of marstacimab is 150 mg SC once weekly.

All participants will be provided the PFP for administration of marstacimab in the study. Use of the 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. In addition, an optional, open-label, single arm, substudy using the PFP will be administered to the first approximately 20 participants rolling over from Study B7841005 who agree to participate in the substudy.

Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE
  • Hemophilia A
  • Hemophilia B
Intervention  ICMJE Drug: 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.
Other Name: marstacimab
Study Arms  ICMJE Experimental: 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.
Intervention: Drug: PF-06741086
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: December 1, 2021)
145
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE July 31, 2030
Estimated Primary Completion Date July 31, 2030   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  • All participants will have a minimum body weight of 35 kg
  • Participants have successfully completed participation in study B7841005, defined as did not require "Early Termination" from study B7841005

Exclusion Criteria:

  • Previous or current treatment for or history of coronary artery disease, venous or arterial thrombosis (CTCAE Grade >3), or ischemic disease (except catherter-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 study B7841005
  • 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
Sex/Gender  ICMJE
Sexes Eligible for Study: Male
Gender Based Eligibility: Yes
Gender Eligibility Description: Male participants only
Ages  ICMJE 12 Years to 74 Years   (Child, Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Pfizer CT.gov Call Center 1-800-718-1021 ClinicalTrials.gov_Inquiries@pfizer.com
Listed Location Countries  ICMJE Canada,   Croatia,   France,   Hong Kong,   India,   Japan,   Korea, Republic of,   Mexico,   Oman,   Serbia,   Spain,   Taiwan,   Turkey,   United States
Removed Location Countries Russian Federation
 
Administrative Information
NCT Number  ICMJE NCT05145127
Other Study ID Numbers  ICMJE B7841007
PHASE 3 ANTI-TFPI OLE ( Other Identifier: Alias Study Number )
2022-500470-33-00 ( Registry Identifier: CTIS(EU) )
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: Yes
Plan Description: Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests.
URL: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests
Current Responsible Party Pfizer
Original Responsible Party Same as current
Current Study Sponsor  ICMJE Pfizer
Original Study Sponsor  ICMJE Same as current
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: Pfizer CT.gov Call Center Pfizer
PRS Account Pfizer
Verification Date January 2023

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP