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

Primary Purpose

Hemophilia A, Hemophilia B

Status
Recruiting
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
PF-06741086
Sponsored by
Pfizer
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Hemophilia A focused on measuring Factor VIII Inhibitor, Factor IX Inhibitor, PF-06741086, Marstacimab, Anti-TFPI

Eligibility Criteria

12 Years - 74 Years (Child, Adult, Older Adult)MaleDoes not accept healthy volunteers

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

Sites / Locations

  • University of IowaRecruiting
  • Northwell Health HTCRecruiting
  • Hamilton Health Sciences - McMaster University Medical CentreRecruiting
  • Hamilton Health Sciences - McMaster University Medical CentreRecruiting
  • Nanfang Hospital, Southern Medical UniversityRecruiting
  • The Affiliated Hospital of Guizhou Medical University
  • Jiangxi Provincial People's HospitalRecruiting
  • Institute of hematology&blood disease hospitalRecruiting
  • Beijing Children's Hospital, Capital Medical University
  • Klinicki bolnicki centar ZagrebRecruiting
  • Hôpital Universitaire Necker Enfants MaladesRecruiting
  • Prince of Wales HospitalRecruiting
  • Queen Mary HospitalRecruiting
  • Nirmal Hospital Pvt Ltd.Recruiting
  • Sahyadri Super Speciality HospitalRecruiting
  • AOU Policlinico Umberto IRecruiting
  • Fondazione IRCCS Cà Granda Ospedale Maggiore PoliclinicoRecruiting
  • Nagoya University Hospital - Transfusion MedicineRecruiting
  • Hiroshima University HospitalRecruiting
  • Severance Hospital, Yonsei University Health SystemRecruiting
  • Kyung Hee University Hospital at GangdongRecruiting
  • Severance Hospital, Yonsei University Health SystemRecruiting
  • Kyungpook National University HospitalRecruiting
  • Centro Multidisciplinario para el Desarrollo Especializado de la Investigacion Clinica en YucatanRecruiting
  • Sultan Qaboos University HospitalRecruiting
  • Institute for Mother and Child healthcare "Dr Vukan Cupic"Recruiting
  • Clinical Center NisRecruiting
  • Hospital Universitario A CorunaRecruiting
  • Hospital Universitario Vall d´HebronRecruiting
  • Hospital Universitario La PazRecruiting
  • Hospital Universitario La PazRecruiting
  • Hospital Universitario Miguel ServetRecruiting
  • ChangHua Christian HospitalRecruiting
  • Acibadem Adana HospitalRecruiting
  • Hacettepe University Faculty of MedicineRecruiting
  • Gazi University Health Research and Application Center Gazi HospitalRecruiting
  • Gaziantep University Şahinbey Research and Practice HospitalRecruiting
  • Istanbul University Oncology InstituteRecruiting
  • Ege University Faculty of MedicineRecruiting
  • Ege University Faculty of MedicineRecruiting
  • Erciyes University Faculty of MedicineRecruiting
  • Ondokuz Mayıs University Healthcare Practice and Research CenterRecruiting

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

PF-06741086

Arm Description

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.

Outcomes

Primary Outcome Measures

Number of subject reporting Adverse Events
Number of subjects reporting Serious Adverse Events
Incidence and severity of thrombotic events
Incidence and severity of thrombotic microangiopathy
Number of subjects reporting Disseminated intravascular coagulalopathy/consumption coagulopathy
Incidence of clinically significant persistent NAb against marstacimab
Incidence and severity of injection site reaction
Clinically significant changes in vital signs from baseline
Incidence of clinically significant laboratory value abnormalities
Incidence of severe hypersensitivity and anaphylactic reactions

Secondary Outcome Measures

Annualized rate of bleeding episodes
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
Incidence of joint bleeds
Incidence of spontaneous bleeds
Incidence of target joint bleeds
Incidence of total bleeds (treated and untreated)
Change in joints measured by the HJHS
Change in number of target joints per subject from baseline
Changes in Health Utilities Measure questionnaire data
Changes in Haem-A-QoL questionnaire data for participants ≥17 years of age
Changes in Haemo-QoL questionnaire data for participants 12 to <17 years of age
Total bypass product consumption

Full Information

First Posted
November 11, 2021
Last Updated
September 6, 2023
Sponsor
Pfizer
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1. Study Identification

Unique Protocol Identification Number
NCT05145127
Brief Title
Open-Label Extension Study of Marstacimab in Hemophilia Participants With or Without Inhibitors
Official 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
Study Type
Interventional

2. Study Status

Record Verification Date
September 2023
Overall Recruitment Status
Recruiting
Study Start Date
November 17, 2021 (Actual)
Primary Completion Date
July 31, 2030 (Anticipated)
Study Completion Date
July 31, 2030 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Pfizer

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Hemophilia A, Hemophilia B
Keywords
Factor VIII Inhibitor, Factor IX Inhibitor, PF-06741086, Marstacimab, Anti-TFPI

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
145 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
PF-06741086
Arm Type
Experimental
Arm Description
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 Type
Drug
Intervention Name(s)
PF-06741086
Other Intervention Name(s)
marstacimab
Intervention Description
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.
Primary Outcome Measure Information:
Title
Number of subject reporting Adverse Events
Time Frame
Baseline up to 7 years
Title
Number of subjects reporting Serious Adverse Events
Time Frame
Baseline up to 7 years
Title
Incidence and severity of thrombotic events
Time Frame
Baseline up to 7 years
Title
Incidence and severity of thrombotic microangiopathy
Time Frame
Baseline up to 7 years
Title
Number of subjects reporting Disseminated intravascular coagulalopathy/consumption coagulopathy
Time Frame
Baseline up to 7 years
Title
Incidence of clinically significant persistent NAb against marstacimab
Time Frame
Baseline up to 7 years
Title
Incidence and severity of injection site reaction
Time Frame
Baseline up to 7 years
Title
Clinically significant changes in vital signs from baseline
Time Frame
Baseline up to 7 years
Title
Incidence of clinically significant laboratory value abnormalities
Time Frame
Baseline up to 7 years
Title
Incidence of severe hypersensitivity and anaphylactic reactions
Time Frame
Baseline up to 7 years
Secondary Outcome Measure Information:
Title
Annualized rate of bleeding episodes
Description
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
Baseline up to 7 years
Title
Total coagulation factor product consumption
Time Frame
Baseline up to 7 years
Title
Incidence of joint bleeds
Time Frame
Baseline up to 7 years
Title
Incidence of spontaneous bleeds
Time Frame
Baseline up to 7 years
Title
Incidence of target joint bleeds
Time Frame
Baseline up to 7 years
Title
Incidence of total bleeds (treated and untreated)
Time Frame
Baseline up to 7 year
Title
Change in joints measured by the HJHS
Time Frame
Baseline up to 7 years
Title
Change in number of target joints per subject from baseline
Time Frame
Baseline up to 7 years
Title
Changes in Health Utilities Measure questionnaire data
Time Frame
Baseline up to 7 years
Title
Changes in Haem-A-QoL questionnaire data for participants ≥17 years of age
Time Frame
Baseline up to 7 years
Title
Changes in Haemo-QoL questionnaire data for participants 12 to <17 years of age
Time Frame
Baseline up to 7 years
Title
Total bypass product consumption
Time Frame
Baseline up to 7 years

10. Eligibility

Sex
Male
Gender Based
Yes
Gender Eligibility Description
Male participants only
Minimum Age & Unit of Time
12 Years
Maximum Age & Unit of Time
74 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Pfizer CT.gov Call Center
Phone
1-800-718-1021
Email
ClinicalTrials.gov_Inquiries@pfizer.com
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Pfizer CT.gov Call Center
Organizational Affiliation
Pfizer
Official's Role
Study Director
Facility Information:
Facility Name
University of Iowa
City
Iowa City
State/Province
Iowa
ZIP/Postal Code
52242
Country
United States
Individual Site Status
Recruiting
Facility Name
Northwell Health HTC
City
New Hyde Park
State/Province
New York
ZIP/Postal Code
11040
Country
United States
Individual Site Status
Recruiting
Facility Name
Hamilton Health Sciences - McMaster University Medical Centre
City
Hamilton
State/Province
Ontario
ZIP/Postal Code
L8N 3Z5
Country
Canada
Individual Site Status
Recruiting
Facility Name
Hamilton Health Sciences - McMaster University Medical Centre
City
Hamilton
State/Province
Ontario
ZIP/Postal Code
L8N3Z5
Country
Canada
Individual Site Status
Recruiting
Facility Name
Nanfang Hospital, Southern Medical University
City
Guangzhou
State/Province
Guangdong
ZIP/Postal Code
510515
Country
China
Individual Site Status
Recruiting
Facility Name
The Affiliated Hospital of Guizhou Medical University
City
Guiyang
State/Province
Guizhou
ZIP/Postal Code
550004
Country
China
Individual Site Status
Not yet recruiting
Facility Name
Jiangxi Provincial People's Hospital
City
Nanchang
State/Province
Jiangxi
ZIP/Postal Code
330006
Country
China
Individual Site Status
Recruiting
Facility Name
Institute of hematology&blood disease hospital
City
Tianjin
State/Province
Tianjin
ZIP/Postal Code
300020
Country
China
Individual Site Status
Recruiting
Facility Name
Beijing Children's Hospital, Capital Medical University
City
Beijing
ZIP/Postal Code
100045
Country
China
Individual Site Status
Not yet recruiting
Facility Name
Klinicki bolnicki centar Zagreb
City
Zagreb
ZIP/Postal Code
10000
Country
Croatia
Individual Site Status
Recruiting
Facility Name
Hôpital Universitaire Necker Enfants Malades
City
Paris
ZIP/Postal Code
75015
Country
France
Individual Site Status
Recruiting
Facility Name
Prince of Wales Hospital
City
Hong Kong
Country
Hong Kong
Individual Site Status
Recruiting
Facility Name
Queen Mary Hospital
City
Hong Kong
Country
Hong Kong
Individual Site Status
Recruiting
Facility Name
Nirmal Hospital Pvt Ltd.
City
Surat
State/Province
Gjuarat
ZIP/Postal Code
395002
Country
India
Individual Site Status
Recruiting
Facility Name
Sahyadri Super Speciality Hospital
City
Pune
State/Province
Maharashtra
ZIP/Postal Code
411004
Country
India
Individual Site Status
Recruiting
Facility Name
AOU Policlinico Umberto I
City
Roma
State/Province
RM
ZIP/Postal Code
00161
Country
Italy
Individual Site Status
Recruiting
Facility Name
Fondazione IRCCS Cà Granda Ospedale Maggiore Policlinico
City
Milano
ZIP/Postal Code
20122
Country
Italy
Individual Site Status
Recruiting
Facility Name
Nagoya University Hospital - Transfusion Medicine
City
Nagoya
State/Province
Aichi
ZIP/Postal Code
466-8560
Country
Japan
Individual Site Status
Recruiting
Facility Name
Hiroshima University Hospital
City
Hiroshima
ZIP/Postal Code
734-8551
Country
Japan
Individual Site Status
Recruiting
Facility Name
Severance Hospital, Yonsei University Health System
City
Seoul
State/Province
Seoul-teukbyeolsi [seoul]
ZIP/Postal Code
03722
Country
Korea, Republic of
Individual Site Status
Recruiting
Facility Name
Kyung Hee University Hospital at Gangdong
City
Seoul
State/Province
Seoul-teukbyeolsi [seoul]
ZIP/Postal Code
05278
Country
Korea, Republic of
Individual Site Status
Recruiting
Facility Name
Severance Hospital, Yonsei University Health System
City
Seoul
State/Province
Seoul-teukbyeolsi[seoul]
ZIP/Postal Code
03722
Country
Korea, Republic of
Individual Site Status
Recruiting
Facility Name
Kyungpook National University Hospital
City
Daegu
State/Province
Taegu-kwangyǒkshi
ZIP/Postal Code
41944
Country
Korea, Republic of
Individual Site Status
Recruiting
Facility Name
Centro Multidisciplinario para el Desarrollo Especializado de la Investigacion Clinica en Yucatan
City
Merida
State/Province
Yucatán
ZIP/Postal Code
97130
Country
Mexico
Individual Site Status
Recruiting
Facility Name
Sultan Qaboos University Hospital
City
Muscat
ZIP/Postal Code
123
Country
Oman
Individual Site Status
Recruiting
Facility Name
Institute for Mother and Child healthcare "Dr Vukan Cupic"
City
Belgrade
ZIP/Postal Code
11070
Country
Serbia
Individual Site Status
Recruiting
Facility Name
Clinical Center Nis
City
Nis
ZIP/Postal Code
18000
Country
Serbia
Individual Site Status
Recruiting
Facility Name
Hospital Universitario A Coruna
City
A Coruna
ZIP/Postal Code
15006
Country
Spain
Individual Site Status
Recruiting
Facility Name
Hospital Universitario Vall d´Hebron
City
Barcelona
ZIP/Postal Code
08035
Country
Spain
Individual Site Status
Recruiting
Facility Name
Hospital Universitario La Paz
City
Madrid
ZIP/Postal Code
28015
Country
Spain
Individual Site Status
Recruiting
Facility Name
Hospital Universitario La Paz
City
Madrid
ZIP/Postal Code
28046
Country
Spain
Individual Site Status
Recruiting
Facility Name
Hospital Universitario Miguel Servet
City
Zaragoza
ZIP/Postal Code
50009
Country
Spain
Individual Site Status
Recruiting
Facility Name
ChangHua Christian Hospital
City
Changhua City
State/Province
Changhua County
ZIP/Postal Code
500
Country
Taiwan
Individual Site Status
Recruiting
Facility Name
Acibadem Adana Hospital
City
Adana
ZIP/Postal Code
01130
Country
Turkey
Individual Site Status
Recruiting
Facility Name
Hacettepe University Faculty of Medicine
City
Ankara
ZIP/Postal Code
06100
Country
Turkey
Individual Site Status
Recruiting
Facility Name
Gazi University Health Research and Application Center Gazi Hospital
City
Ankara
ZIP/Postal Code
06560
Country
Turkey
Individual Site Status
Recruiting
Facility Name
Gaziantep University Şahinbey Research and Practice Hospital
City
Gaziantep
ZIP/Postal Code
27310
Country
Turkey
Individual Site Status
Recruiting
Facility Name
Istanbul University Oncology Institute
City
Istanbul
ZIP/Postal Code
34093
Country
Turkey
Individual Site Status
Recruiting
Facility Name
Ege University Faculty of Medicine
City
Izmir
ZIP/Postal Code
35040
Country
Turkey
Individual Site Status
Recruiting
Facility Name
Ege University Faculty of Medicine
City
Izmir
ZIP/Postal Code
35100
Country
Turkey
Individual Site Status
Recruiting
Facility Name
Erciyes University Faculty of Medicine
City
Kayseri
ZIP/Postal Code
38039
Country
Turkey
Individual Site Status
Recruiting
Facility Name
Ondokuz Mayıs University Healthcare Practice and Research Center
City
Samsun
ZIP/Postal Code
55200
Country
Turkey
Individual Site Status
Recruiting

12. IPD Sharing Statement

Plan to Share IPD
Yes
IPD Sharing 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.
IPD Sharing URL
https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests
Links:
URL
https://pmiform.com/clinical-trial-info-request?StudyID=B7841007
Description
To obtain contact information for a study center near you, click here.

Learn more about this trial

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

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