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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

Titles & IDs
Public title
Open-Label Extension Study of Marstacimab in Hemophilia Participants With or Without Inhibitors
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
Secondary ID [1] 0 0
PHASE 3 ANTI-TFPI OLE
Secondary ID [2] 0 0
B7841007
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Hemophilia A 0 0
Hemophilia B 0 0
Condition category
Condition code
Blood 0 0 0 0
Clotting disorders
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders

Intervention/exposure
Study type
Interventional
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.
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Number of subject reporting Adverse Events
Timepoint [1] 0 0
Baseline up to 7 years
Primary outcome [2] 0 0
Number of subjects reporting Serious Adverse Events
Timepoint [2] 0 0
Baseline up to 7 years
Primary outcome [3] 0 0
Incidence and severity of thrombotic events
Timepoint [3] 0 0
Baseline up to 7 years
Primary outcome [4] 0 0
Incidence and severity of thrombotic microangiopathy
Timepoint [4] 0 0
Baseline up to 7 years
Primary outcome [5] 0 0
Number of subjects reporting Disseminated intravascular coagulalopathy/consumption coagulopathy
Timepoint [5] 0 0
Baseline up to 7 years
Primary outcome [6] 0 0
Incidence of clinically significant persistent NAb against marstacimab
Timepoint [6] 0 0
Baseline up to 7 years
Primary outcome [7] 0 0
Incidence and severity of injection site reaction
Timepoint [7] 0 0
Baseline up to 7 years
Primary outcome [8] 0 0
Clinically significant changes in vital signs from baseline
Timepoint [8] 0 0
Baseline up to 7 years
Primary outcome [9] 0 0
Incidence of clinically significant laboratory value abnormalities
Timepoint [9] 0 0
Baseline up to 7 years
Primary outcome [10] 0 0
Incidence of severe hypersensitivity and anaphylactic reactions
Timepoint [10] 0 0
Baseline up to 7 years
Secondary outcome [1] 0 0
Annualized rate of bleeding episodes
Timepoint [1] 0 0
Baseline up to 7 years
Secondary outcome [2] 0 0
Total coagulation factor product consumption
Timepoint [2] 0 0
Baseline up to 7 years
Secondary outcome [3] 0 0
Incidence of joint bleeds
Timepoint [3] 0 0
Baseline up to 7 years
Secondary outcome [4] 0 0
Incidence of spontaneous bleeds
Timepoint [4] 0 0
Baseline up to 7 years
Secondary outcome [5] 0 0
Incidence of target joint bleeds
Timepoint [5] 0 0
Baseline up to 7 years
Secondary outcome [6] 0 0
Incidence of total bleeds (treated and untreated)
Timepoint [6] 0 0
Baseline up to 7 year
Secondary outcome [7] 0 0
Change in joints measured by the HJHS
Timepoint [7] 0 0
Baseline up to 7 years
Secondary outcome [8] 0 0
Change in number of target joints per subject from baseline
Timepoint [8] 0 0
Baseline up to 7 years
Secondary outcome [9] 0 0
Changes in Health Utilities Measure questionnaire data
Timepoint [9] 0 0
Baseline up to 7 years
Secondary outcome [10] 0 0
Changes in Haem-A-QoL questionnaire data for participants =17 years of age
Timepoint [10] 0 0
Baseline up to 7 years
Secondary outcome [11] 0 0
Changes in Haemo-QoL questionnaire data
Timepoint [11] 0 0
Baseline up to 7 years
Secondary outcome [12] 0 0
Total bypass product consumption
Timepoint [12] 0 0
Baseline up to 7 years
Secondary outcome [13] 0 0
Changes in EQ-5D questionnaire data
Timepoint [13] 0 0
Baseline up to 7 years

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"
Minimum age
1 Year
Maximum age
74 Years
Sex
Males
Can healthy volunteers participate?
No
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

Study design
Purpose of the study
Treatment
Allocation to intervention
Not applicable
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
17/11/2021
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
31/07/2030
Actual
Sample size
Target
245
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
VIC
Recruitment hospital [1] 0 0
Royal Children's Hospital - Melbourne
Recruitment postcode(s) [1] 0 0
3052 - Melbourne
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Iowa
Country [2] 0 0
United States of America
State/province [2] 0 0
New York
Country [3] 0 0
United States of America
State/province [3] 0 0
Washington
Country [4] 0 0
Canada
State/province [4] 0 0
Alberta
Country [5] 0 0
Canada
State/province [5] 0 0
Ontario
Country [6] 0 0
China
State/province [6] 0 0
Guangdong
Country [7] 0 0
China
State/province [7] 0 0
Guizhou
Country [8] 0 0
China
State/province [8] 0 0
Hubei
Country [9] 0 0
China
State/province [9] 0 0
Jiangxi
Country [10] 0 0
China
State/province [10] 0 0
Tianjin
Country [11] 0 0
China
State/province [11] 0 0
Beijing
Country [12] 0 0
Croatia
State/province [12] 0 0
Zagreb
Country [13] 0 0
France
State/province [13] 0 0
Paris
Country [14] 0 0
Hong Kong
State/province [14] 0 0
Hong Kong
Country [15] 0 0
India
State/province [15] 0 0
Gjuarat
Country [16] 0 0
India
State/province [16] 0 0
Maharashtra
Country [17] 0 0
India
State/province [17] 0 0
WEST Bengal
Country [18] 0 0
Israel
State/province [18] 0 0
Hamerkaz
Country [19] 0 0
Italy
State/province [19] 0 0
RM
Country [20] 0 0
Italy
State/province [20] 0 0
Milano
Country [21] 0 0
Japan
State/province [21] 0 0
Aichi
Country [22] 0 0
Japan
State/province [22] 0 0
Nagano
Country [23] 0 0
Japan
State/province [23] 0 0
Nara
Country [24] 0 0
Japan
State/province [24] 0 0
Saitama
Country [25] 0 0
Japan
State/province [25] 0 0
Hiroshima
Country [26] 0 0
Korea, Republic of
State/province [26] 0 0
Seoul-teukbyeolsi [seoul]
Country [27] 0 0
Korea, Republic of
State/province [27] 0 0
Seoul-teukbyeolsi[seoul]
Country [28] 0 0
Korea, Republic of
State/province [28] 0 0
Taegu-kwangyokshi
Country [29] 0 0
Mexico
State/province [29] 0 0
Yucatán
Country [30] 0 0
Oman
State/province [30] 0 0
Muscat
Country [31] 0 0
Serbia
State/province [31] 0 0
Belgrade
Country [32] 0 0
Serbia
State/province [32] 0 0
Nis
Country [33] 0 0
South Africa
State/province [33] 0 0
Gauteng
Country [34] 0 0
South Africa
State/province [34] 0 0
Benoni
Country [35] 0 0
South Africa
State/province [35] 0 0
Johannesburg
Country [36] 0 0
Spain
State/province [36] 0 0
A Coruna
Country [37] 0 0
Spain
State/province [37] 0 0
Barcelona
Country [38] 0 0
Spain
State/province [38] 0 0
Madrid
Country [39] 0 0
Spain
State/province [39] 0 0
Zaragoza
Country [40] 0 0
Taiwan
State/province [40] 0 0
Changhua County
Country [41] 0 0
Taiwan
State/province [41] 0 0
Taichung
Country [42] 0 0
Turkey
State/province [42] 0 0
Adana
Country [43] 0 0
Turkey
State/province [43] 0 0
Ankara
Country [44] 0 0
Turkey
State/province [44] 0 0
Gaziantep
Country [45] 0 0
Turkey
State/province [45] 0 0
Istanbul
Country [46] 0 0
Turkey
State/province [46] 0 0
Izmir
Country [47] 0 0
Turkey
State/province [47] 0 0
Kayseri
Country [48] 0 0
Turkey
State/province [48] 0 0
Samsun

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Pfizer
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Pfizer CT.gov Call Center
Address 0 0
Pfizer
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Pfizer CT.gov Call Center
Address 0 0
Country 0 0
Phone 0 0
1-800-718-1021
Fax 0 0
Email 0 0
ClinicalTrials.gov_Inquiries@pfizer.com
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
Yes
What data in particular will be shared?
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.
When will data be available (start and end dates)?
Available to whom?
Available for what types of analyses?
How or where can data be obtained?
IPD available at link: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests


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