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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT05611801




Registration number
NCT05611801
Ethics application status
Date submitted
27/10/2022
Date registered
10/11/2022
Date last updated
10/10/2024

Titles & IDs
Public title
A Clinical Trial of Study Medicine (Marstacimab) in Pediatric Patients With Hemophilia A or Hemophilia B
Scientific title
AN OPEN-LABEL STUDY IN PEDIATRIC (<18 YEARS OF AGE), SEVERE HEMOPHILIA A PARTICIPANTS (COAGULATION FACTOR ACTIVITY <1%) WITH OR WITHOUT INHIBITORS OR MODERATELY SEVERE TO SEVERE HEMOPHILIA B PARTICIPANTS (COAGULATION FACTOR ACTIVITY =2%) WITH OR WITHOUT INHIBITORS COMPARING 12 MONTHS OF HISTORICAL STANDARD TREATMENT TO MARSTACIMAB PROPHYLAXIS
Secondary ID [1] 0 0
ANTI-TFPI PEDIATRIC STUDY
Secondary ID [2] 0 0
B7841008
Universal Trial Number (UTN)
Trial acronym
BASIS KIDS
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 - marstacimab

Experimental: marstacimab (PF-06741086) - Weekly subcutaneous injections.


Treatment: Drugs: marstacimab
marstacimab
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Annualized bleeding rate (ABR) of treated bleeding events
Timepoint [1] 0 0
Baseline to end of 12-month treatment period
Primary outcome [2] 0 0
Incidence of adverse events and serious adverse events
Timepoint [2] 0 0
Screening through end of follow-up period (approximately 14 months)
Primary outcome [3] 0 0
Incidence and severity of thrombotic events
Timepoint [3] 0 0
Baseline to end of 12-month treatment period
Primary outcome [4] 0 0
Incidence and severity of thrombotic microangiopathy
Timepoint [4] 0 0
Baseline to end of 12-month treatment period
Primary outcome [5] 0 0
Incidence and severity of disseminated intravascular coagulation/consumption coagulopathy events
Timepoint [5] 0 0
Baseline to end of 12-month treatment period
Primary outcome [6] 0 0
Immunogenicity (incidence of ADA and clinically significant persistent NAb against marstacimab)
Timepoint [6] 0 0
Baseline to end of 12-month treatment period
Primary outcome [7] 0 0
Incidence and severity of injection site reaction
Timepoint [7] 0 0
Baseline to end of 12-month treatment period
Primary outcome [8] 0 0
Incidence of severe hypersensitivity and anaphylactic reactions
Timepoint [8] 0 0
Baseline to end of 12-month treatment period
Secondary outcome [1] 0 0
Incidence of joint bleeds (treated)
Timepoint [1] 0 0
Baseline to end of 12-month treatment period
Secondary outcome [2] 0 0
Incidence of spontaneous bleeds (treated)
Timepoint [2] 0 0
Baseline to end of 12-month treatment period
Secondary outcome [3] 0 0
Incidence of target joint bleeds (treated)
Timepoint [3] 0 0
Baseline to end of 12-month treatment period
Secondary outcome [4] 0 0
Incidence of total bleeds (treated and untreated)
Timepoint [4] 0 0
Baseline to end of 12-month treatment period
Secondary outcome [5] 0 0
Number of target joints
Timepoint [5] 0 0
Baseline to end of 12-month treatment period
Secondary outcome [6] 0 0
Change from baseline in joint health as measured by the HJHS for participants =4 years of age
Timepoint [6] 0 0
Baseline to end of 12-month treatment period
Secondary outcome [7] 0 0
Changes in quality of life measured by Haem-A-QoL/Haemo-QoL (using age-dependent versions for participants =8 years of age)
Timepoint [7] 0 0
Baseline to end of 12-month treatment period
Secondary outcome [8] 0 0
Changes in quality of life measured by pedHAL (using age-dependent versions for participants =4 years of age)
Timepoint [8] 0 0
Baseline to end of 12-month treatment period
Secondary outcome [9] 0 0
Changes in quality of life measured by Patient Global Impression of Change - Hemophilia for participants =4 years of age
Timepoint [9] 0 0
Baseline to end of 12-month treatment period
Secondary outcome [10] 0 0
Changes in quality of life measured by Health Utilities Measure (EQ-5D-Y) for participants =4 years of age
Timepoint [10] 0 0
Baseline to end of 12-month treatment period

Eligibility
Key inclusion criteria
* Male participants of appropriate age and required minimum weight
* Participants aged 12 to 17 years must be at least 25 kgs at time of consent.
* Participants aged 6 to 11 years must be at least 19 kgs at time of consent.
* Minimum weight requirement for participants aged 1 to 5 years is to be determined.
* Participants with a diagnosis of severe hemophilia A or moderately severe to severe hemophilia B
* Participants must have at least 1 year of diary and/or medical records available in which exogenous FVIII or FIX replacement or bypass agent infusions and hemophilic bleeding episodes were consistently documented over the 12 months prior to the time of consent.

Participants who are enrolled into the Non-Inhibitor Cohort must also meet the following criteria:

* No current detectable inhibitor and no documented history of inhibitors in the 5 years prior to consent
* Must have at least 50 exposure days to FVIII/FIX replacement products
* Must be at least 80% compliant with a stable and effective routine prophylaxis regimen with FVIII/FIX replacement products, for at least 12 months prior to consent

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 12 months prior to the time of consent
* Participants who have documented inhibitors while on factor-replacement therapy but who do not meet the high 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 discussion and agreement from the Pfizer medical monitor.
* Hemophilia A participants with on-demand treatment regimen with =12 bleeding episodes or hemophilia B participants with on-demand treatment regimen with =8 bleeding episodes (spontaneous or traumatic) necessitating treatment with bypass factor in the 12 months prior to informed consent
* Participants must be on an on-demand bypass treatment regimen during the 12 months prior to informed consent
Minimum age
1 Year
Maximum age
17 Years
Sex
Males
Can healthy volunteers participate?
No
Key exclusion criteria
* Known coronary artery, thrombotic, or ischemic disease, or current evidence of congenital or acquired thrombophilic disease such as Anti-thrombin III deficiency, Factor V Leiden mutation, prothrombin 20210 mutation, protein C deficiency, protein S deficiency and antiphospholipid syndrome.
* Known planned surgical procedure during the planned study period
* Known hemostatic defect other than hemophilia A or B
* Abnormal hematology, renal or hepatic function laboratory results at screening
* Other acute or chronic medical or psychiatric condition 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
* Individuals with known allergic reaction or hypersensitivity to hamster protein or other components of the study intervention
* Current routine prophylaxis with bypassing agent, non-coagulation non-factor replacement therapy (eg, emicizumab), or any previous treatment with a gene therapy product for treatment of hemophilia
* Participants with inhibitors who are being treated using a prophylaxis treatment regimen with a bypass agent, and, participants who have previously received non-factor-based hemophilia therapy (eg, fitusiran, concizumab, emicizumab) will be considered on a case-by-case basis, only after discussion and agreement between the investigator and the Pfizer medical monitor
* Regular use of immunomodulatory medications (eg, IVIG, routine systemic corticosteroids, rituximab)
* Use of systemic antifibrinolytics, medications that may increase the risk of bleeding, and certain non-steroidal anti-inflammatory drugs within 120 hours of first dose of study intervention and while on study
* Ongoing or planned use of ITI, or prophylaxis with FVIII or FIX replacement at any time after initiation of treatment with study intervention
* Participation in other studies involving investigational drug(s) or investigational vaccine(s) within 30 days (or as determined by local requirements) or 5 half-lives prior to study entry or during study participation
* Previous exposure to marstacimab during participation in other marstacimab clinical studies
* CD4 cell count =200/uL if HIV-positive
* Abnormal ECG of clinical relevance that may affect participant safety or interpretation of study results
* Investigator site staff members directly involved in the conduct of the study and their family members, site staff members otherwise supervised by the investigator, and sponsor and sponsor delegate employees directly involved in the conduct of the study and their 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
9/12/2022
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
10/09/2028
Actual
Sample size
Target
100
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW,VIC
Recruitment hospital [1] 0 0
Sydney Children's Hospital - Randwick
Recruitment hospital [2] 0 0
Royal Children's Hospital - Melbourne
Recruitment postcode(s) [1] 0 0
2031 - Randwick
Recruitment postcode(s) [2] 0 0
3052 - Melbourne
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Utah
Country [2] 0 0
Argentina
State/province [2] 0 0
Mendoza
Country [3] 0 0
Austria
State/province [3] 0 0
Wien
Country [4] 0 0
Brazil
State/province [4] 0 0
Espírito Santo
Country [5] 0 0
Canada
State/province [5] 0 0
Alberta
Country [6] 0 0
Canada
State/province [6] 0 0
Manitoba
Country [7] 0 0
Canada
State/province [7] 0 0
Ontario/canada
Country [8] 0 0
China
State/province [8] 0 0
Beijing
Country [9] 0 0
China
State/province [9] 0 0
Guangdong
Country [10] 0 0
China
State/province [10] 0 0
Guizhou
Country [11] 0 0
China
State/province [11] 0 0
Hubei
Country [12] 0 0
China
State/province [12] 0 0
Jiangxi
Country [13] 0 0
China
State/province [13] 0 0
Tianjin
Country [14] 0 0
Czechia
State/province [14] 0 0
Brno-mesto
Country [15] 0 0
Denmark
State/province [15] 0 0
Hovedstaden
Country [16] 0 0
Denmark
State/province [16] 0 0
Midtjylland
Country [17] 0 0
France
State/province [17] 0 0
Paris
Country [18] 0 0
Germany
State/province [18] 0 0
Berlin
Country [19] 0 0
India
State/province [19] 0 0
Gujarat
Country [20] 0 0
India
State/province [20] 0 0
Maharashtra
Country [21] 0 0
India
State/province [21] 0 0
WEST Bengal
Country [22] 0 0
Israel
State/province [22] 0 0
Hamerkaz
Country [23] 0 0
Italy
State/province [23] 0 0
Liguria
Country [24] 0 0
Italy
State/province [24] 0 0
Milano
Country [25] 0 0
Italy
State/province [25] 0 0
Roma
Country [26] 0 0
Italy
State/province [26] 0 0
Parma
Country [27] 0 0
Italy
State/province [27] 0 0
Torino
Country [28] 0 0
Japan
State/province [28] 0 0
Hyogo
Country [29] 0 0
Japan
State/province [29] 0 0
Nagano
Country [30] 0 0
Japan
State/province [30] 0 0
Nara
Country [31] 0 0
Japan
State/province [31] 0 0
Saitama
Country [32] 0 0
Japan
State/province [32] 0 0
Saga
Country [33] 0 0
Korea, Republic of
State/province [33] 0 0
Seoul-teukbyeolsi [seoul]
Country [34] 0 0
Korea, Republic of
State/province [34] 0 0
Taegu-kwangyokshi
Country [35] 0 0
Mexico
State/province [35] 0 0
Veracruz
Country [36] 0 0
Saudi Arabia
State/province [36] 0 0
ASH Sharqiyah
Country [37] 0 0
Saudi Arabia
State/province [37] 0 0
An Narjis, Riyadh
Country [38] 0 0
Saudi Arabia
State/province [38] 0 0
Riyadh
Country [39] 0 0
Slovakia
State/province [39] 0 0
Bratislava
Country [40] 0 0
Slovakia
State/province [40] 0 0
Kosice
Country [41] 0 0
Slovakia
State/province [41] 0 0
Martin
Country [42] 0 0
South Africa
State/province [42] 0 0
Gauteng
Country [43] 0 0
Spain
State/province [43] 0 0
Madrid
Country [44] 0 0
Spain
State/province [44] 0 0
Zaragoza
Country [45] 0 0
Taiwan
State/province [45] 0 0
Changhua
Country [46] 0 0
Taiwan
State/province [46] 0 0
Taichung
Country [47] 0 0
Taiwan
State/province [47] 0 0
Taipei
Country [48] 0 0
Turkey
State/province [48] 0 0
I?stanbul
Country [49] 0 0
Turkey
State/province [49] 0 0
I?zmir
Country [50] 0 0
Turkey
State/province [50] 0 0
Kayseri
Country [51] 0 0
Turkey
State/province [51] 0 0
Adana
Country [52] 0 0
Turkey
State/province [52] 0 0
Ankara
Country [53] 0 0
Turkey
State/province [53] 0 0
Samsun
Country [54] 0 0
United Kingdom
State/province [54] 0 0
England
Country [55] 0 0
United Kingdom
State/province [55] 0 0
Birmingham
Country [56] 0 0
United Kingdom
State/province [56] 0 0
Leeds
Country [57] 0 0
United Kingdom
State/province [57] 0 0
London
Country [58] 0 0
United Kingdom
State/province [58] 0 0
Manchester

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

Ethics approval
Ethics application status

Summary
Brief summary
The purpose of this clinical trial is to learn about the safety and effects of the study medicine (called marstacimab) for the potential treatment of hemophilia in pediatric patients.

This study will enroll pediatric participants from ages 1 to 17 years in a sequential manner. The study will open enrollment to adolescent participants aged 12 to 17 years first. Then children aged 6 to 11 years will be permitted to enroll. Lastly, children aged 1 to 5 years will be permitted to enroll.

This study will enroll participants who:

* have severe Hemophilia A or moderately severe to severe Hemophilia B (with or without inhibitors)
* have accurate historical records documenting all factor VIII, factor IX, or bypass agent infusions and hemophilia bleed events for at least 1 year prior to entering the study
* if a non-inhibitor patient, must be on a stable routine prophylaxis regimen with factor VIII or factor IX replacement products for at least 12 months prior to study entry
* if an inhibitor patient, must be on an on-demand bypass treatment regimen during the 12 months prior to study entry

All participants in this study will receive marstacimab to use prophylactically. Marstacimab will be given once a week as a subcutaneous (under the skin) shot. The first dose of marstacimab will be given at the study site by the study site staff. During the 12-month treatment period, weekly doses of marstacimab can be given at home, or if preferred, the doses may be given by the study site staff.

To help us determine if the study medicine is safe and effective, we will compare participant experiences when they are taking the study medicine to a historical period when they were not. Researchers want to see if the study medicine works to prevent the bleeding episodes commonly experienced by patients with Hemophilia.

Participants will be in this study for about 14 months (approximately 1 month in a Screening period, 12 months receiving treatment, and 1 month in a follow-up period) during which they will visit the study site at least 10 times. If preferred, and if local regulations allow it, 2 of the study visits can be completed at the participant's home instead of at the study site. There will also be 6 scheduled telephone calls approximately every 2 months.
Trial website
https://clinicaltrials.gov/study/NCT05611801
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
[email protected]
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/study/NCT05611801