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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT03938792




Registration number
NCT03938792
Ethics application status
Date submitted
2/05/2019
Date registered
6/05/2019

Titles & IDs
Public title
Study of the Efficacy and Safety PF-06741086 in Adult and Teenage Participants With Severe Hemophilia A or Moderately Severe to Severe Hemophilia B
Scientific title
An Open-Label Study in Adolescent and Adult 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 Comparing Standard Treatment to PF-06741086 Prophylaxis
Secondary ID [1] 0 0
2018-003660-31
Secondary ID [2] 0 0
B7841005
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 - Participants will be assigned to treatment with PF-06741086 after a 6 month Observation Phase on their current hemophilia regimen.


Treatment: Drugs: 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 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
Through Observational Phase (6months) and Active Treatment Phase (12 months) for total of approximately 18 months
Primary outcome [2] 0 0
Incidence and severity of thrombotic events
Timepoint [2] 0 0
Through Observational Phase (6 months) and Active Treatment Phase (12 months) for total of approximately 18 months
Primary outcome [3] 0 0
Incidence of anti drug antibody [ADA] against PF-06741086
Timepoint [3] 0 0
Throughout Active Treatment Phase (12 months)
Primary outcome [4] 0 0
Incidence of clinically significant persistent neutralizing antibody [NAb] against PF-06741086
Timepoint [4] 0 0
Throughout Active Treatment Phase (12 months)
Primary outcome [5] 0 0
Incidence and severity of injection site reaction
Timepoint [5] 0 0
Throughout Active Treatment Phase (12 months)
Primary outcome [6] 0 0
Number of participants with clinically significant changes from baseline in physical exam
Timepoint [6] 0 0
From Baseline through Observation and Active Treatment (approximately 18 months)
Primary outcome [7] 0 0
Incidence of clinically significant laboratory value abnormalities
Timepoint [7] 0 0
From Screening through Observation and Active Treatment (approximately 18 months)
Primary outcome [8] 0 0
Incidence of severe hypersensitivity and anaphylactic reactions
Timepoint [8] 0 0
From Screening through Observational and Active Treatment (approximately 18 months)
Primary outcome [9] 0 0
Incidence of adverse events and serious adverse events
Timepoint [9] 0 0
From screening through Observation and Active treatment (approximately 18 months)
Primary outcome [10] 0 0
Number of participants with clinically significant changes from baseline in vital signs
Timepoint [10] 0 0
From Baseline through Observation and Active Treatment (approximately 18 months)
Primary outcome [11] 0 0
Incidence and severity of thromboticangiopathy
Timepoint [11] 0 0
Throughout Active Treatment Phase (12 months)
Primary outcome [12] 0 0
Incidence of intravascular coagulation/consumption coagulopathy
Timepoint [12] 0 0
Throughout Active Treatment Phase (12 months)
Secondary outcome [1] 0 0
Incidence of joint bleeds
Timepoint [1] 0 0
Through Observational Phase (6 months) and Active Treatment Phase (12 months) for total of approximately 18 months
Secondary outcome [2] 0 0
Incidence of spontaneous bleeds
Timepoint [2] 0 0
Through Observational Phase (6 months) and Active Treatment Phase (12 months) for total of approximately 18 months
Secondary outcome [3] 0 0
Incidence of target joint bleeds
Timepoint [3] 0 0
Through Observational Phase (6 months) and Active Treatment Phase (12 months) for total of approximately 18 months
Secondary outcome [4] 0 0
Incidence of total bleeds (treated and untreated)
Timepoint [4] 0 0
Through Observational and Active Treatment Phases (18 Months)
Secondary outcome [5] 0 0
Change from baseline in the Hemophilia Joint Health Score (HJHS)
Timepoint [5] 0 0
Through Observational Phase (6 months) and Active Treatment Phase (12 months) for total of approximately 18 months
Secondary outcome [6] 0 0
Change from baseline in (Haemophilia Adult Quality of Life Questionnaire (Haem-A-QoL)
Timepoint [6] 0 0
Through Observational Phase (6 months) and Active Treatment Phase (12 months) for total of approximately 18 months
Secondary outcome [7] 0 0
Change from baseline in Haemophilia Quality of Life Questionnaire for Children (Haemo-QoL)
Timepoint [7] 0 0
Through Observational Phase (6 months) and Active Treatment Phase (12 months) for total of approximately 18 months
Secondary outcome [8] 0 0
Change from baseline in Hemophilia Adult Activities List (HAL)
Timepoint [8] 0 0
Through Observational Phase (6 months) and Active Treatment Phase (12 months) for total of approximately 18 months
Secondary outcome [9] 0 0
Change from baseline in Hemophilia Pediatric Activities List (PedHAL)
Timepoint [9] 0 0
Through Observational Phase (6 months) and Active Treatment Phase (12 months) for total of approximately 18 months
Secondary outcome [10] 0 0
Patient Global Impression of Change - Hemophilia (PGIC-H)
Timepoint [10] 0 0
Through Observational Phase (6 months) and Active Treatment Phase (12 months) for total of approximately 18 months
Secondary outcome [11] 0 0
Change from baseline in EuroQol 5 Dimensions 5 Level (EQ-5D-5L)
Timepoint [11] 0 0
Through Observational Phase (6 months) and Active Treatment Phase (12 months) for total of approximately 18 months

Eligibility
Key inclusion criteria
Inclusion Criteria

* Participants with a diagnosis of severe hemophilia A or moderately severe to severe hemophilia B with a minimum weight of 35 kg at screening.
* Participant or legally authorized representative, or participant's caregiver capable of giving signed informed consent (or minor assent, when applicable).

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

* No detectable or documented history of inhibitors
* Participants on FVIII/FIX routine prophylaxis who have demonstrated at least 80% compliance with scheduled prophylaxis regimen during 6 months prior to enrollment and are willing to continue to receive routine prophylaxis treatment with FVIII/FIX replacement during the Observational Phase.
* Participants with on-demand treatment regimen with =6 acute bleeding episodes (spontaneous or traumatic) that required coagulation factor infusion during the 6 months period prior to enrollment and willing to continue to receive on demand treatment during the Observational Phase.

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 6 months prior to enrolment into the Observational Phase
* Hemophilia A participants with on-demand treatment regimen with =6 bleeding episodes or hemophilia B participants with =4 bleeding episodes (spontaneous or traumatic) necessitating treatment with bypass factor during the 6 months prior to Enrollment into Observational Phase and willing to continue to receive on-demand treatment during the Observational Phase.
* Participants who have documented inhibitors while on factor-replacement therapy but who do not meet the 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 prior agreement from the Pfizer Medical Monitor
* Participants who meet the bleeding criteria noted above and who are on routine prophylaxis (defined as treatment by IV injection of bypass factor to prevent bleeding) and have demonstrated at least 80% compliance with scheduled prophylaxis regimen during the 6 months prior to enrollment, may be considered for eligibility on a case-by-case basis with discussion and agreement from the Pfizer medical monitor.
Minimum age
12 Years
Maximum age
74 Years
Sex
Males
Can healthy volunteers participate?
No
Key exclusion criteria
Exclusion Criteria

* Previous or current treatment for and/or history of coronary artery diseases, venous or arterial thrombosis or ischemic disease
* Known planned surgical procedure during the planned study period.
* Known hemostatic defect other than hemophilia A or B.
* Abnormal renal or hepatic function
* Current unstable liver or biliary disease
* Abnormal hematologic parameters
* Other acute or chronic medical or psychiatric condition or laboratory abnormality 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,
* Current routine prophylaxis with bypassing agent or non-coagulation non-factor- replacement therapy, or any previous treatment with a gene therapy product for treatment of hemophilia (participants treated with prophylaxis using bypassing agents or who had prior treatment with non-factor products may be considered on a case-by-case basis).
* Regular, concomitant therapy with immunomodulatory drugs

- Ongoing or planned use of immune tolerance induction during the Observational Phase or Active Treatment Phase, or prophylaxis with FVIII or FIX replacement at any time after initiation of treatment with study intervention during the Active Treatment Phase
* Previous exposure to PF 06741086 during participation in studies B7841002 and B7841003.
* Participation in other studies involving investigational drug(s) or investigational vaccines within 30 days (or as determined by local requirements) or 5 half-lives prior to study entry and/or during study participation.
* CD4 cell count =200/uL if human immunodeficiency virus (HIV)-positive
* Screening ECG that demonstrates clinically relevant abnormalities that may affect participant safety or interpretation of study results.
* Individuals with hypersensitivity or an allergic reaction to hamster protein or other components of the study intervention.
* Investigator site staff members directly involved in the conduct of the study and their family members, site staff members otherwise supervised by the investigator, or participants who are Pfizer employees, including their family members, directly involved in the conduct of the study.

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
Crossover
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Active, not recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
9/03/2020
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
16/06/2025
Actual
Sample size
Target
Accrual to date
Final
186
Recruitment in Australia
Recruitment state(s)
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Florida
Country [2] 0 0
United States of America
State/province [2] 0 0
Iowa
Country [3] 0 0
United States of America
State/province [3] 0 0
New York
Country [4] 0 0
United States of America
State/province [4] 0 0
Washington
Country [5] 0 0
Bulgaria
State/province [5] 0 0
Sofia
Country [6] 0 0
Bulgaria
State/province [6] 0 0
Stara Zagora
Country [7] 0 0
Canada
State/province [7] 0 0
Ontario
Country [8] 0 0
China
State/province [8] 0 0
Guangdong
Country [9] 0 0
China
State/province [9] 0 0
Guizhou
Country [10] 0 0
China
State/province [10] 0 0
Jiangxi
Country [11] 0 0
China
State/province [11] 0 0
Tianjin
Country [12] 0 0
China
State/province [12] 0 0
Beijing
Country [13] 0 0
Croatia
State/province [13] 0 0
Zagreb
Country [14] 0 0
France
State/province [14] 0 0
Paris
Country [15] 0 0
Hong Kong
State/province [15] 0 0
Hong Kong
Country [16] 0 0
India
State/province [16] 0 0
Gujarat
Country [17] 0 0
India
State/province [17] 0 0
Maharashtra
Country [18] 0 0
India
State/province [18] 0 0
Tamil NADU
Country [19] 0 0
Italy
State/province [19] 0 0
Milan
Country [20] 0 0
Italy
State/province [20] 0 0
RM
Country [21] 0 0
Italy
State/province [21] 0 0
Perugia
Country [22] 0 0
Japan
State/province [22] 0 0
Aichi
Country [23] 0 0
Japan
State/province [23] 0 0
Hokkaido
Country [24] 0 0
Japan
State/province [24] 0 0
Saitama
Country [25] 0 0
Japan
State/province [25] 0 0
Tokyo
Country [26] 0 0
Japan
State/province [26] 0 0
Hiroshima
Country [27] 0 0
Korea, Republic of
State/province [27] 0 0
Daegu
Country [28] 0 0
Korea, Republic of
State/province [28] 0 0
Seoul
Country [29] 0 0
Mexico
State/province [29] 0 0
Nuevo LEON
Country [30] 0 0
Mexico
State/province [30] 0 0
Yucatán
Country [31] 0 0
Oman
State/province [31] 0 0
Muscat
Country [32] 0 0
Russian Federation
State/province [32] 0 0
Samara
Country [33] 0 0
Saudi Arabia
State/province [33] 0 0
Jeddah
Country [34] 0 0
Saudi Arabia
State/province [34] 0 0
Riyadh
Country [35] 0 0
Serbia
State/province [35] 0 0
Belgrade
Country [36] 0 0
Serbia
State/province [36] 0 0
Kragujevac
Country [37] 0 0
Serbia
State/province [37] 0 0
Nis
Country [38] 0 0
South Africa
State/province [38] 0 0
Gauteng
Country [39] 0 0
Spain
State/province [39] 0 0
A Coruna
Country [40] 0 0
Spain
State/province [40] 0 0
Barcelona
Country [41] 0 0
Spain
State/province [41] 0 0
Madrid
Country [42] 0 0
Spain
State/province [42] 0 0
Salamanca
Country [43] 0 0
Spain
State/province [43] 0 0
Zaragoza
Country [44] 0 0
Taiwan
State/province [44] 0 0
Changhua County
Country [45] 0 0
Taiwan
State/province [45] 0 0
Taichung
Country [46] 0 0
Turkey
State/province [46] 0 0
Adana
Country [47] 0 0
Turkey
State/province [47] 0 0
Ankara
Country [48] 0 0
Turkey
State/province [48] 0 0
Antalya
Country [49] 0 0
Turkey
State/province [49] 0 0
Gaziantep
Country [50] 0 0
Turkey
State/province [50] 0 0
Istanbul
Country [51] 0 0
Turkey
State/province [51] 0 0
Izmir
Country [52] 0 0
Turkey
State/province [52] 0 0
Kayseri
Country [53] 0 0
Turkey
State/province [53] 0 0
Samsun
Country [54] 0 0
Turkey
State/province [54] 0 0
Trabzon

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
[email protected]
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/NCT03938792