Brief Title
Study of the Efficacy and Safety PF-06741086 in Adult and Teenage Patients With Severe Hemophilia A or B
Official Title
An Open-Label Study in Adolescent and Adult Severe (Coagulation Factor Activity <1%) Hemophilia A or B Patients With or Without Inhibitors Comparing Standard Treatment to PF-06741086 Prophylaxis
Brief Summary
Treatment with PF-06741086 is anticipated to demonstrate a clinically relevant advantage
and/or a major contribution to patient care in comparison to current methods of treatment for
hemophilia A or B because it works differently than factor replacement products and will work
in the presence of inhibitors. The potential for once weekly (QW) subcutaneous (SC)
administration provides for treatment options in the absence of reliable vascular access,
increased convenience and may enable better compliance. Combined, these qualities should
result in a reduction of bleeding episodes.
Study Phase
Phase 3
Study Type
Interventional
Primary Outcome
Annualized bleeding rate (ABR) of treated bleeding events
Secondary Outcome
Total coagulation factor and/or bypass product consumption
Condition
Hemophilia A
Intervention
PF-06741086
Study Arms / Comparison Groups
PF-06741086
Description: Participants will be assigned to treatment with PF-06741086 after a 6 month Observation Phase on their current hemophilia regimen.
Publications
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Recruitment Status
Drug
Estimated Enrollment
145
Start Date
March 9, 2020
Completion Date
April 27, 2023
Primary Completion Date
April 27, 2023
Eligibility Criteria
Inclusion Criteria
- Participants with a diagnosis of severe hemophilia A or B
- 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 outside the US and Canada with FVIII/FIX replacement, and willing to
continue to receive routine prophylaxis treatment with FVIII/FIX replacement during
the Observational Phase. Participants in the US and Canada receiving routine
prophylaxis treatment not eligible to be enrolled.
- Participants with on-demand treatment regimen with ≥6 acute bleeding episodes
(spontaneous and/or traumatic) that required coagulation factor infusion during the 6
months period prior to Screening 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
approval from the Pfizer Medical Monitor.
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 30 days prior to Baseline of Observational
Phase
- Participants with on-demand treatment regimen with ≥6 bleeding episodes (spontaneous
and/or traumatic) necessitating treatment with bypass factor for at least 6 months
prior to screening and willing to continue to receive on-demand treatment during the
Observational Phase.
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
- Abnormal coagulation activity
- 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
- Regular, concomitant therapy with immunomodulatory drugs
- Ongoing or planned use of immune tolerance induction or prophylaxis with FVIII or FIX
replacement during the Active Treatment Phase.
- Previous exposure to PF 06741086 during to participation in studies B7841002 and
B7841003.
- Participation in other studies involving investigational drug(s) 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
- Clinically relevant ECG 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.
Gender
Male
Ages
12 Years - 74 Years
Accepts Healthy Volunteers
No
Contacts
Pfizer CT.gov Call Center, 1-800-718-1021, [email protected]
Location Countries
Bulgaria
Location Countries
Bulgaria
Administrative Informations
NCT ID
NCT03938792
Organization ID
B7841005
Secondary IDs
2018-003660-31
Responsible Party
Sponsor
Study Sponsor
Pfizer
Study Sponsor
Pfizer CT.gov Call Center, Study Director, Pfizer
Verification Date
July 2020