Brief 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
Official 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
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
August 12, 2023
Primary Completion Date
August 12, 2023
Eligibility 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.
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.
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
Brazil
Location Countries
Brazil
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
November 2021