Clinical trial: Study of the Efficacy and Safety PF-06741086 in Adult and Teenage Patients With Severe Hemophilia A or B (NCT03938792)

Eligibility criteria:
Inclusion Criteria

          -  Participants with a diagnosis of severe hemophilia A or 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

          -  Participants with on-demand treatment regimen with ≥6 bleeding episodes (spontaneous
             and/or traumatic) necessitating treatment with bypass factor during the 6 months prior
             to enrollment in the Observation Phase and willing to continue to receive on-demand
             treatment during this 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

        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.

          -  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

          -  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.
Male
No healthy subjects accepted to join the trial.
Subject must be at least 12 Years old.
Subject must be at most 74 Years

Patient description (medical language): A 20-year-old man comes to the emergency due to bleeding after a tooth extraction.  The bleeding has persisted for approximately 30 minutes despite constant direct pressure.  He is a known case of Hemophilia type A treated with FVIII. Blood pressure is 95/60 mm Hg and pulse is 105/min. His weight is 70 Kg. Family history is positive for Hemophilia type A in his maternal uncle. He also has a lipoma on his left arm which he plans to remove surgically. His FVIII activity is 40%.

Patient description (patient's own words): I got my tooth extracted and I could not stop bleeding for 30 min. I'm a 20 yo dude but I have Hemophilia type A treated with FVIII. My blood pressure was 95/60 and my pulse 105/min. I'm 70 kg. My uncle on my mom's side also has type A hemophilia. I also have a fatty swelling on my left arm and I was supposed to remove it. My FVIII activity was 40%.

Does the patient match the trial criteria? 0