Study to Evaluate Safety and Tolerability of a Single Dose of PF-06741086 in Chinese Adult Participants With Severe Hemophilia

Brief Title

Study to Evaluate Safety and Tolerability of a Single Dose of PF-06741086 in Chinese Adult Participants With Severe Hemophilia

Official Title

A PHASE 1, SINGLE-ARM, OPEN-LABEL, NON-RANDOMIZED, NON-CONTROLLED MULTICENTER STUDY TO EVALUATE THE PHARMACOKINETICS, PHARMACODYNAMICS, SAFETY, AND TOLERABILITY OF A SINGLE SUBCUTANEOUS DOSE OF PF-06741086 IN CHINESE ADULT PARTICIPANTS WITH SEVERE HEMOPHILIA

Brief Summary

      This Phase 1 study will be a single-arm, open-label, non-randomized, non-controlled
      investigation of the safety, tolerability, pharmacokinetics, and pharmacodynamics of
      PF-06741086 in Chinese adult participants with severe hemophilia.

Study Phase

Phase 1

Study Type

Interventional

Primary Outcome

Percentage of participants by severity and causal relationship of treatment emergent adverse events (TEAEs)

Secondary Outcome

 Plasma PF 06741086 concentrations

Condition

Severe Hemophilia

Intervention

PF-06741086

Study Arms / Comparison Groups

 single arm

Description:  PF-06741086 300mg subcutaneous(SC)

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

Biological

Estimated Enrollment

Start Date

April 16, 2021

Completion Date

August 10, 2021

Primary Completion Date

August 10, 2021

Eligibility Criteria

        Inclusion Criteria:

          -  Participant must be male and 18 to <75 years of age with a minimum body weight of 30
             kg at screening.

          -  Participants with a diagnosis of severe hemophilia A or B (FVIII or FIX activity <1%,
             respectively)

          -  Participants without inhibitor must also meet the following criteria:

               -  No detectable or documented history of inhibitors

               -  Participants with on-demand treatment regimen with ≥6 acute bleeding episodes
                  (spontaneous or traumatic) that required coagulation factor infusion during the 4
                  months period prior to enrollment and willing to continue to receive on demand
                  treatment during the study.

          -  Participants with Inhibitor 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 4 months prior to screening.

               -  Participants with on-demand treatment regimen with ≥6 bleeding episodes
                  (spontaneous and/or traumatic) necessitating treatment with bypass factor for at
                  least 4 months prior to screening and willing to continue to receive on-demand
                  treatment during the study.

        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.

          -  Corrected QT interval (QTc) >450 msec for male participants or QTc >480 msec in
             participants with bundle branch block.

          -  Individuals with hypersensitivity or an allergic reaction to hamster protein or other
             components of the study intervention.

          -  A positive urine drug screen

          -  Current routine prophylaxis with bypassing agent or non coagulation factor-replacement
             therapy (eg, emicizumab)

          -  Regular, concomitant therapy with immunomodulatory drugs

          -  Ongoing or planned use of immune tolerance induction or prophylaxis with FVIII or FIX
             replacement during the study.

          -  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

          -  Baseline ECG that demonstrates clinically relevant abnormalities 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, or
             participants who are Pfizer employees, including their family members, directly
             involved in the conduct of the study

Gender

Male

Ages

18 Years - 74 Years

Accepts Healthy Volunteers

No

Contacts

Pfizer CT.gov Call Center, ,

Location Countries

China

Location Countries

China

Administrative Informations

NCT ID

NCT04878731

Organization ID

B7841010

Responsible Party

Sponsor

Study Sponsor

Pfizer

Study Sponsor

Pfizer CT.gov Call Center, Study Director, Pfizer

Verification Date

September 2021