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

Pfizer

Status and phase

Completed

Phase 1

Conditions

Severe Hemophilia

Treatments

Biological: PF-06741086

Study type

Interventional

Funder types

Industry

Identifiers

NCT04878731

B7841010

Details and patient eligibility

About

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.

Enrollment

6 patients

Sex

Male

Ages

18 to 74 years old

Volunteers

No Healthy Volunteers

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