Safety, PK/PD study in adult patients with severe Haemophilia A or B
Research type
Research Study
Full title
An Ascending Single Dose Study to Evaluate the Safety, Tolerability and Pharmacokinetics/Pharmacodynamics of PF 05280602, a Recombinant Factor VIIA Variant (813D), in Adult Haemophilia A and B Subjects with or without Inhibitors.
IRAS ID
90877
Contact name
Pratima Chowdary
Sponsor organisation
Pfizer Inc., 235 East 42nd Street, New York, NY 10017
Eudract number
2011-002170-23
Clinicaltrials.gov Identifier
Research summary
Haemophilia A and B are bleeding disorders caused by a deficiency of Factor VIII (haemophilia A) or Factor IX (haemophilia B). Treatment involves factor replacement therapy to either treat or prevent against bleeding disorders. Some patients develop antibodies against the replacement factor therapy making them ineffective. In such cases, patients may receive higher doses of their replacement factors to stop the bleed while others must receive what is known as bypass agent. The study drug, PF-05280602 is a bypass therapy. PF-05280602 is a variant of activated recombinant human factor VII. PF-05280602 (Factor VIIa) is a protein which helps the blood to form a clot when bleeding occurs. This drug is being developed for the treatment and prevention of bleeding episodes in patients with haemophilia A or B who are not able to be treated successfully with routine replacement therapy. This research study aims to test PF-05280602, a new drug that has not been approved for sale. The purpose of this first in human research study is to: ?½ Test the safety of the study drug, PF-05280602 in up to 6 separate doses ?½ Measure the amount of PF-05280602 in the blood after dosing (known as pharmacokinetics) ?½ Assess the body??s reaction to the drug (known as pharmacodynamics) Patients will receive a single dose of PF-05280602 and then be evaluated for up to 60 days. In this study, PF-05280602 will be given to the participant in a non-bleeding state, therefore it is not intended to provide a therapeutic benefit to the study participant. In this study, up to 6 separate doses of the study drug will be evaluated (in a group of 6 participants), starting at the low dose group and moving toward higher doses after establishing safety in the lower doses first. Depending on when participants enter the study will depend on which dose they will receive. Safety will be reviewed on an ongoing basis by the Investigator, an independent external Data Monitoring Committee (eDMC) and by the study sponsor to determine if at any time the limits of safety have been met and enrolment should be stopped. To further mitigate risks to participants, there will be a pause between enrolment of the first 3 participants in each dose group to allow for an evaluation through 48 hours post study drug administration. Once a dosing group (6 participants) has completed enrolment and at least 15 days of follow-up, the eDMC and the study sponsor, Pfizer will review all safety data and determine if safety has been established and if enrolment in the next higher dose group may begin. Up to 36 males are planned to be enrolled in the study in approximately 20 different sites in 13 countries.
REC name
London - Hampstead Research Ethics Committee
REC reference
12/LO/0486
Date of REC Opinion
1 May 2012
REC opinion
Favourable Opinion