Study of PF-06741086 in Hemophilia A or B With or Without Inhibitors
Research type
Research Study
Full title
An Open-Label Study in Adolescent and Adult Severe (Coagulation Factor Activity <1%) Hemophilia A or B Patients With or Without Inhibitors Comparing Standard Treatment to PF-06741086 Prophylaxis
IRAS ID
272193
Contact name
Sirjana Gurung
Contact email
Sponsor organisation
Pfizer Inc.
Eudract number
2018-003660-31
Clinicaltrials.gov Identifier
Clinicaltrials.gov Identifier
126734, IND Number
Duration of Study in the UK
3 years, 6 months, 14 days
Research summary
Summary of Research
Haemophilia is a rare condition that affects the blood's ability to clot. The blood doesn’t clot normally because it lacks sufficient blood-clotting factors (proteins in the blood) that combine with platelets to form a clot. Individuals with this condition may bleed for a longer time after an injury or a cut than a person without this condition. There are two types of haemophilia, A and B. Haemophilia A (most common type) is caused by a deficiency of a protein called factor VIII which helps blood to form clots. Haemophilia B is caused by deficiency of factor IX. Haemophilia is usually inherited and most people with this condition are male. The purpose of this research study is to learn about the safety and effects of the study drug (a drug that is not approved for sale) on participants with this condition.
Treatment with this study drug (PF-06741086) is expected to show an improvement in participants with haemophilia A or B and largely contribute towards patient care in comparison to current methods of treatment for this condition. This study drug works differently than treatments with factor replacement products and will work in the presence of inhibitors (molecules that bind to proteins in the blood and stop them from working properly). The study drug will be given once a week to participants through an injection under the skin during the ‘Active Treatment Phase’ of the study. This study will provide treatment options in the absence of reliable vascular access (a pathway to the bloodstream), increased convenience to patients with Haemophilia and may enable better compliance. Combined, these qualities should result in a reduction of bleeding episodes in participants with Haemophilia A or B.
Approximately 145 participants are expected to participate in the study across 94 different research study sites in about 24 countries.
Summary of Results
Public Plain Language Study Results Summary will be posted to Pfizer.com by 28-Apr-2026 (within 12-months of global end of study).REC name
HSC REC A
REC reference
20/NI/0005
Date of REC Opinion
24 Jul 2020
REC opinion
Further Information Favourable Opinion