Date: 2015-08-25
Type of information: Recruitment of the first patient
phase: 2-3
Announcement: recruitment of the first patient
Company: CSL (Australia)
Product: recombinant fusion protein linking coagulation factor VIIa with albumin (rVIIa-FP)
Action
mechanism: fusion protein/coagulation factor. This fusion protein is formed by linking recombinant Factor VIIa with albumin. Because albumin is the most abundant natural protein in plasma and has a very long half-life (i.e., more than 20 days), the fusion protein is expected to exhibit a good tolerability profile and improved pharmacokinetics that may allow for less frequent dosing. Preclinical studies have confirmed a half-life extension of greater than 8-fold.
Disease: hemophilia A with inhibitors, hemophilia B with inhibitors
Therapeutic area: Rare diseases - Genetic diseases - Hematological diseases
Country: Malaysia, UK
Trial
details: This Phase II/III study in patients with hemophilia A or B who have developed an inhibitor is a part of the PROLONG-7FP clinical development program. This program aims to demonstrate the therapeutic advantages of rVIIa-FP in patients with hemophilia A or hemophilia B who have developed inhibitors as well as in patients with congenital FVII deficiency. A Phase I PK study in patients with congenital FVII deficiency is ongoing. The purpose of this study is to investigate the pharmacokinetics (PK), efficacy, and safety of rVIIa-FP (CSL689). The study will enroll a total of 54 male subjects, 12 to 65 years of age, with hemophilia types A or B who have developed inhibitors to FVIII or FIX. The study consists of 3 sequential parts (Parts 1, 2, 3): The purpose of Part 1 (PK part) is to evaluate the PK of a single treatment of CSL689 (low dose or high dose) and compare with the PK of a single treatment of Eptacog alfa (low dose or high dose). In Part 1, CSL689 and Eptacog alfa will be given by the doctor at the study center. The purpose of Part 2 (Dose-evaluation part) is to identify which of the 2 tested dose levels of CSL689 shows the best efficacy and safety in stopping acute bleeding events (this dose will be called the \"population best dose\"). The purpose of the final Part 3 (Repeated-dose part) is to confirm the efficacy and safety of the \"population best dose\" identified in Part 2. In Parts 2 and 3, subjects will self-administer a specified number of CSL689 infusions at home on-demand (ie, when a bleeding event occurs), will keep an electronic diary, and will visit the center at monthly intervals. This study is expected to last for up to 16 months for the subjects participating in all 3 parts, and up to 9 months for the subjects participating in Part 3 only. (NCT02484638)
Latest
news: * On August 25, 2015, CSL Behring announced that the first patient has been enrolled in its Phase II/III clinical study evaluating the pharmacokinetics (PK), efficacy, and safety of the company\'s recombinant fusion protein linking coagulation factor VIIa with albumin (rVIIa-FP) for on-demand treatment in patients with congenital hemophilia A or B who have developed an inhibitor to factor VIII or factor IX replacement therapy. The study will enroll approximately 54 male patients, the first of whom was enrolled in Malaysia.
