With the completion of enrollment of adult patients with haemophilia A into the PROTECT VIII trial (PROphylaxis in hemophilia A patienTs via directly pEgylated long-aCTing rFVIII), Bayer HealthCare has started enrolling children in an international phase III trial to evaluate its investigational compound BAY 94-9027 for the treatment of haemophilia A. BAY 94-9027 is designed to extend the circulating half-life of rFVIII activity through site specific attachment of a polyethylene glycol (PEG) polymer to the light chain of the rFVIII molecule, while preserving its full biologic activity.
The PROTECT VIII Kids trial is a multi-centre, multinational, partially randomized, open-label trial evaluating the safety and efficacy of BAY 94-9027, a recombinant human factor VIII (rFVIII), can be used prophylactically to prolong duration of protection from bleeding events for up to one week, while also being used to treat acute bleeds.
“Children with haemophilia, and their parents, can find the frequent infusions associated with a prophylactic treatment regimen as a significant burden. It’s that understanding that drives us at Bayer to develop new and innovative molecules like BAY 94-9027,” said Kemal Malik, MD, member of the Bayer HealthCare executive committee and head of Global Development.
Bayer is committed to developing and improving treatment solutions that meet the needs of the hemophilia community, particularly children.
The study will enroll 50 previously treated patients (PTP) worldwide. Subjects will be children up to 12 years of age, who have severe hemophilia A (<1% FVIIIc) and a documented history of at least 50 exposure days (ED) with any FVIII product. Subjects who are currently treated with primary or secondary prophylaxis or are not receiving a regularly prescribed infusion regimen (on-demand or intermittent prophylaxis) may be enrolled. The total duration of the individual treatment is 50 exposure days.
Subjects will also be offered participation in an optional extension study, during which observations will be collected for at least 50 additional exposure days.
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