Phase III PROTECT VIII Kids Trial will Evaluate the Safety and Efficacy of BAY 94-9027 for Prophylaxis and Treatment of Bleeding in Previously Treated Children with Severe Hemophilia A
BERLIN, Germany I July 8, 2013 I Following the completion of enrollment of adult patients with hemophilia A into the PROTECT VIII trial (PROphylaxis in hemophilia A patienTs via directly pEgylated long-aCTing rFVIII), Bayer HealthCare announced today that the company has started to enroll children in an international Phase III trial to evaluate its investigational compound BAY 94-9027 for the treatment of hemophilia A. The PROTECT VIII Kids trial is designed to investigate whether 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.
BAY 94-9027 has been 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.
“Children with hemophilia, 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.
About the Phase III Study
PROTECT VIII Kids is a multicenter, multinational, partially randomized, open-label trial evaluating the safety and efficacy of BAY 94-9027, at different dosing frequencies, in both prophylactic and on-demand treatment of bleeding in male children with severe hemophilia A. 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.
About Hemophilia A
Hemophilia A, also known as factor VIII deficiency or classic hemophilia is a largely inherited bleeding disorder in which one of the proteins needed to form blood clots in the body is missing or reduced. Hemophilia A, the most common type of hemophilia, is caused by deficient or defective blood coagulation proteins, known as factor VIII. Hemophilia A is characterized by prolonged or spontaneous bleeding, especially into the muscles, joints or internal organs.
About Bayer HealthCare
The Bayer Group is a global enterprise with core competencies in the fields of health care, agriculture and high-tech materials. Bayer HealthCare, a subgroup of Bayer AG with annual sales of EUR 18.6 billion (2012), is one of the world’s leading, innovative companies in the healthcare and medical products industry and is based in Leverkusen, Germany. The company combines the global activities of the Animal Health, Consumer Care, Medical Care and Pharmaceuticals divisions. Bayer HealthCare’s aim is to discover, develop, manufacture and market products that will improve human and animal health worldwide. Bayer HealthCare has a global workforce of 55,300 employees (Dec 31, 2012) and is represented in more than 100 countries. More information at www.healthcare.bayer.com.
SOURCE: Bayer HealthCare Pharmaceuticals
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Phase III PROTECT VIII Kids Trial will Evaluate the Safety and Efficacy of BAY 94-9027 for Prophylaxis and Treatment of Bleeding in Previously Treated Children with Severe Hemophilia A
BERLIN, Germany I July 8, 2013 I Following the completion of enrollment of adult patients with hemophilia A into the PROTECT VIII trial (PROphylaxis in hemophilia A patienTs via directly pEgylated long-aCTing rFVIII), Bayer HealthCare announced today that the company has started to enroll children in an international Phase III trial to evaluate its investigational compound BAY 94-9027 for the treatment of hemophilia A. The PROTECT VIII Kids trial is designed to investigate whether 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.
BAY 94-9027 has been 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.
“Children with hemophilia, 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.
About the Phase III Study
PROTECT VIII Kids is a multicenter, multinational, partially randomized, open-label trial evaluating the safety and efficacy of BAY 94-9027, at different dosing frequencies, in both prophylactic and on-demand treatment of bleeding in male children with severe hemophilia A. 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.
About Hemophilia A
Hemophilia A, also known as factor VIII deficiency or classic hemophilia is a largely inherited bleeding disorder in which one of the proteins needed to form blood clots in the body is missing or reduced. Hemophilia A, the most common type of hemophilia, is caused by deficient or defective blood coagulation proteins, known as factor VIII. Hemophilia A is characterized by prolonged or spontaneous bleeding, especially into the muscles, joints or internal organs.
About Bayer HealthCare
The Bayer Group is a global enterprise with core competencies in the fields of health care, agriculture and high-tech materials. Bayer HealthCare, a subgroup of Bayer AG with annual sales of EUR 18.6 billion (2012), is one of the world’s leading, innovative companies in the healthcare and medical products industry and is based in Leverkusen, Germany. The company combines the global activities of the Animal Health, Consumer Care, Medical Care and Pharmaceuticals divisions. Bayer HealthCare’s aim is to discover, develop, manufacture and market products that will improve human and animal health worldwide. Bayer HealthCare has a global workforce of 55,300 employees (Dec 31, 2012) and is represented in more than 100 countries. More information at www.healthcare.bayer.com.
SOURCE: Bayer HealthCare Pharmaceuticals
Post Views: 30
