A Trial Investigating Safety and Efficacy of Treatment With BAY94-9027 in Severe Hemophilia A (PROTECT-VIII)
- Full Text View
- Tabular View
- No Study Results Posted
- Disclaimer
- How to Read a Study Record
Purpose
Haemophilia A is an inherited disorder in which one of the proteins, Factor VIII, needed to form blood clots is missing or not present in sufficient levels. In a person with haemophilia A, the clotting process is slowed and the person experiences bleeds that can result in serious problems and potential disability.
The current standard treatment for severe haemophilia A is regularly scheduled infusion of FVIII to keep levels high enough to prevent bleeding. Due to the short half-life of FVIII, prophylaxis may require treatment as often as every other day.
In this trial safety and efficacy of a long-acting recombinant factor VIII molecule is evaluated in subjects with severe Hemophilia A.
120-140 patients will receive open label treatment with long-acting rFVIII either on-demand to treat bleeds or prophylactically for 36 weeks in the main trial plus an optional extension to continue treatment for at least 100 total exposure days (ED). Patients on prophylactic treatment will receive study drug at dosing intervals between once and twice a week depending on their observed bleeding. Patients will attend the treatment centre for routine blood samples and be required to keep an electronic diary.
Male patients aged 12-65, with severe hemophilia A, previously treated with FVIII for at least 50 exposure days may be eligible for this study.
| Condition | Intervention | Phase |
|---|---|---|
|
Hemophilia A |
Biological: BAY94-9027 |
Phase 2 Phase 3 |
| Study Type: | Interventional |
| Study Design: | Allocation: Non-Randomized Endpoint Classification: Safety/Efficacy Study Intervention Model: Crossover Assignment Masking: Open Label Primary Purpose: Treatment |
| Official Title: | A Phase II/III, Multicenter, Partially Randomized, Open Label Trial Investigating Safety and Efficacy of On-demand and Prophylactic Treatment With BAY94-9027 in Severe Hemophilia A |
- Annualized number of total bleeds [ Time Frame: In the period of 36 weeks after Baseline ] [ Designated as safety issue: No ]
- Safety variables will be summarized using descriptive statistics based on adverse events collection [ Time Frame: Baseline visit up until end of treatment ] [ Designated as safety issue: Yes ]
- Quantification of blood loss in major surgery [ Time Frame: Pre-surgery infusion up to 24 hours after surgery ] [ Designated as safety issue: No ]
- Pharmacokinetic parameters will be measured by Tmax, Cmax, t1/2, AUC, and incremental recovery [ Time Frame: Baseline, 36 weeks ] [ Designated as safety issue: No ]
| Estimated Enrollment: | 130 |
| Study Start Date: | April 2012 |
| Estimated Study Completion Date: | December 2014 |
| Estimated Primary Completion Date: | December 2014 (Final data collection date for primary outcome measure) |
| Arms | Assigned Interventions |
|---|---|
|
Experimental: Arm 1
On-demand treatment of BAY94-9027 at individual dose and number of infusions based upon location and severity of bleeds
|
Biological: BAY94-9027
Intravenous infusion of BAY94-9027
|
|
Experimental: Arm 2
Prophylaxis treatment of BAY94-9027; 2 infusions per week over 10 weeks followed by 2 infusions per week over 26 weeks in the main trial; and at least 1 day per week in the extension for at least 100 ED
|
Biological: BAY94-9027
Intravenous infusion of BAY94-9027
|
|
Experimental: Arm 3
Prophylaxis treatment of BAY94-9027; 2 infusions per week over 10 weeks followed by infusion every 5 days over 26 weeks in the main trial; and at least 1 day per week in the extension for at least 100 ED
|
Biological: BAY94-9027
Intravenous infusion of BAY94-9027
|
|
Experimental: Arm 4
Prophylaxis treatment of BAY94-9027; 2 infusions per week over 10 weeks followed by infusion every 7 days over 26 weeks in the main trial; and at least 1 day per week in the extension for at least 100 ED
|
Biological: BAY94-9027
Intravenous infusion of BAY94-9027
|
Detailed Description:
Subjects in prophylactic treatment arms will undergo clinical evaluation at 10 weeks. Those with adequate control of bleeding will undergo randomization to every 5 or 7 day infusion. Those with continued bleeding will remain in treatment arm and have an increase in dose.
Part B-major surgery - optional sub study included to collect information on efficacy of BAY94-9027 in major surgical setting. Due to rarity of surgery in this population, enrollment to this sub-study may be independent of participation in main study.
Eligibility| Ages Eligible for Study: | 12 Years to 65 Years |
| Genders Eligible for Study: | Male |
| Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- Male; 12-65 years of age
- Subjects with severe hemophilia A
- Previously treated with factor VIII for a minimum of 150 exposure days
Exclusion Criteria:
- Inhibitors to FVIII (current evidence or history)
- Any other inherited or acquired bleeding disorder in addition to Hemophilia A
- Platelet count < 100,000/mm3
- Creatinine > 2x upper limit of normal or AST/ALT (aspartate aminotransferase/alanine aminotransferase) > 5x upper limit of normal
Contacts and Locations| Contact: Bayer Clinical Trials Contact | clinical-trials-contact@bayerhealthcare.com |
Show 93 Study Locations| Study Director: | Bayer Study Director | Bayer |
More Information
Additional Information:
No publications provided
| Responsible Party: | Therapeutic Area Head, Bayer Healthcare AG |
| ClinicalTrials.gov Identifier: | NCT01580293 History of Changes |
| Other Study ID Numbers: | 13024, 2011-005210-11 |
| Study First Received: | March 28, 2012 |
| Last Updated: | April 29, 2013 |
| Health Authority: | Argentina: Administracion Nacional de Medicamentos, Alimentos y Tecnologia Medica Austria: Federal Office for Safety in Health Care Belgium: Federal Agency for Medicinal Products and Health Products Canada: Health Canada Colombia: INVIMA Instituto Nacional de Vigilancia de Medicamentos y Alimentos Denmark: Danish Health and Medicines Authority France: Afssaps - Agence française de sécurité sanitaire des produits de santé (Saint-Denis) Germany: Paul-Ehrlich-Institut Israel: Ministry of Health Italy: The Italian Medicines Agency Japan: Pharmaceuticals and Medical Devices Agency Netherlands: The Central Committee on Research Involving Human Subjects (CCMO) Norway: Norwegian Medicines Agency Poland: Office for Registration of Medicinal Products, Medical Devices and Biocidal Products Turkey: Ministry of Health United Kingdom: Medicines and Healthcare Products Regulatory Agency United States: Food and Drug Administration Taiwan: Department of Health Sweden: Medical Products Agency Singapore: Health Sciences Authority Korea: Food and Drug Administration |
Keywords provided by Bayer:
|
Hemophilia A, factor VIII, prophylaxis |
Additional relevant MeSH terms:
|
Hemophilia A Blood Coagulation Disorders, Inherited Blood Coagulation Disorders Hematologic Diseases Coagulation Protein Disorders Hemorrhagic Disorders |
Genetic Diseases, Inborn Factor VIII Coagulants Hematologic Agents Therapeutic Uses Pharmacologic Actions |
ClinicalTrials.gov processed this record on May 23, 2013