Trial Condition(s):
Hemophilia A
Safety and Efficacy of BAY94-9027 in Previously Treated Male Children with Haemophilia A
Trial Purpose
Hemophilia A is an inherited blood disorder in which one protein, Factor VIII, needed to form blood clots is missing or not present in sufficient levels. Hemophilia A causes the clotting process to be slowed and the person experiences bleeds causing serious problems that could lead to disability. The current standard treatment for severe hemophilia A is infusion of FVIII to stop bleeding, or regular scheduled treatment to prevent bleeds from occuring. 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 being evaluated in 50 male subjects, < 12 years of age, with severe Hemophilia A. These subjects will receive open label treatment with long-acting rFVIII for approximately 6 months (or longer until 50 exposure days) on a regular schedule at least once every 7-days. Doses and dose intervals may be adapted to the subject's clinical need. A second group of patients will receive open label treatment with the same drug for 12 weeks on a regular schedule of 2x/week. Patients will attend the treatment center for routine blood samples and will be required to keep an electronic diary.
Subjects will be offered participation in an optional extension study to collect observations for at least an additional 50 exposure days.
Inclusion Criteria
- Males < 12 years of age - Subjects with severe hemophilia A - Previously treated with FVIII for > 50 exposure days
Exclusion Criteria
- Subjects with current evidence of or history of inhibitors to FVIII - Any other inherited or acquired bleeding disorder - Platelet counts < 100,000/mm^3 - Creatinine > 2x the upper limit of normal - Aspartate aminotransferase (AST) / Alanine aminotransferase (ALT) > 5x the upper limit of normal
Trial Summary
Enrollment Goal
Trial Dates
Phase
Could I receive a placebo?
Products
Accepts Healthy Volunteers
Where to Participate
| Locations | Status | ||
|---|---|---|---|
Locations Investigative Site Milano, Italy, 20122 | Status Completed | Contact Us: E-mail: [email protected] Phone: (+)1-888-84 22937 | |
Locations Investigative Site Padova, Italy, 35128 | Status Completed | Contact Us: E-mail: [email protected] Phone: (+)1-888-84 22937 | |
Locations Investigative Site Palermo, Italy, 90127 | Status Completed | Contact Us: E-mail: [email protected] Phone: (+)1-888-84 22937 | |
Locations Investigative Site Ramat Gan, Israel, 5262000 | Status Completed | Contact Us: E-mail: [email protected] Phone: (+)1-888-84 22937 | |
Locations Investigative Site UTRECHT, Netherlands, 3584 CX | Status Completed | Contact Us: E-mail: [email protected] Phone: (+)1-888-84 22937 | |
Locations Investigative Site AMSTERDAM, Netherlands | Status Completed | Contact Us: E-mail: [email protected] Phone: (+)1-888-84 22937 | |
Locations Investigative Site Columbus, United States, 43205-2696 | Status Completed | Contact Us: E-mail: [email protected] Phone: (+)1-888-84 22937 | |
Locations Investigative Site Varna, Bulgaria, 9010 | Status Completed | Contact Us: E-mail: [email protected] Phone: (+)1-888-84 22937 | |
Locations Investigative Site Plovdiv, Bulgaria, 4002 | Status Completed | Contact Us: E-mail: [email protected] Phone: (+)1-888-84 22937 | |
Locations Investigative Site Sofia, Bulgaria, 1527 | Status Completed | Contact Us: E-mail: [email protected] Phone: (+)1-888-84 22937 | |
Locations Investigative Site Cincinnati, United States, 45229 | Status Completed | Contact Us: E-mail: [email protected] Phone: (+)1-888-84 22937 | |
Locations Investigative Site Cleveland, United States, 44106-6007 | Status Completed | Contact Us: E-mail: [email protected] Phone: (+)1-888-84 22937 | |
Locations Investigative Site Hershey, United States, 17033 | Status Completed | Contact Us: E-mail: [email protected] Phone: (+)1-888-84 22937 | |
Locations Investigative Site LEUVEN, Belgium, 3000 | Status Completed | Contact Us: E-mail: [email protected] Phone: (+)1-888-84 22937 | |
Locations Investigative Site La Plata, Argentina, 1900 | Status Completed | Contact Us: E-mail: [email protected] Phone: (+)1-888-84 22937 | |
Locations Investigative Site Timisoara, Romania, 300011 | Status Completed | Contact Us: E-mail: [email protected] Phone: (+)1-888-84 22937 | |
Locations Investigative Site Bucharest, Romania, 011026 | Status Completed | Contact Us: E-mail: [email protected] Phone: (+)1-888-84 22937 | |
Locations Investigative Site Bucharest, Romania, 022328 | Status Completed | Contact Us: E-mail: [email protected] Phone: (+)1-888-84 22937 | |
Locations Investigative Site GENT, Belgium, 9000 | Status Completed | Contact Us: E-mail: [email protected] Phone: (+)1-888-84 22937 | |
Locations Investigative Site Newcastle Upon Tyne, United Kingdom, NE1 4LP | Status Completed | Contact Us: E-mail: [email protected] Phone: (+)1-888-84 22937 | |
Locations Investigative Site Lodz, Poland, 91-738 | Status Completed | Contact Us: E-mail: [email protected] Phone: (+)1-888-84 22937 | |
Locations Investigative Site Olsztyn, Poland, 10-561 | Status Completed | Contact Us: E-mail: [email protected] Phone: (+)1-888-84 22937 | |
Locations Investigative Site Vilnius, Lithuania, 08661 | Status Completed | Contact Us: E-mail: [email protected] Phone: (+)1-888-84 22937 | |
Locations Investigative Site Sacramento, United States, 95817 | Status Completed | Contact Us: E-mail: [email protected] Phone: (+)1-888-84 22937 | |
Locations Investigative Site Manchester, United Kingdom, M13 9WL | Status Completed | Contact Us: E-mail: [email protected] Phone: (+)1-888-84 22937 | |
Locations Investigative Site Sheffield, United Kingdom, S10 2TH | Status Completed | Contact Us: E-mail: [email protected] Phone: (+)1-888-84 22937 | |
Locations Investigative Site Toronto, Canada, M5G 1X8 | Status Completed | Contact Us: E-mail: [email protected] Phone: (+)1-888-84 22937 | |
Locations Investigative Site Calgary, Canada, T3B 6A8 | Status Completed | Contact Us: E-mail: [email protected] Phone: (+)1-888-84 22937 | |
Locations Investigative Site Wien, Austria, 1090 | Status Completed | Contact Us: E-mail: [email protected] Phone: (+)1-888-84 22937 | |
Locations Investigative Site Pensacola, United States, 32504 | Status Completed | Contact Us: E-mail: [email protected] Phone: (+)1-888-84 22937 | |
Locations Investigative Site Bristol, United Kingdom, BS2 8AE | Status Completed | Contact Us: E-mail: [email protected] Phone: (+)1-888-84 22937 | |
Locations Investigative Site Esplugues de LLobregat, Spain, 08950 | Status Completed | Contact Us: E-mail: [email protected] Phone: (+)1-888-84 22937 | |
Locations Investigative Site Christchurch, New Zealand, 8011 | Status Completed | Contact Us: E-mail: [email protected] Phone: (+)1-888-84 22937 | |
Locations Investigative Site Oslo, Norway, 0027 | Status Completed | Contact Us: E-mail: [email protected] Phone: (+)1-888-84 22937 | |
Locations Investigative Site Thessaloniki, Greece, 54642 | Status Completed | Contact Us: E-mail: [email protected] Phone: (+)1-888-84 22937 | |
Locations Investigative Site Hamilton, New Zealand, 3204 | Status Completed | Contact Us: E-mail: [email protected] Phone: (+)1-888-84 22937 | |
Locations Investigative Site Salt Lake City, United States, 84113 | Status Completed | Contact Us: E-mail: [email protected] Phone: (+)1-888-84 22937 |
Trial Design
A multi-center, phase III, non-controlled, open-label trial to evaluate the pharmacokinetics, safety, and efficacy of BAY94-9027 for prophylaxis and treatment of bleeding in previously treated children (age <12 years) with severe hemophilia A
Trial Type:
Interventional
Intervention Type:
Biological/Vaccine
Trial Purpose:
Treatment
Allocation:
Non-randomized
Blinding:
Open Label
Assignment:
Single Group Assignment
Trial Arms:
3
Primary Outcome
- Annualized number of all bleedsTimeframe: At least 50 exposure days (ED) over 6 months, on average 245 days
- Pharmacokinetics profile of BAY94-9027 based on blood concentration over the defined time periodPharmacokinetics profile includes maximum concentration (Cmax), half-life (t1/2), area under the concentration versus time curve (AUC), mean residence time (MRT), volume of distribution at steady state (Vss), and clearance (CL)Timeframe: Pre-dose to 72 hours post-dose
- Response of acute bleeding events to treatment based on a 4-point scale (poor, moderate, good, or excellent)Timeframe: At least 50 exposure days (ED) over 6 months, on average 245 days
- Characterization of a potential immune responseTimeframe: 12 weeks
- Inhibitor development in the extension studyTimeframe: At least 50 additional EDs to achieve at least 100 cumulative EDs, on average 5 years
Secondary Outcome
- Inhibitor development in the main studyTimeframe: After 10 to 15 and 50 exposure days (ED) over 6 months, on average 245 days
- Assessment of incremental recovery in main studyTimeframe: At least 50 exposure days (ED) over 6 months, on average 245 days
- Number of participants with adverse events as a measure of safety and tolerabilityTimeframe: From the start of study treatment up to 7 days after the last dose (Main study: on average 245+7 days; Part 2: 12 weeks+7 days; Extension study: on average 5 years+7 days)
Additional Information
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