check_circleStudy Completed
Hemophilia A
Bayer Identifier:
15912
ClinicalTrials.gov Identifier:
EudraCT Number:
EU CT Number:
Not Available
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.
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.
Key Participants Requirements
Sex
MaleAge
NaN - 12 YearsTrial summary
Enrollment Goal
73Trial Dates
May 2013 - February 2020Phase
Phase 3Could I Receive a placebo
NoProducts
Jivi (Damoctocog, BAY94-9027)Accepts Healthy Volunteer
NoWhere to participate
Status | Institution | Location |
---|---|---|
Withdrawn | Debrecen, 4032, Hungary | |
Withdrawn | Budapest, 1089, Hungary | |
Completed | Milano, 20122, Italy | |
Completed | Padova, 35128, Italy | |
Completed | Palermo, 90127, Italy | |
Completed | Ramat Gan, 5262000, Israel | |
Completed | UTRECHT, 3584 CX, Netherlands | |
Completed | AMSTERDAM, Netherlands | |
Withdrawn | Boston, 02115, United States | |
Completed | Columbus, 43205-2696, United States | |
Completed | Varna, 9010, Bulgaria | |
Completed | Plovdiv, 4002, Bulgaria | |
Completed | Sofia, 1527, Bulgaria | |
Completed | Cincinnati, 45229, United States | |
Completed | Cleveland, 44106-6007, United States | |
Completed | Hershey, 17033, United States | |
Withdrawn | Chicago, 60612, United States | |
Completed | LEUVEN, 3000, Belgium | |
Completed | La Plata, 1900, Argentina | |
Completed | Timisoara, 300011, Romania | |
Completed | Bucharest, 011026, Romania | |
Completed | Bucharest, 022328, Romania | |
Withdrawn | Little Rock, 72202, United States | |
Completed | GENT, 9000, Belgium | |
Withdrawn | Corrientes, 3400, Argentina | |
Completed | Newcastle Upon Tyne, NE1 4LP, United Kingdom | |
Completed | Lodz, 91-738, Poland | |
Withdrawn | Szczecin, 71-252, Poland | |
Completed | Olsztyn, 10-561, Poland | |
Completed | Vilnius, 08661, Lithuania | |
Completed | Sacramento, 95817, United States | |
Completed | Manchester, M13 9WL, United Kingdom | |
Completed | Sheffield, S10 2TH, United Kingdom | |
Completed | Toronto, M5G 1X8, Canada | |
Completed | Calgary, T3B 6A8, Canada | |
Completed | Wien, 1090, Austria | |
Completed | Pensacola, 32504, United States | |
Completed | Bristol, BS2 8AE, United Kingdom | |
Withdrawn | Frankfurt, 60596, Germany | |
Completed | Esplugues de LLobregat, 08950, Spain | |
Withdrawn | Sevilla, 41013, Spain | |
Completed | Christchurch, 8011, New Zealand | |
Completed | Oslo, 0027, Norway | |
Completed | Thessaloniki, 54642, Greece | |
Withdrawn | Ljubljana, 1525, Slovenia | |
Completed | Hamilton, 3204, New Zealand | |
Completed | Salt Lake City, 84113, United States |
Primary Outcome
- Annualized number of all bleedsdate_rangeTime Frame:At least 50 exposure days (ED) over 6 months, on average 245 daysenhanced_encryptionNoSafety Issue:
- 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)date_rangeTime Frame:Pre-dose to 72 hours post-doseenhanced_encryptionNoSafety Issue:
- Response of acute bleeding events to treatment based on a 4-point scale (poor, moderate, good, or excellent)date_rangeTime Frame:At least 50 exposure days (ED) over 6 months, on average 245 daysenhanced_encryptionNoSafety Issue:
- Characterization of a potential immune responsedate_rangeTime Frame:12 weeksenhanced_encryptionYesSafety Issue:
- Inhibitor development in the extension studydate_rangeTime Frame:At least 50 additional EDs to achieve at least 100 cumulative EDs, on average 5 years
Secondary Outcome
- Inhibitor development in the main studydate_rangeTime Frame:After 10 to 15 and 50 exposure days (ED) over 6 months, on average 245 daysenhanced_encryptionNoSafety Issue:
- Assessment of incremental recovery in main studydate_rangeTime Frame: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 tolerabilitydate_rangeTime Frame: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)enhanced_encryptionYesSafety Issue:
Trial design
Trial Type
InterventionalIntervention Type
Biological/VaccineTrial Purpose
TreatmentAllocation
Non-randomizedBlinding
Open LabelAssignment
Single Group AssignmentTrial Arms
3Additional Information
Click here and search for drug information provided by the FDA.Click here and search for information on any recalls, market or product safety alerts by the FDA which might have occurred with this product.Click here to find information about studies related to Bayer Healthcare products conducted in Europe.Click here to find results for studies related to Bayer products.