Safety and Efficacy of BAY94-9027 in Previously Treated Male Children With Haemophilia A
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
2 other identifiers
interventional
73
17 countries
37
Brief Summary
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for phase_3
Started May 2013
Longer than P75 for phase_3
37 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
January 23, 2013
CompletedFirst Posted
Study publicly available on registry
January 25, 2013
CompletedStudy Start
First participant enrolled
May 29, 2013
CompletedPrimary Completion
Last participant's last visit for primary outcome
March 19, 2015
CompletedStudy Completion
Last participant's last visit for all outcomes
February 19, 2020
CompletedAugust 21, 2020
August 1, 2020
1.8 years
January 23, 2013
August 19, 2020
Conditions
Keywords
Outcome Measures
Primary Outcomes (5)
Annualized number of all bleeds
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 period
Pharmacokinetics 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)
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)
At least 50 exposure days (ED) over 6 months, on average 245 days
Characterization of a potential immune response
12 weeks
Inhibitor development in the extension study
At least 50 additional EDs to achieve at least 100 cumulative EDs, on average 5 years
Secondary Outcomes (3)
Inhibitor development in the main study
After 10 to 15 and 50 exposure days (ED) over 6 months, on average 245 days
Assessment of incremental recovery in main study
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 tolerability
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)
Study Arms (3)
Main study
EXPERIMENTALParticipants were treated and prophylaxis administered with BAY94-9027 at a dose of 25-60 international units/kilogram (IU/kg) twice per week or 45-60 IU/kg every 5 days or 60 IU/kg every 7 days as an intravenous (IV) infusion as per clinical needs of each subject up to at least 50 exposure days (EDs) and a minimum of at least 6 months.
Part 2 (Expansion group)
EXPERIMENTALParticipants were administered with BAY94-9027 at a dose of 25-60 IU/kg twice per week for prophylaxis for 12 weeks.
Extension study
EXPERIMENTALParticipants were treated and prophylaxis administered with BAY94-9027 at a dose of 25- 60 IU/kg twice per week or 45-60 IU/kg every 5 days or 60 IU/kg every 7 days as an IV infusion as per clinical needs of each subject for at least 50 EDs or until marketing authorization of the drug.
Interventions
Study drug dosing was adjusted to the clinical needs of each subject in the range of 25-60 IU/kg/administration, intravenous infusion, at least 50 EDs and a minimum of at least 6 months
Eligibility Criteria
You may qualify if:
- Males \< 12 years of age
- Subjects with severe hemophilia A
- Previously treated with FVIII for \> 50 exposure days
You may not qualify if:
- 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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- Bayerlead
Study Sites (37)
Unknown Facility
Sacramento, California, 95817, United States
Unknown Facility
Pensacola, Florida, 32504, United States
Unknown Facility
Cincinnati, Ohio, 45229, United States
Unknown Facility
Cleveland, Ohio, 44106-6007, United States
Unknown Facility
Columbus, Ohio, 43205-2696, United States
Unknown Facility
Hershey, Pennsylvania, 17033, United States
Unknown Facility
Salt Lake City, Utah, 84113, United States
Unknown Facility
La Plata, Buenos Aires, 1900, Argentina
Unknown Facility
Vienna, 1090, Austria
Unknown Facility
Ghent, 9000, Belgium
Unknown Facility
Leuven, 3000, Belgium
Unknown Facility
Plovdiv, 4002, Bulgaria
Unknown Facility
Sofia, 1527, Bulgaria
Unknown Facility
Varna, 9010, Bulgaria
Unknown Facility
Calgary, Alberta, T3B 6A8, Canada
Unknown Facility
Toronto, Ontario, M5G 1X8, Canada
Unknown Facility
Thessaloniki, 54642, Greece
Unknown Facility
Ramat Gan, 5262000, Israel
Unknown Facility
Milan, Lombardy, 20122, Italy
Unknown Facility
Palermo, Sicily, 90127, Italy
Unknown Facility
Padua, Veneto, 35128, Italy
Unknown Facility
Vilnius, 08661, Lithuania
Unknown Facility
Amsterdam, Netherlands
Unknown Facility
Utrecht, 3584 CX, Netherlands
Unknown Facility
Christchurch, 8011, New Zealand
Unknown Facility
Hamilton, 3204, New Zealand
Unknown Facility
Oslo, 0027, Norway
Unknown Facility
Lodz, 91-738, Poland
Unknown Facility
Olsztyn, 10-561, Poland
Unknown Facility
Bucharest, 011026, Romania
Unknown Facility
Bucharest, 022328, Romania
Unknown Facility
Timișoara, 300011, Romania
Unknown Facility
Esplugues de Llobregat, Barcelona, 08950, Spain
Unknown Facility
Newcastle upon Tyne, Tyne and Wear, NE1 4LP, United Kingdom
Unknown Facility
Bristol, BS2 8AE, United Kingdom
Unknown Facility
Manchester, M13 9WL, United Kingdom
Unknown Facility
Sheffield, S10 2TH, United Kingdom
Related Publications (1)
Mancuso ME, Biss T, Fischer K, Maas Enriquez M, Steele M, Wang M, Tseneklidou-Stoeter D, Ahuja S, Kenet G. PROTECT VIII kids extension study: Long-term safety and efficacy of BAY 94-9027 (damoctocog alfa pegol) in children with severe haemophilia A. Haemophilia. 2021 May;27(3):434-444. doi: 10.1111/hae.14294. Epub 2021 Mar 16.
PMID: 33724632DERIVED
Related Links
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Study Officials
- STUDY DIRECTOR
Bayer Study Director
Bayer
Study Design
- Study Type
- interventional
- Phase
- phase 3
- Allocation
- NON RANDOMIZED
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SINGLE GROUP
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
January 23, 2013
First Posted
January 25, 2013
Study Start
May 29, 2013
Primary Completion
March 19, 2015
Study Completion
February 19, 2020
Last Updated
August 21, 2020
Record last verified: 2020-08