Clinical trial NCT01775618: Safety and Efficacy of BAY94-9027 in Previously Treated Male Children With Haemophilia A
Official title: 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
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Hemophilia A
Keywords: Hemophilia A
Factor VIII
Prophylaxis
Pediatric
Lead sponsor: Bayer
Sponsor class: INDUSTRY
First posted: 2013-01-25
Enrollment: 73
Sex: MALE
Maximum age (years): 12
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.


Eligibility criteria: 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

Primary outcomes:
measure: Annualized number of all bleeds
timeFrame: At least 50 exposure days (ED) over 6 months, on average 245 days
measure: Pharmacokinetics profile of BAY94-9027 based on blood concentration over the defined time period
timeFrame: Pre-dose to 72 hours post-dose
measure: 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
measure: Characterization of a potential immune response
timeFrame: 12 weeks
measure: Inhibitor development in the extension study
timeFrame: At least 50 additional EDs to achieve at least 100 cumulative EDs, on average 5 years
Secondary outcomes:
measure: Inhibitor development in the main study
timeFrame: After 10 to 15 and 50 exposure days (ED) over 6 months, on average 245 days
measure: Assessment of incremental recovery in main study
timeFrame: At least 50 exposure days (ED) over 6 months, on average 245 days
measure: Number of participants with adverse events as a measure of safety and tolerability
timeFrame: 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)
Other outcomes:
Locations:
city: Sacramento
country: United States
state: California
city: Pensacola
country: United States
state: Florida
city: Cincinnati
country: United States
state: Ohio
city: Cleveland
country: United States
state: Ohio
city: Columbus
country: United States
state: Ohio
city: Hershey
country: United States
state: Pennsylvania
city: Salt Lake City
country: United States
state: Utah
city: La Plata
country: Argentina
state: Buenos Aires
city: Wien
country: Austria
city: Gent
country: Belgium
city: Leuven
country: Belgium
city: Plovdiv
country: Bulgaria
city: Sofia
country: Bulgaria
city: Varna
country: Bulgaria
city: Calgary
country: Canada
state: Alberta
city: Toronto
country: Canada
state: Ontario
city: Thessaloniki
country: Greece
city: Ramat Gan
country: Israel
city: Milano
country: Italy
state: Lombardia
city: Palermo
country: Italy
state: Sicilia
city: Padova
country: Italy
state: Veneto
city: Vilnius
country: Lithuania
city: Amsterdam
country: Netherlands
city: Utrecht
country: Netherlands
city: Christchurch
country: New Zealand
city: Hamilton
country: New Zealand
city: Oslo
country: Norway
city: Lodz
country: Poland
city: Olsztyn
country: Poland
city: Bucharest
country: Romania
city: Bucharest
country: Romania
city: Timisoara
country: Romania
city: Esplugues de LLobregat
country: Spain
state: Barcelona
city: Newcastle Upon Tyne
country: United Kingdom
state: Tyne And Wear
city: Bristol
country: United Kingdom
city: Manchester
country: United Kingdom
city: Sheffield
country: United Kingdom
Study officials:
affiliation: Bayer
name: Bayer Study Director
role: STUDY_DIRECTOR
References:
citation: 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.