Clinical trial NCT01233258: A Trial to Compare Prophylaxis Therapy to On-demand Therapy With a New Full Length Recombinant FVIII in Patients With Severe Hemophilia A
Official title: A Phase II/III, Randomized, Cross-over, Open-label Trial to Demonstrate Superiority of Prophylaxis Over On-demand Therapy in Previously Treated Subjects With Severe Hemophilia A Treated With Plasma Protein-free Recombinant FVIII Formulated With Sucrose (BAY 81-8973)
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Hemophilia A
Keywords: Haemophilia treatment
rFVIII
Lead sponsor: Bayer
Sponsor class: INDUSTRY
First posted: 2010-11-03
Enrollment: 80
Sex: MALE
Minimum age (years): 12
Maximum age (years): 65
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: CROSSOVER
Primary purpose: TREATMENT

Brief summary: The objective of the trial is to demonstrate that 2-3 times per week prophylaxis therapy with BAY81-8973 is superior to on-demand therapy with BAY81-8973 in patients with severe Hemophilia A. The hypothesis is that prophylaxis will result in fewer bleeds than on-demand treatment.


Eligibility criteria: Inclusion Criteria:

* Male, aged 12 to 65 years
* Severe hemophilia A
* History of more than 150 exposure days (ED) with clotting factor concentrates
* Currently receiving episodic treatment with FVIII; no regular prophylaxis for more than 6 consecutive months in the past 5 years
* No current Factor VIII inhibitor or history of inhibitor
* Willing to use electronic patient diary

Exclusion Criteria:

* Presence of another bleeding disease that is different from hemophilia A
* Thrombocytopenia
* Abnormal renal function
* Presence of active liver disease
* Known hypersensitivity to FVIII

Primary outcomes:
measure: Annualized Number of All Bleeds
timeFrame: Up to 12 months (6 months per mode of potency assignment according to the randomized cross-over design)
Secondary outcomes:
measure: Annualized Number of All Bleeds During CS/EP Period
timeFrame: Up to 6 months (6 months on CS/EP potency assignment)
measure: Annualized Number of All Bleeds During CS/ADJ Period
timeFrame: Up to 6 months (6 months on CS/ADJ potency assignment)
measure: Percentage of Bleeds Per Participant Controlled With ≤ 2 Injections in Participants Treated on Demand With rFVIII (BAY81-8973)
timeFrame: Up to 12 months (6 months per mode of potency assignment according to the randomized cross-over design)
Other outcomes:
measure: Number of Bleeds During Treatment
timeFrame: 12 months
measure: Number of Participants With Inhibitory Antibody Formation
timeFrame: 3, 6, 9 and 12 months after baseline
Locations:
city: Dayton
country: United States
state: Ohio
city: Houston
country: United States
state: Texas
city: Buenos Aires
country: Argentina
state: Ciudad Auton. de Buenos Aires
city: Rosario
country: Argentina
state: Santa Fe
city: Corrientes
country: Argentina
city: Guangzhou
country: China
state: Guangdong
city: Suzhou
country: China
state: Jiangsu
city: Beijing
country: China
city: Shanghai
country: China
city: Tianjin
country: China
city: Bogotá
country: Colombia
state: Distrito Capital de Bogotá
city: Bucaramanga
country: Colombia
state: Santander
city: Olomouc
country: Czech Republic
city: Hyderabad
country: India
city: Jakarta
country: Indonesia
city: Nishinomiya
country: Japan
state: Hyogo
city: Kashihara
country: Japan
state: Nara
city: Shinjuku-ku
country: Japan
state: Tokyo
city: Suginami
country: Japan
state: Tokyo
city: Hiroshima
country: Japan
city: Guadalajara
country: Mexico
state: Jalisco
city: San Luis Potosí
country: Mexico
city: Timisoara
country: Romania
state: Timis
city: Baia Mare
country: Romania
city: Bucharest
country: Romania
city: Bucharest
country: Romania
city: Barnaul
country: Russian Federation
city: Khabarovsk
country: Russian Federation
city: St. Petersburg
country: Russian Federation
city: Yekaterinburg
country: Russian Federation
city: Beograd
country: Serbia
city: Kragujevac
country: Serbia
city: Nis
country: Serbia
city: Novi Sad
country: Serbia
city: Bratislava
country: Slovakia
city: Johannesburg
country: South Africa
state: Gauteng
city: Pretoria
country: South Africa
state: Gauteng
city: Taichung
country: Taiwan
city: Taipei
country: Taiwan
city: Bangkok
country: Thailand
city: Bangkok
country: Thailand
city: Adana
country: Turkey
city: Antalya
country: Turkey
city: Izmir
country: Turkey
city: Kiev
country: Ukraine
city: Lviv
country: Ukraine
city: Simferopol
country: Ukraine
Study officials:
affiliation: Bayer
name: Bayer Study Director
role: STUDY_DIRECTOR
References:
citation: Kitchen S, Katterle Y, Beckmann H, Maas Enriquez M. Chromogenic assay for BAY 81-8973 potency assignment has no impact on clinical outcome or monitoring in patient samples. J Thromb Haemost. 2016 Jun;14(6):1192-9. doi: 10.1111/jth.13322. Epub 2016 May 3.
citation: Oldenburg J, Windyga J, Hampton K, Lalezari S, Tseneklidou-Stoeter D, Beckmann H, Maas Enriquez M. Safety and efficacy of BAY 81-8973 for surgery in previously treated patients with haemophilia A: results of the LEOPOLD clinical trial programme. Haemophilia. 2016 May;22(3):349-53. doi: 10.1111/hae.12839. Epub 2016 Mar 1.
citation: Kavakli K, Yang R, Rusen L, Beckmann H, Tseneklidou-Stoeter D, Maas Enriquez M; LEOPOLD II Study Investigators. Prophylaxis vs. on-demand treatment with BAY 81-8973, a full-length plasma protein-free recombinant factor VIII product: results from a randomized trial (LEOPOLD II). J Thromb Haemost. 2015 Mar;13(3):360-9. doi: 10.1111/jth.12828.