A Trial to Compare Prophylaxis Therapy to On-demand Therapy With a New Full Length Recombinant FVIII in Patients With Severe Hemophilia A

NCT ID: NCT01233258

Last Updated: 2016-11-28

Study Results

Results available

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE3

Total Enrollment

80 participants

Study Classification

INTERVENTIONAL

Study Start Date

2011-01-31

Study Completion Date

2012-12-31

Brief Summary

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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.

Detailed Description

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Conditions

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Hemophilia A

Keywords

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Haemophilia treatment rFVIII

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

CROSSOVER

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Arm 1: rFVIII on demand first CS/EP then CS/ADJ

Participants received on-demand treatment with recombinant factor VIII (rFVIII, BAY81-8973) assayed by CS/EP (Chromogenic Substrate Assay per European Pharmacopoeia) for 6 months, followed by cross-over to study drug assayed by CS/ADJ (Chromogenic Substrate Assay/label adjusted to one-stage assay) for 6 months.

Group Type EXPERIMENTAL

rFVIII (BAY81-8973) on demand

Intervention Type BIOLOGICAL

Participants received on-demand treatment with rFVIII (BAY81-8973) assayed by CS/EP (Chromogenic Substrate Assay per European Pharmacopoeia) for 6 months and by CS/ADJ (Chromogenic Substrate Assay/label adjusted to one-stage assay) for 6 months, sequence according to randomization

Arm 2: rFVIII on demand first CS/ADJ then CS/EP

Participants received on-demand treatment with rFVIII (BAY81-8973) assayed by CS/ADJ for 6 months, followed by cross-over to study drug assayed by CS/EP for 6 months.

Group Type EXPERIMENTAL

rFVIII (BAY81-8973) on demand

Intervention Type BIOLOGICAL

Participants received on-demand treatment with rFVIII (BAY81-8973) assayed by CS/EP (Chromogenic Substrate Assay per European Pharmacopoeia) for 6 months and by CS/ADJ (Chromogenic Substrate Assay/label adjusted to one-stage assay) for 6 months, sequence according to randomization

Arm 3: rFVIII prophylaxis low-dose first CS/EP then CS/ADJ

Participants received low dose prophylaxis treatment at 20, 25 or 30 IU/kg twice per week with rFVIII(BAY81-8973) measured by CS/ EP for 6 months then crossed over to study drug measured by CS/ADJ for 6 months.

Group Type EXPERIMENTAL

rFVIII (BAY81-8973) prophylaxis low-dose

Intervention Type BIOLOGICAL

Participants received low dose prophylaxis treatment at 20, 25 or 30 IU/kg twice per week with rFVIII (BAY81-8973) assayed by CS/EP for 6 months and by CS/ADJ for 6 months, sequence according to randomization.

Arm 4: rFVIII prophylaxis low-dose first CS/ADJ then CS/EP

Participants received low dose prophylaxis treatment at 20, 25 or 30 IU/kg twice per week with rFVIII (BAY81-8973) measured by CS/ADJ for 6 months then crossed over to study drug measured by CS/ EP for 6 months.

Group Type EXPERIMENTAL

rFVIII (BAY81-8973) prophylaxis low-dose

Intervention Type BIOLOGICAL

Participants received low dose prophylaxis treatment at 20, 25 or 30 IU/kg twice per week with rFVIII (BAY81-8973) assayed by CS/EP for 6 months and by CS/ADJ for 6 months, sequence according to randomization.

Arm 5: rFVIII prophylaxis high-dose first CS/EP then CS/ADJ

Participants received high dose prophylaxis treatment at 30, 35 or 40 IU/kg 3 times per week with rFVIII (BAY81-8973) measured by CS/ EP for 6 months then crossed over to study drug measured by CS/ADJ for 6 months.

Group Type EXPERIMENTAL

rFVIII (BAY81-8973) prophylaxis high-dose

Intervention Type BIOLOGICAL

Participants received high dose prophylaxis treatment at 30, 35 or 40 IU/kg 3 times per week with rFVIII (BAY81-8973) assayed by CS/EP for 6 months and by CS/ADJ for 6 months, sequence according to randomization.

Arm 6: rFVIII prophylaxis high-dose first CS/ADJ then CS/EP

Participants received high dose prophylaxis treatment at 30, 35 or 40 IU/kg 3 times per week with rFVIII(BAY81-8973) measured by CS/ADJ for 6 months then crossed over to study drug measured by CS/ EP for 6 months.

Group Type EXPERIMENTAL

rFVIII (BAY81-8973) prophylaxis high-dose

Intervention Type BIOLOGICAL

Participants received high dose prophylaxis treatment at 30, 35 or 40 IU/kg 3 times per week with rFVIII (BAY81-8973) assayed by CS/EP for 6 months and by CS/ADJ for 6 months, sequence according to randomization.

Interventions

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rFVIII (BAY81-8973) on demand

Participants received on-demand treatment with rFVIII (BAY81-8973) assayed by CS/EP (Chromogenic Substrate Assay per European Pharmacopoeia) for 6 months and by CS/ADJ (Chromogenic Substrate Assay/label adjusted to one-stage assay) for 6 months, sequence according to randomization

Intervention Type BIOLOGICAL

rFVIII (BAY81-8973) prophylaxis low-dose

Participants received low dose prophylaxis treatment at 20, 25 or 30 IU/kg twice per week with rFVIII (BAY81-8973) assayed by CS/EP for 6 months and by CS/ADJ for 6 months, sequence according to randomization.

Intervention Type BIOLOGICAL

rFVIII (BAY81-8973) prophylaxis high-dose

Participants received high dose prophylaxis treatment at 30, 35 or 40 IU/kg 3 times per week with rFVIII (BAY81-8973) assayed by CS/EP for 6 months and by CS/ADJ for 6 months, sequence according to randomization.

Intervention Type BIOLOGICAL

Eligibility Criteria

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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
Minimum Eligible Age

12 Years

Maximum Eligible Age

65 Years

Eligible Sex

MALE

Accepts Healthy Volunteers

No

Sponsors

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Bayer

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Bayer Study Director

Role: STUDY_DIRECTOR

Bayer

Locations

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Dayton, Ohio, United States

Site Status

Houston, Texas, United States

Site Status

Buenos Aires, Ciudad Auton. de Buenos Aires, Argentina

Site Status

Corrientes, Corrientes Province, Argentina

Site Status

Rosario, Santa Fe Province, Argentina

Site Status

Guangzhou, Guangdong, China

Site Status

Suzhou, Jiangsu, China

Site Status

Beijing, , China

Site Status

Shanghai, , China

Site Status

Tianjin, , China

Site Status

Bogotá, Bogota D.C., Colombia

Site Status

Bucaramanga, Santander Department, Colombia

Site Status

Olomouc, , Czechia

Site Status

Hyderabad, , India

Site Status

Jakarta, , Indonesia

Site Status

Hiroshima, Hiroshima, Japan

Site Status

Nishinomiya, Hyōgo, Japan

Site Status

Kashihara, Nara, Japan

Site Status

Shinjuku-ku, Tokyo, Japan

Site Status

Suginami, Tokyo, Japan

Site Status

Guadalajara, Jalisco, Mexico

Site Status

San Luis Potosí City, San Luis Potosí, Mexico

Site Status

Timișoara, Timiș County, Romania

Site Status

Baia Mare, , Romania

Site Status

Bucharest, , Romania

Site Status

Bucharest, , Romania

Site Status

Barnaul, , Russia

Site Status

Khabarovsk, , Russia

Site Status

Saint Petersburg, , Russia

Site Status

Yekaterinburg, , Russia

Site Status

Belgrade, , Serbia

Site Status

Kragujevac, , Serbia

Site Status

Niš, , Serbia

Site Status

Novi Sad, , Serbia

Site Status

Bratislava, , Slovakia

Site Status

Johannesburg, Gauteng, South Africa

Site Status

Pretoria, Gauteng, South Africa

Site Status

Taichung, , Taiwan

Site Status

Taipei, , Taiwan

Site Status

Bangkok, Thailand, Thailand

Site Status

Bangkok, Thailand, Thailand

Site Status

Adana, , Turkey (Türkiye)

Site Status

Antalya, , Turkey (Türkiye)

Site Status

Izmir, , Turkey (Türkiye)

Site Status

Kiev, , Ukraine

Site Status

Lviv, , Ukraine

Site Status

Simferopol, , Ukraine

Site Status

Countries

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United States Argentina China Colombia Czechia India Indonesia Japan Mexico Romania Russia Serbia Slovakia South Africa Taiwan Thailand Turkey (Türkiye) Ukraine

References

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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.

Reference Type RESULT
PMID: 27002680 (View on PubMed)

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.

Reference Type RESULT
PMID: 26931631 (View on PubMed)

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.

Reference Type DERIVED
PMID: 25546368 (View on PubMed)

Related Links

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http://www.clinicaltrialsregister.eu/

Click here to find information about studies related to Bayer Healthcare products conducted in Europe

Other Identifiers

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2009-012150-20

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

14319

Identifier Type: -

Identifier Source: org_study_id