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

Bayer80 enrolled

Overview

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.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Hemophilia A

Interventions

rFVIII (BAY81-8973) on demandBIOLOGICAL

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

rFVIII (BAY81-8973) prophylaxis low-doseBIOLOGICAL

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.

rFVIII (BAY81-8973) prophylaxis high-doseBIOLOGICAL

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.

Eligibility

Sex: MALEMin age: 12 YearsMax age: 65 Years

Medical Language ↔ Plain English

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

Locations (47)

Outcomes

Primary Outcomes

Annualized Number of All Bleeds

The annualized number of bleeds experienced by participants

Time frame: Up to 12 months (6 months per mode of potency assignment according to the randomized cross-over design)

Secondary Outcomes

Annualized Number of All Bleeds During CS/EP Period

The annualized number of bleeds experienced by participants while they were taking rFVIII (BAY81-8973) assayed by CS/EP

Time frame: Up to 6 months (6 months on CS/EP potency assignment)

Annualized Number of All Bleeds During CS/ADJ Period

The annualized number of bleeds experienced by participants while they were taking rFVIII (BAY81-8973) assayed by CS/ADJ

Time frame: Up to 6 months (6 months on CS/ADJ potency assignment)

Percentage of Bleeds Per Participant Controlled With ≤ 2 Injections in Participants Treated on Demand With rFVIII (BAY81-8973)

The percentage of bleeds per participant on on-demand treatment that stopped after two or fewer injections

Time frame: Up to 12 months (6 months per mode of potency assignment according to the randomized cross-over design)

Data from ClinicalTrials.gov

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Dayton, Ohio, United States

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Houston, Texas, United States

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Buenos Aires, Ciudad Auton. de Buenos Aires, Argentina

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Corrientes, Corrientes Province, Argentina

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Rosario, Santa Fe Province, Argentina

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Guangzhou, Guangdong, China

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Suzhou, Jiangsu, China

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Beijing, China

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Shanghai, China

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Tianjin, China

...and 37 more locations