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rFVIII on demand

Phase 3

Hemophilia A | Monoclonal antibody | Hematology |Bayer AG|Last Updated: Nov 28, 2016

Success Probability

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Market & Valuation

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Trial Design

RandomizedCONTROLLEDDMC
Total Trials1
Total Enrollment80

FDA Designations

No designations recorded

Clinical trial landscape

rFVIII on demand · 1 trial · 1 indication

Phase 3 1
NCT01233258A Trial to Compare Prophylaxis Therapy to On-demand Therapy With a New Full Length Recombinant FVIII in Patients With Severe Hemophilia AHemophilia A
COMPLETED80 Analytics
PHASE3COMPLETED
A Trial to Compare Prophylaxis Therapy to On-demand Therapy With a New Full Length Recombinant FVIII in Patients With Severe Hemophilia A
Hemophilia AUnlock trial analytics

Study Endpoints

Primary Endpoints

Annualized Number of All Bleeds
Up to 12 months (6 months per mode of potency assignment according to the randomized cross-over design)

The annualized number of bleeds experienced by participants

Secondary Endpoints

Annualized Number of All Bleeds During CS/EP Period
Up to 6 months (6 months on CS/EP potency assignment)
Annualized Number of All Bleeds During CS/ADJ Period
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)
Up to 12 months (6 months per mode of potency assignment according to the randomized cross-over design)
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Study Design & Arms

AllocationRANDOMIZED
MaskingNONE
ModelCROSSOVER
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
Arm 1: rFVIII on demand first CS/EP then CS/ADJEXPERIMENTALParticipants 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.
Arm 2: rFVIII on demand first CS/ADJ then CS/EPEXPERIMENTALParticipants 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.
Arm 3: rFVIII prophylaxis low-dose first CS/EP then CS/ADJEXPERIMENTALParticipants 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.
Arm 4: rFVIII prophylaxis low-dose first CS/ADJ then CS/EPEXPERIMENTALParticipants 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.
Arm 5: rFVIII prophylaxis high-dose first CS/EP then CS/ADJEXPERIMENTALParticipants 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.
Arm 6: rFVIII prophylaxis high-dose first CS/ADJ then CS/EPEXPERIMENTALParticipants 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.

Interventions

NameTypeDescription
rFVIII (BAY81-8973) on demandBIOLOGICALParticipants 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-doseBIOLOGICALParticipants 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-doseBIOLOGICALParticipants 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.
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Eligibility Criteria

Age Range12 Years to 65 Years
SexMALE
Healthy VolunteersNo
Study Sites47

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 Facto...

Countries:United StatesArgentinaChinaColombiaCzechiaIndiaIndonesiaJapanMexicoRomaniaRussiaSerbiaSlovakiaSouth AfricaTaiwanThailandTurkey (Türkiye)Ukraine
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Frequently asked questions about rFVIII on demand

What is rFVIII on demand used for?

rFVIII on demand is a recombinant factor VIII therapy used for the on-demand treatment of bleeding episodes in patients with severe Hemophilia A. It is administered to control bleeding when it occurs, rather than as a preventive prophylaxis regimen.

Who makes rFVIII on demand?

rFVIII on demand is developed by Bayer AG, a German pharmaceutical company traded on the OTC market under the ticker BAYRY. The company is conducting clinical trials to evaluate this therapy in patients with severe Hemophilia A.

What phase is rFVIII on demand in?

rFVIII on demand is in Phase 3 clinical development. It is an investigational therapy and has not been approved by regulatory authorities. A Phase 3 trial comparing prophylaxis to on-demand therapy has been completed.

What clinical trials is rFVIII on demand in?

rFVIII on demand was evaluated in a completed Phase 3 trial, NCT01233258, which enrolled 80 male patients aged 12 years and older with severe Hemophilia A. The trial compared prophylaxis therapy to on-demand therapy and was conducted across multiple countries including the United States, Argentina, China, and Japan.

Is rFVIII on demand the same as a full length recombinant FVIII?

rFVIII on demand refers to the on-demand treatment regimen using a new full length recombinant factor VIII. The clinical trial NCT01233258 specifically evaluated this full length recombinant FVIII product when given on-demand, as opposed to prophylactic dosing.