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Antihemophilic factor, recombinant, manufactured protein-free

Phase 2

Hemophilia A | Monoclonal antibody | Hematology |Takeda Pharmaceutical Company Limited|Last Updated: Aug 24, 2021

Target and mechanism

ModalityMonoclonal antibody

Also known as Antihemophilic Factor (Recombinant), Antihemophilic Factor

Success Probability

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Market & Valuation

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Trial Design

RandomizedACTIVE_CONTROLLEDDMCBiomarker
Total Trials4
Total Enrollment243

FDA Designations

No designations recorded

Clinical trial landscape

Antihemophilic factor, recombinant, manufactured protein-free · 4 trials · 1 indication

Phase 2 3Phase 1 1
NCT00157040Study of Pharmacokinetics, Efficacy, and Safety of a Recombinant and Protein-Free Factor VIII (rAHF-PFM) in Pediatric Patients With Hemophilia AHemophilia A
COMPLETED50 Analytics
NCT00157053Study of Pharmacokinetics, Efficacy and Safety of a Recombinant and Protein-Free Factor VIII (rAHF-PFM) in Hemophilia A Patients - A Continuation of Clinical Study 069901Hemophilia A
COMPLETED82 Analytics
NCT00157105Safety and Efficacy Study of a Recombinant and Protein-Free Factor VIII (rAHF-PFM) in Hemophilia A Patients Undergoing SurgeryHemophilia A
COMPLETED59 Analytics
PHASE2COMPLETED
Study of Pharmacokinetics, Efficacy, and Safety of a Recombinant and Protein-Free Factor VIII (rAHF-PFM) in Pediatric Patients With Hemophilia A
Hemophilia AUnlock trial analytics
PHASE2COMPLETED
Study of Pharmacokinetics, Efficacy and Safety of a Recombinant and Protein-Free Factor VIII (rAHF-PFM) in Hemophilia A Patients - A Continuation of Clinical Study 069901
Hemophilia AUnlock trial analytics
PHASE2COMPLETED
Safety and Efficacy Study of a Recombinant and Protein-Free Factor VIII (rAHF-PFM) in Hemophilia A Patients Undergoing Surgery
Hemophilia AUnlock trial analytics

Study Endpoints

Primary Endpoints

The terminal phase half-life of Recombinant Antihemophilic Factor (rAHF-PFM)
Within 30 minutes prior to the pharmacokinetic infusion and at 1 hour ± 5 minutes, 9 ± 1 hour, 24 ± 2 hours, and 48 ± 2 hours after the infusion

Terminal phase half-life of rAHF-PFM was to be determined using the biphasic linear regression model.

Terminal phase half-life for Recombinant Antihemophilic Factor (rAHF-PFM)
Within 30 minutes prior to the pharmacokinetic infusion and at 1 hour ± 5 minutes, 9 ± 1 hour, 24 ± 2 hours, and 48 ± 2 hours after the infusion.
Percent of participants experiencing treatment-related adverse experiences (AEs)
Throughout the study period of approximately 3.5 years
Area Under the Curve
Pharmacokinetic evaluations: 30 minutes pre-infusion up to 48 hours post-infusion

Area under the factor VIII (FVIII) plasma concentration versus time curve (AUC) from 0 to 48 hours estimated using the linear trapezoidal method

Secondary Endpoints

Total Area Under the Curve
Pharmacokinetic evaluations: 30 minutes pre-infusion up to 48 hours post-infusion
Adjusted in Vivo Incremental Recovery
Pharmacokinetic evaluations: 30 minutes pre-infusion to 30 minutes post-infusion
Terminal Half-life
Pharmacokinetic evaluations: 30 minutes pre-infusion up to 48 hours post-infusion
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Study Design & Arms

AllocationNON_RANDOMIZED
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
1EXPERIMENTALADVATE reconstituted in 2 mL sterile water for infusion
2ACTIVE_COMPARATORADVATE reconstituted in 5 mL sterile water for infusion

Interventions

NameTypeDescription
Antihemophilic factor, recombinant, manufactured protein-freeDRUG -
Antihemophilic factor, recombinant, manufactured protein-free (rAHF-PFM). (Antihemophilic factor is also known as Factor VIII)BIOLOGICALSubjects are randomized to receive an infusion of rAHF-PFM reconstituted in 2 mL sterile water for infusion (SWFI) followed (after a wash-out period) by rAHF-PFM reconstituted in 5 mL SWFI or in 5 mL then 2 mL SWFI(cross-over design). Each subject will receive 2 infusions.
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Eligibility Criteria

Age RangeN/A to 6 Years
SexALL
Healthy VolunteersNo
Study Sites12

Inclusion Criteria: * Subject is less than 6 years of age * Severe or moderately-severe hemophilia A as defined by a baseline factor VIII level \<= 2%, documented at screening or on the basis of historical data (e.g., at hemophilia diagnosis) * Documented medical history of at least 50 exposure day...

Countries:United StatesCanadaPuerto RicoAustriaBelgiumFranceGermanyItalySwedenUnited Kingdom
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