Recent Updates
Recently added Catalysts

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

Molecular targetF8
Target classExogenous Protein
ModalityMonoclonal antibody

Also known as Antihemophilic Factor (Recombinant), Antihemophilic Factor

Success Probability

Subscribe to view

Market & Valuation

Subscribe to view

Trial Design

RandomizedACTIVE_CONTROLLEDDMC
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
Unlock Study Endpoints

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.
Unlock Study Design Details

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
Unlock Eligibility Criteria

Frequently asked questions about Antihemophilic factor, recombinant, manufactured protein-free

What is antihemophilic factor, recombinant, manufactured protein-free used for?

It is used to treat Hemophilia A, a bleeding disorder caused by deficient or missing clotting factor VIII. The product is a recombinant, protein-free form of factor VIII developed by Takeda Pharmaceutical Company Limited. It is studied for use in patients with Hemophilia A, including those undergoing surgery and pediatric patients.

What does antihemophilic factor, recombinant, manufactured protein-free target?

It targets F8, also known as factor VIII, an exogenous protein. By supplying functional factor VIII, it replaces the clotting factor that is missing or deficient in people with Hemophilia A, supporting normal blood coagulation. The target class is an exogenous protein rather than a small molecule or antibody target.

Who makes antihemophilic factor, recombinant, manufactured protein-free?

Takeda Pharmaceutical Company Limited develops antihemophilic factor, recombinant, manufactured protein-free. Takeda trades under the ticker TAK. The company is the sponsor behind the clinical studies of this recombinant factor VIII product in Hemophilia A.

What phase is antihemophilic factor, recombinant, manufactured protein-free in?

It is in Phase 2 clinical development. Four trials have been completed, with no active trials currently listed. The studies enrolled a total of 243 participants and included randomized, active-controlled designs. It is an investigational product and is not described as approved.

What clinical trials is antihemophilic factor, recombinant, manufactured protein-free in?

Completed trials include NCT00952822, a Phase 1 pharmacokinetic study with 52 participants; NCT00157105, a Phase 2 surgical study with 59 participants; NCT00157053, a Phase 2 continuation study with 82 participants; and NCT00157040, a Phase 2 pediatric study with 50 participants. All four studies were in Hemophilia A.

Is antihemophilic factor, recombinant, manufactured protein-free the same as rAHF-PFM or Factor VIII?

Yes. Antihemophilic factor, recombinant, manufactured protein-free is also known as rAHF-PFM. Antihemophilic factor is also known as Factor VIII, so searches for Antihemophilic Factor (Recombinant) refer to the same product. These names describe the same recombinant, protein-free factor VIII therapy.