Study of Recombinant Factor VIII Fc Fusion Protein (rFVIIIFc) in Subjects With Severe Hemophilia A

Phase 1

Completed

• Conditions: Severe Hemophilia A
• Interventions: Drug: rFVIIIFc

• Registration Number: NCT01027377
• Lead Sponsor: Bioverativ Therapeutics Inc.

Brief Summary

The study is to investigate the safety, tolerability, and pharmacokinetics (the determination of the concentration of the administered drug in blood over time) of recombinant Factor VIII Fc fusion protein (rFVIIIFc) in previously-treated subjects with severe hemophilia A.

Detailed Description

Not available

Study Timeline

• Study Start: 2009-12
• Study First Submitted: 2009-12-04
• Study First Submitted QC: 2009-12-04
• Study First Posted: 2009-12-07
• Primary Completion: 2010-07
• Study Completion: 2011-05
• Status Verified: 2018-08
• Last Update Submitted: 2020-12-16
• Last Update Posted: 2020-12-19

Recruitment & Eligibility

• Status: COMPLETED
• Sex: Male
• Target Recruitment: 16

Inclusion Criteria

1. Male and 12 years of age and older and weigh at least 40 kg
2. Diagnosed with severe hemophilia A (baseline Factor VIII level less than 1%)
3. History of at least 100 exposure days to any Factor VIII product

Exclusion Criteria

1. History of Factor VIII inhibitors
2. Kidney or liver dysfunction
3. Diagnosed with another coagulation defect other than hemophilia A

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: CROSSOVER

Arm && Interventions

Group	Intervention	Description
A	rFVIIIFc	Cohort to receive a single low dose intravenous injection of commercially available rFVIII with safety and PK assessments followed by a single low dose intravenous injection of rFVIIIFc with safety and PK assessments
B	rFVIIIFc	Cohort to receive a single high dose intravenous injection of commercially available rFVIII with safety and PK assessments followed by a single high dose intravenous injection of rFVIIIFc with safety and PK assessments

Primary Outcome Measures

Name	Time	Method
Safety will be assessed by physical examination, vital signs, laboratory changes overtime, adverse events, and assessment of inhibitor (Bethesda) and antibody development against rFVIIIFc.	78 weeks

Secondary Outcome Measures

Name	Time	Method
Pharmacokinetic parameters will be measured such as, but not limited to Tmax, Cmax, t1/2, CL, Vd, AUC, MRT, and incremental recovery.	78 weeks
To determine the pharmacokinetics parameters after single administration of 25 and 65 IU/kg of rFVIIFc compared to advate	78 weeks
To determine the pharmacodynamic activity of FVIII over time for both doses of rFVIIIFc	78 weeks

Trial Locations

Locations (1)

Research Site; 🇮🇱 Tel Aviv, Israel