Phase 2 N=22 Randomized Treatment

Clinical Study to Investigate the Pharmacokinetics, Efficacy, Safety and Immunogenicity of a Recombinant FVIII in Patients With Severe Hemophilia A

Hemophilia A

Bottom Line

View on ClinicalTrials.gov: NCT00989196 ↗

Enrolled (actual)

Serious AEs

9.1%

Results posted

Jan 2014

Primary outcome: Primary: The Area Under the Concentration Curve for Human-cl rhFVIII Compared to Kogenate FS — 0.39; 0.38 h IU/mL (IU/kg)

Study Design & Population

Study type: Interventional
Phase: Phase 2
Interventions: Human-cl rhFVIII (Biological); Kogenate FS (Biological)
Age: Pediatric, Adult, Older Adult · 12+ yrs
Sex: Male
Sponsor: Octapharma
Primary completion: Oct 2011

Outcome Measures

Outcome	Result	p-value
PRIMARY The Area Under the Concentration Curve for Human-cl rhFVIII Compared to Kogenate FS	0.39; 0.38	—
SECONDARY Invivo Half-life (T1/2) for Human-cl rhFVIII Compared to Kogenate FS	14.73; 16.14	—
SECONDARY Maximum Plasma Concentration (Cmax) for Human-cl rhFVIII Compared to Kogenate FS	1.462; 1.394	—
SECONDARY Time to Reach Maximum Plasma Concentration (Tmax) for Human-cl rhFVIII Compared to Kogenate FS	0.35; 0.34	—
SECONDARY Mean Residence Time (MRT) for Human-cl rhFVIII Compared to Kogenate FS	19.45; 20	—
SECONDARY Volume of Distribution at Steady State (Vss) for Human-cl rhFVIII Compared to Kogenate FS	49.58; 53.32	—
SECONDARY Clearance (CL) for Human-cl rhFVIII Compared to Kogenate FS	2.94; 2.75	—
SECONDARY Efficacy of On-demand Treatment of Bleeding Episodes	60.3; 34.1; 5.5; 0	—
SECONDARY Immunogenicity (Number of Patients That Developed an Inhibitor During the Course of the Study)	—	—

Summary

This is a clinical study to investigate the pharmacokinetics, efficacy, safety and immunogenicity of human-cl rhFVIII, a newly developed human cell-line derived recombinant FVIII concentrate in previously treated patients with severe Hemophilia A.

Eligibility Criteria

Inclusion Criteria

Severe hemophilia A (FVIII:C <= 1%)
Male subjects between 12 and 65 years of age
Body weight 25 kg to 110 kg
Previously treated with FVIII concentrate for at least 150 EDs

Exclusion Criteria

Other coagulation disorder than hemophilia A
Present or past FVIII inhibitor activity

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT00989196). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.