Clinical trial NCT00989196: Clinical Study to Investigate the Pharmacokinetics, Efficacy, Safety and Immunogenicity of a Recombinant FVIII in Patients With Severe Hemophilia A
Official title: 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
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Hemophilia A
Lead sponsor: Octapharma
Sponsor class: INDUSTRY
First posted: 2009-10-02
Enrollment: 22
Sex: MALE
Minimum age (years): 12
Maximum age (years): 65
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: CROSSOVER
Primary purpose: TREATMENT

Brief 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

Primary outcomes:
measure: The Area Under the Concentration Curve for Human-cl rhFVIII Compared to Kogenate FS
timeFrame: At baseline (prior to infusion), 0.25, 0.5, 0.75, 1, 3, 6, 9, 12, 24, 30 and 48 hours after the end of the infusion.
Secondary outcomes:
measure: Invivo Half-life (T1/2) for Human-cl rhFVIII Compared to Kogenate FS
timeFrame: At baseline (prior to infusion), 0.25, 0.5, 0.75, 1, 3, 6, 9, 12, 24, 30 and 48 hours after the end of the infusion.
measure: Maximum Plasma Concentration (Cmax) for Human-cl rhFVIII Compared to Kogenate FS
timeFrame: At baseline (prior to infusion), 0.25, 0.5, 0.75, 1, 3, 6, 9, 12, 24, 30 and 48 hours after the end of the infusion.
measure: Time to Reach Maximum Plasma Concentration (Tmax) for Human-cl rhFVIII Compared to Kogenate FS
timeFrame: At baseline (prior to infusion), 0.25, 0.5, 0.75, 1, 3, 6, 9, 12, 24, 30 and 48 hours after the end of the infusion.
measure: Mean Residence Time (MRT) for Human-cl rhFVIII Compared to Kogenate FS
timeFrame: At baseline (prior to infusion), 0.25, 0.5, 0.75, 1, 3, 6, 9, 12, 24, 30 and 48 hours after the end of the infusion.
measure: Volume of Distribution at Steady State (Vss) for Human-cl rhFVIII Compared to Kogenate FS
timeFrame: At baseline (prior to infusion), 0.25, 0.5, 0.75, 1, 3, 6, 9, 12, 24, 30 and 48 hours after the end of the infusion.
measure: Clearance (CL) for Human-cl rhFVIII Compared to Kogenate FS
timeFrame: At baseline (prior to infusion), 0.25, 0.5, 0.75, 1, 3, 6, 9, 12, 24, 30 and 48 hours after the end of the infusion.
measure: Efficacy of On-demand Treatment of Bleeding Episodes
timeFrame: From 1st treatment after PK cycle 2 until study end.
measure: Immunogenicity (Number of Patients That Developed an Inhibitor During the Course of the Study)
timeFrame: study entry, then immediately before both PK cycles, in the 48 hour sample of both PK cycles, after 10 to 15 EDs with human-cl rhFVIII, at the 3-month visit (± 2 weeks), then every 3 months (± 2 weeks) until study completion, and after >50 EDs (except for
Other outcomes:
Locations:
city: Los Angeles
country: United States
facility: UCLA Orthodpedic Hospital
state: California
city: Sacramento
country: United States
facility: University of California, Davis
state: California
city: Denver
country: United States
facility: University of Colorado
state: Colorado
city: Washington
country: United States
facility: Georgetown University
state: District of Columbia
city: Chicago
country: United States
facility: RUSH University Medical Center
state: Illinois
city: New Brunswick
country: United States
facility: University of Medicine and Dentistry
state: New Jersey
city: Sofia
country: Bulgaria
facility: Prof. Lissitchkov
city: Hannover
country: Germany
facility: Medizinische Hochschule
state: Niedersachsen
city: Berlin
country: Germany
facility: Vivantes Klinikum
Study officials:
affiliation: Octapharma
name: Sigurd Knaub, PhD
role: STUDY_DIRECTOR
References: