Now Available for Public Comment: Notice of Proposed Rulemaking (NPRM) for FDAAA 801 and NIH Draft Reporting Policy for NIH-Funded Trials

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

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Octapharma
ClinicalTrials.gov Identifier:
NCT00989196
First received: September 30, 2009
Last updated: December 16, 2013
Last verified: December 2013

September 30, 2009
December 16, 2013
May 2010
October 2011   (final data collection date for primary outcome measure)
The Area Under the Concentration Curve for Human-cl rhFVIII Compared to Kogenate FS [ Time Frame: 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. ] [ Designated as safety issue: No ]
After the infusion of 50IU/kg bw of Human-cl rhFVIII and Kogenate FS respectively, FVIII activity levels were measured at various time points before and after the infusion. FVIII level results derived from the chromogenic FVIII assay were used to calculate the mean of the area under the curve normalized to the dose administered.
Not Provided
Complete list of historical versions of study NCT00989196 on ClinicalTrials.gov Archive Site
  • Pharmacokinetic Parameter: Invivo Half-life (T1/2) for Human-cl rhFVIII Compared to Kogenate FS [ Time Frame: 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. ] [ Designated as safety issue: No ]
    After the infusion of 50IU/kg bw of Human-cl rhFVIII and Kogenate FS respectively, FVIII activity levels were measured at various time points before and after the infusion. FVIII level results derived from the chromogenic FVIII assay were used to calculate the mean of the area under the curve normalized to the dose administered.
  • Maximum Plasma Concentration (Cmax) for Human-cl rhFVIII Compared to Kogenate FS [ Time Frame: 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. ] [ Designated as safety issue: No ]
    After the infusion of 50IU/kg bw of Human-cl rhFVIII and Kogenate FS respectively, FVIII activity levels were measured at various time points before and after the infusion. FVIII level results derived from the chromogenic FVIII assay were used to calculate the mean of the area under the curve normalized to the dose administered.
  • Time to Reach Maximum Plasma Concentration (Tmax) for Human-cl rhFVIII Compared to Kogenate FS [ Time Frame: 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. ] [ Designated as safety issue: No ]
    After the infusion of 50IU/kg bw of Human-cl rhFVIII and Kogenate FS respectively, FVIII activity levels were measured at various time points before and after the infusion. FVIII level results derived from the chromogenic FVIII assay were used to calculate the mean of the area under the curve normalized to the dose administered.
  • Mean Residence Time (MRT) for Human-cl rhFVIII Compared to Kogenate FS [ Time Frame: 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. ] [ Designated as safety issue: No ]
    After the infusion of 50IU/kg bw of Human-cl rhFVIII and Kogenate FS respectively, FVIII activity levels were measured at various time points before and after the infusion. FVIII level results derived from the chromogenic FVIII assay were used to calculate the mean of the area under the curve normalized to the dose administered.
  • Volume of Distribution at Steady State (Vss) for Human-cl rhFVIII Compared to Kogenate FS [ Time Frame: 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. ] [ Designated as safety issue: No ]
    After the infusion of 50IU/kg bw of Human-cl rhFVIII and Kogenate FS respectively, FVIII activity levels were measured at various time points before and after the infusion. FVIII level results derived from the chromogenic FVIII assay were used to calculate the mean of the area under the curve normalized to the dose administered.
  • Clearance (CL) for Human-cl rhFVIII Compared to Kogenate FS [ Time Frame: 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. ] [ Designated as safety issue: No ]
    After the infusion of 50IU/kg bw of Human-cl rhFVIII and Kogenate FS respectively, FVIII activity levels were measured at various time points before and after the infusion. FVIII level results derived from the chromogenic FVIII assay were used to calculate the mean of the area under the curve normalized to the dose administered.
  • Efficacy of On-demand Treatment of Bleeding Episodes [ Time Frame: From 1st treatment after PK cycle 2 until study end. ] [ Designated as safety issue: No ]

    After each infusion of IMP and at the end of a BE, the following efficacy assessment is made by the subject (together with the Investigator in case of on-site treatment):

    Excellent: Abrupt pain relief and/or unequivocal improvement in objective signs of bleeding within approximately 8 hours after a single infusion.

    Good: Definite pain relief and/or improvement in signs of bleeding within approximately 8 - 12 hours after an infusion requiring up to 2 infusions for complete resolution.

    Moderate: Probable or slight beneficial effect within approximately 12 hours after the first infusion requiring more than two infusions for complete resolution.

    None: No improvement within 12 hours, or worsening of symptoms, requiring more than 2 infusions for complete resolution.

    The assessment was made at the end of a BE in case more than one infusion was needed.

  • Immunogenicity (Number of Patients That Developed an Inhibitor During the Course of the Study) [ Time Frame: 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 ] [ Designated as safety issue: Yes ]
    Inhibitor activity was determined by the modified Bethesda assay (Nijmegen modification) at 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 some patients who may finish the study before they achieve 50 EDs), with human-cl rhFVIII (i.e. at the study completion visit).
Not Provided
Not Provided
Not Provided
 
Clinical Study to Investigate the Pharmacokinetics, Efficacy, Safety and Immunogenicity of a Recombinant FVIII in Patients With Severe Hemophilia 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

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.

Not Provided
Interventional
Phase 2
Allocation: Randomized
Endpoint Classification: Pharmacokinetics Study
Intervention Model: Crossover Assignment
Masking: Open Label
Primary Purpose: Treatment
Hemophilia A
  • Biological: Human-cl rhFVIII
    50 IU/kg for PK dose
  • Biological: Kogenate FS
    50 IU/kg for PK dose
  • Experimental: Human-cl rhFVIII
    Intervention: Biological: Human-cl rhFVIII
  • Active Comparator: Kogenate FS
    Intervention: Biological: Kogenate FS
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Completed
22
September 2012
October 2011   (final data collection date for primary outcome measure)

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
Male
12 Years to 65 Years
No
Contact information is only displayed when the study is recruiting subjects
United States,   Bulgaria,   Germany
 
NCT00989196
GENA-01
Yes
Octapharma
Octapharma
Not Provided
Study Director: Sigurd Knaub, PhD Octapharma
Octapharma
December 2013

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP