Assess the Efficacy and Safety of Personalized Prophylaxis Human-cl rhFVIII in Patients With Severe Haemophilia A
- Conditions
- Severe Haemophilia A
- Interventions
- Biological: Human cl rhFVIII
- Registration Number
- NCT02256917
- Lead Sponsor
- Octapharma
- Brief Summary
The rationale of this study is to further fine-tune and individualize prophylactic treatment of patients with severe Haemophilia A with the goal of keeping the trough FVIII level above 1% between doses. Because trough FVIII levels are likely to be important predictors of the efficacy of prophylaxis, the focus of this study is on pharmacokinetic (PK) data.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- Male
- Target Recruitment
- 58
- Severe Haemophilia A (FVIII:C < 1%)
- Male patients >= 18 years of age
- Previous treatment with a FVIII concentrate for at least 150 EDs
- Good documentation regarding dosing and bleeding frequency in the 6 months preceding study start
- Immunocompetence (CD4+ count > 200/uL)
- Any coagulation disorder other than Haemophilia A
- Present of past FVIII inhibitor activity
- Severe liver or kidney disease
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description Human-cl rhFVIII Human cl rhFVIII -
- Primary Outcome Measures
Name Time Method Annualized Total Bleeding Rate of Individually Tailored Prophylaxis 6 months Total annualized bleeding rate (ABR) of individually tailored prophylaxis (GENA-21b) compared to historical bleeding rate in patients having received on-demand treatment (GENA-01) with Human-cl rhFVIII
- Secondary Outcome Measures
Name Time Method Annualized Total Bleeding Rate in Patients With 2x/Week (or Less) Prophylaxis 6 months Total annualized bleeding rate (ABR) in patients with 2x/week (or less) prophylaxis (GENA-21b) compared to historical bleeding rate in patients having received on-demand treatment (GENA-01) with Human-cl rhFVIII
Annualized Spontaneous Bleeding Rate of Individually Tailored Prophylaxis 6 months Spontaneous annualized bleeding rate (ABR) of individually tailored prophylaxis (GENA-21b) compared to historical bleeding rate in patients having received on-demand treatment (GENA-01) with Human-cl rhFVIII
Mean Prophylactic Dosing Interval 6 months Mean over mean actual dosing intervals between two prophylactic treatments per patient. The mean time (hours) between two prophylactic doses of Human-cl rhFVIII in the prophylactic treatment Phase II per patient
Mean Residence Time (MRT) of Human-cl rhFVIII Before injection (within 1 h before injection) and up to 72 h (± 2 h) after the end of injection MRT of Human-cl rhFVIII measured using the one-stage (OS) assay
AUC Divided by the Dose (AUCnorm) of Human-cl rhFVIII Before injection (within 1 h before injection) and up to 72 h (± 2 h) after the end of injection AUCnorm of Human-cl rhFVIII measured using the one-stage (OS) assay
Clearance (CL) of Human-cl rhFVIII Before injection (within 1 h before injection) and up to 72 h (± 2 h) after the end of injection CL of Human-cl rhFVIII measured using the one-stage (OS) assay
In-vivo Recovery (IVR) of Human-cl rhFVIII Before injection (within 1 h before injection) and up to 72 h (± 2 h) after the end of injection IVR of Human-cl rhFVIII measured using the one-stage (OS) assay and will be determined from the FVIII level before the infusion and the peak level after the infusion of Human-cl rhFVIII
Number of Patients With Adverse Events (AEs) At each study visit over the study duration (7-9 months) AEs were documented at each (scheduled or unscheduled) study visit. Severity and seriousness of all AEs were documented by the investigator according to pre-defined criteria
Half Life (t1/2) of Human-cl rhFVIII Before injection (within 1 h before injection) and up to 72 h (± 2 h) after the end of injection T1/2 of Human-cl rhFVIII measured using the one-stage (OS) assay
Median Prophylactic Dosing Interval 6 months Median over median actual dosing intervals between two prophylactic treatments per patient. The median time (hours) between two prophylactic doses of Human-cl rhFVIII in the prophylactic treatment Phase II per patient
Volume of Distribution at Steady State (Vss) of Human-cl rhFVIII Before injection (within 1 h before injection) and up to 72 h (± 2 h) after the end of injection Vss of Human-cl rhFVIII measured using the one-stage (OS) assay
Usage of Human-cl rhFVIII (FVIII IU/kg BW Per Week Per Patient) 6 months Average weekly consumption of Human-cl rhFVIII reported as IU/kg BW per week per patient was determined during individualized prophylactic treatment
Trial Locations
- Locations (20)
PHI Institute of Transfusion Medicine of Republic of Macedonia
🇲🇰Skopje, North Macedonia
Ogikubo Hospital
🇯🇵Tokyo, Japan
McMaster University
🇨🇦Hamilton, Ontario, Canada
Centre Régional de Traitement de l'Hémophilie
🇫🇷Bron, France
Centre Hospitalier Universitaire Félix Guyon
🇫🇷La Réunion, France
Helsinki University Hospital
🇫🇮Helsinki, Finland
CHU Estaing
🇫🇷Clermont-Ferrand, France
Centre Régional de Traitement de l'hémophilie
🇫🇷Nantes, France
Hôpital Purpan - Centre de Traitment Regional de l'Hemophilie Pole
🇫🇷Toulouse, France
Nagoya University Hospital
🇯🇵Nagoya, Aichi, Japan
Hospital of the Univ of Occupational and Environmental Health
🇯🇵Kitakyushu, Fukuoka, Japan
St. Marianna Univ School of Medicine Hospital
🇯🇵Kawasaki, Kanagawa, Japan
Nara Medical University Hospital
🇯🇵Kashihara, Nara, Japan
Gunma University Hospital
🇯🇵Maebashi, Japan
Osaka National Hospital
🇯🇵Osaka, Japan
Teikyo University Hospital
🇯🇵Tokyo, Japan
University Medical Center Groningen
🇳🇱Groningen, Netherlands
University Hospital Centre Zagreb
🇭🇷Zagreb, Croatia
Octapharma Research Site
🇨🇦St. John's, Newfoundland and Labrador, Canada
University Medical Centre Ljubljana
🇸🇮Ljubljana, Slovenia