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Assess the Efficacy and Safety of Personalized Prophylaxis Human-cl rhFVIII in Patients With Severe Haemophilia A

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ClinicalTrials.gov Identifier: NCT02256917
Recruitment Status : Completed
First Posted : October 6, 2014
Results First Posted : December 3, 2019
Last Update Posted : January 19, 2021
Sponsor:
Information provided by (Responsible Party):
Octapharma

Tracking Information
First Submitted Date  ICMJE September 30, 2014
First Posted Date  ICMJE October 6, 2014
Results First Submitted Date  ICMJE August 28, 2019
Results First Posted Date  ICMJE December 3, 2019
Last Update Posted Date January 19, 2021
Actual Study Start Date  ICMJE May 2015
Actual Primary Completion Date September 2018   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: November 13, 2019)		 Annualized Total Bleeding Rate of Individually Tailored Prophylaxis [ Time Frame: 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
Original Primary Outcome Measures  ICMJE
 (submitted: October 1, 2014)		 Annualized total bleeding rate of individually tailored prophylaxis [ Time Frame: 2.5 years ]
Annualized total bleeding rate of individually tailored prophylaxis and compare to historical data from the GENA-01 study
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: November 13, 2019)
  • Annualized Spontaneous Bleeding Rate of Individually Tailored Prophylaxis [ Time Frame: 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
  • Annualized Total Bleeding Rate in Patients With 2x/Week (or Less) Prophylaxis [ Time Frame: 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
  • Median Prophylactic Dosing Interval [ Time Frame: 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
  • Mean Prophylactic Dosing Interval [ Time Frame: 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
  • AUC Divided by the Dose (AUCnorm) of Human-cl rhFVIII [ Time Frame: 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
  • In-vivo Recovery (IVR) of Human-cl rhFVIII [ Time Frame: 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
  • Half Life (t1/2) of Human-cl rhFVIII [ Time Frame: 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
  • Mean Residence Time (MRT) of Human-cl rhFVIII [ Time Frame: 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
  • Clearance (CL) of Human-cl rhFVIII [ Time Frame: 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
  • Volume of Distribution at Steady State (Vss) of Human-cl rhFVIII [ Time Frame: 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) [ Time Frame: 6 months ]
    Average weekly consumption of Human-cl rhFVIII reported as IU/kg BW per week per patient was determined during individualized prophylactic treatment
  • Number of Patients With Adverse Events (AEs) [ Time Frame: 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
Original Secondary Outcome Measures  ICMJE
 (submitted: October 1, 2014)
  • Annualized spontaneous bleeding rate of individually tailored prophylaxis [ Time Frame: 2.5 years ]
    Annualized spontaneous bleeding rate of individually tailored prophylaxis compared to historical bleeding rate in patients having received on-demand treatment with Human-cl rhFVIII
  • Annualized total bleeding rate in patients with 2x/week (or less) prophylaxis [ Time Frame: 2.5 years ]
    Annualized total bleeding rate in patients with 2x/week (or less) prophylaxis compared to historical bleeding rate in patients having received on-demand treatment with Human-cl rhFVIII
  • Median prophylactic dosing interval [ Time Frame: 2.5 years ]
  • Usage of Human-cl rhFVIII (FVIII IU/kg bw per month per patient) [ Time Frame: 2.5 years ]
  • Number of patients with Adverse events [ Time Frame: 2.5 years ]
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Assess the Efficacy and Safety of Personalized Prophylaxis Human-cl rhFVIII in Patients With Severe Haemophilia A
Official Title  ICMJE Prospective, Open-label, Multi-centre Phase 3b Study to Assess the Efficacy and Safety of Personalized Prophylaxis With Human-cl rhFVIII in Previously Treated Adult Patients With Severe Haemophilia A
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 Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Prevention
Condition  ICMJE Severe Haemophilia A
Intervention  ICMJE Biological: Human cl rhFVIII
Study Arms  ICMJE Experimental: Human-cl rhFVIII
Intervention: Biological: Human cl rhFVIII
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: October 8, 2018)		 58
Original Estimated Enrollment  ICMJE
 (submitted: October 1, 2014)		 55
Actual Study Completion Date  ICMJE September 2018
Actual Primary Completion Date September 2018   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  • 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)

Exclusion Criteria:

  • Any coagulation disorder other than Haemophilia A
  • Present of past FVIII inhibitor activity
  • Severe liver or kidney disease
Sex/Gender  ICMJE
Sexes Eligible for Study: Male
Ages  ICMJE 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Canada,   Croatia,   Finland,   France,   Japan,   Netherlands,   North Macedonia,   Slovenia,   United States
Removed Location Countries Macedonia, The Former Yugoslav Republic of
 
Administrative Information
NCT Number  ICMJE NCT02256917
Other Study ID Numbers  ICMJE GENA-21B
Has Data Monitoring Committee No
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Current Responsible Party Octapharma
Original Responsible Party Same as current
Current Study Sponsor  ICMJE Octapharma
Original Study Sponsor  ICMJE Same as current
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Principal Investigator: Craig M Kessler, MD Georgetown University
PRS Account Octapharma
Verification Date December 2020

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