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Completed

NCT Number: NCT02256917

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

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.

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Key information

Conditions

Severe Haemophilia A Blood Coagulation Disorders Blood Coagulation Disorders, Inherited Coagulation Protein Disorders Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Hematologic Diseases Hemic and Lymphatic Diseases Hemophilia A Hemorrhagic Disorders

Age range

18 year and older

Sex eligibility

Male

Study type

Interventional

Phase

Phase 3

Primary location

Octapharma Research Site, Edmonton, Alberta, Canada

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Human cl rhFVIII

Biological

Primary outcomes

  1. 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

Secondary outcomes

  1. 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

  2. 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

  3. 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

  4. 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

  5. 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

  6. 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

  7. 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

  8. 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

  9. 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

  10. 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

  11. 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

  12. 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

Sponsors and collaborators

Lead sponsor

Octapharma

Industry

Registry information

Official study title

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

Important dates

Study start
2015
Primary completion
2018
Study completion
2018
First posted
Oct 6, 2014
Registry last updated
Jan 19, 2021

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

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This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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