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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

Study Description
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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.

Condition or disease Intervention/treatment Phase
Severe Haemophilia A Biological: Human cl rhFVIII Phase 3

Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 58 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Prevention
Official 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
Actual Study Start Date : May 2015
Actual Primary Completion Date : September 2018
Actual Study Completion Date : September 2018

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Hemophilia
Drug Information available for: Chlorine

Arms and Interventions
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Arm Intervention/treatment
Experimental: Human-cl rhFVIII Biological: Human cl rhFVIII


Outcome Measures
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Primary Outcome Measures :
  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 Outcome Measures :
  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


Eligibility Criteria
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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

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
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02256917


Locations
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Sponsors and Collaborators
Octapharma
Investigators
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Principal Investigator: Craig M Kessler, MD Georgetown University
  Study Documents (Full-Text)

Documents provided by Octapharma:
Study Protocol  [PDF] July 24, 2019
Statistical Analysis Plan  [PDF] October 30, 2018

More Information
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Responsible Party: Octapharma
ClinicalTrials.gov Identifier: NCT02256917    
Other Study ID Numbers: GENA-21B
First Posted: October 6, 2014    Key Record Dates
Results First Posted: December 3, 2019
Last Update Posted: January 19, 2021
Last Verified: December 2020
Additional relevant MeSH terms:
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Hemophilia A
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
 Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn