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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
Last Update Posted : February 22, 2019
Sponsor:
Information provided by (Responsible Party):
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 PK data.

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

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 58 participants
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

Arm Intervention/treatment
Experimental: Human-cl rhFVIII Biological: Human cl rhFVIII



Primary Outcome Measures :
  1. 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


Secondary Outcome Measures :
  1. 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

  2. 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

  3. Median prophylactic dosing interval [ Time Frame: 2.5 years ]
    Median prophylactic dosing interval reported in days

  4. Usage of Human-cl rhFVIII (FVIII IU/kg bw per month per patient) [ Time Frame: 2.5 years ]
    Usage of Human-cl rhFVIII reported as IU/kg bw per month per patient

  5. Number of patients with Adverse events [ Time Frame: 2.5 years ]
    Number and percentage of patients experiencing adverse events during the study



Information from the National Library of Medicine

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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+ cound > 200/ul)

Exclusion Criteria:

  • Any coagulation disorder other than Haemophilia A
  • Present of past FVIII inhibitor activity
  • Severe liver or kidney disease

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


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Sponsors and Collaborators
Octapharma
Investigators
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Principal Investigator: Craig M Kessler, MD Georgetown University

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Responsible Party: Octapharma
ClinicalTrials.gov Identifier: NCT02256917     History of Changes
Other Study ID Numbers: GENA-21B
First Posted: October 6, 2014    Key Record Dates
Last Update Posted: February 22, 2019
Last Verified: February 2019
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
Factor VIII
Coagulants