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Study to Investigate the Long-term Efficacy and Safety of Human-cl rhFVIII in Previously Treated Patients (PTPs)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT01341912
Recruitment Status : Completed
First Posted : April 26, 2011
Results First Posted : February 3, 2014
Last Update Posted : February 3, 2014
Information provided by (Responsible Party):

Brief Summary:
The purpose of the study is to study the long-term efficacy, safety and tolerability of Human-cl rhFVIII in previously treated patients with severe hemophilia A.

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

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 3 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Clinical Study to Investigate the Long-Term Efficacy, Safety and Immunogenicity of Human-cl rhFVIII in Previously Treated Patients With Severe Haemophilia A - Extension Study to GENA-01
Study Start Date : June 2011
Actual Primary Completion Date : August 2012
Actual Study Completion Date : August 2012

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
Recombinant FVIII derived from a human cell line.
Biological: Human-cl rhFVIII
Human-cl rhFVIII is administered intravenously on demand for bleeding episodes and prophylactically in case of surgery. The dosage depends on the severity of the bleeding episodes and the surgery.

Primary Outcome Measures :
  1. Long-term Immunogenicity [ Time Frame: up to 3 years ]
    Patients will be monitored for inhibitors against FVIII every 3 months. Blood samples were drawn and inhibitor activity was determined by the modified Bethesda assay (Nijmegen modification) in the central lab.

Secondary Outcome Measures :
  1. To Determine Long-term Efficacy of Human-cl rhFVIII in the Treatment of Bleeding Episodes and in Surgical Prophylaxis [ Time Frame: up to 3 years ]

    The efficacy of human-cl rhFVIII will be determined using a 4 point efficacy assessment scale.

    After each infusion of IMP and at the end of a BE, the following efficacy assessment is made by the subject (together with the Investigator in case of on-site treatment):

    Excellent: Abrupt pain relief and/or unequivocal improvement in objective signs of bleeding within approximately 8 hours after a single infusion.

    Good: Definite pain relief and/or improvement in signs of bleeding within approximately 8 - 12 hours after an infusion requiring up to 2 infusions for complete resolution.

    Moderate: Probable or slight beneficial effect within approximately 12 hours after the first infusion requiring more than two infusions for complete resolution.

    None: No improvement within 12 hours, or worsening of symptoms, requiring more than 2 infusions for complete resolution.

    The assessment was made at the end of a BE in case more than one infusion was needed.

Information from the National Library of Medicine

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Ages Eligible for Study:   12 Years to 65 Years   (Child, Adult, Older Adult)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Completion of GENA-01 study with at least 50 Exposure Days (EDs) and at least 6 months study participation and immediate enrollment into GENA-11

Exclusion Criteria:

  • Development of FVIII inhibitors (<=0.6 BU), during the course of the GENA-01 study
  • Development of any severe liver or kidney disease (ALT and AST level > 5 times of upper limit of normal, creatine >120 micro mol/L) during the course of the GENA-01 study

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 identifier (NCT number): NCT01341912

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United States, District of Columbia
Georgetown University
Washington, District of Columbia, United States, 20057
Haematological Hospital SHAT "Joan Pavel"
Sofia, Bulgaria
Sponsors and Collaborators

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Responsible Party: Octapharma Identifier: NCT01341912     History of Changes
Other Study ID Numbers: GENA-11
First Posted: April 26, 2011    Key Record Dates
Results First Posted: February 3, 2014
Last Update Posted: February 3, 2014
Last Verified: December 2013
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