- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT01341912
Study to Investigate the Long-term Efficacy and Safety of Human-cl rhFVIII in Previously Treated Patients (PTPs)
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 Overview
Status
Conditions
Intervention / Treatment
Study Type
Enrollment (Actual)
Phase
- Phase 3
Contacts and Locations
Study Locations
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Sofia, Bulgaria
- Haematological Hospital SHAT "Joan Pavel"
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District of Columbia
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Washington, District of Columbia, United States, 20057
- Georgetown University
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Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Genders Eligible for Study
Description
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
Study Plan
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: N/A
- Interventional Model: Single Group Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
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Experimental: Human-cl rhFVIII
Recombinant FVIII derived from a human cell line.
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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.
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What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Long-term Immunogenicity
Time Frame: up to 3 years
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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.
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up to 3 years
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Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
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
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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. |
up to 3 years
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Collaborators and Investigators
Sponsor
Study record dates
Study Major Dates
Study Start
Primary Completion (Actual)
Study Completion (Actual)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Estimate)
Study Record Updates
Last Update Posted (Actual)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Additional Relevant MeSH Terms
Other Study ID Numbers
- GENA-11
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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