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.|
|ClinicalTrials.gov Identifier: NCT01341912|
Recruitment Status : Completed
First Posted : April 26, 2011
Results First Posted : February 3, 2014
Last Update Posted : February 3, 2014
|Condition or disease||Intervention/treatment||Phase|
|Severe Hemophilia A||Biological: Human-cl rhFVIII||Phase 3|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||3 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|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|
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.
- 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.
- 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.
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): NCT01341912
|United States, District of Columbia|
|Washington, District of Columbia, United States, 20057|
|Haematological Hospital SHAT "Joan Pavel"|