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

Severe Hemophilia A Clinical Trial

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01341912
• Other study ID #: GENA-11
• Status: Completed
• Phase: Phase 3
• Start date: June 2011
• Est. completion date: August 2012

Study information

• Verified date: March 2020
• Source: Octapharma
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 3
• Est. completion date: August 2012
• Est. primary completion date: August 2012
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 12 Years to 65 Years

Eligibility

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

Related Conditions & MeSH terms

• Hemophilia A
• Severe Hemophilia A

Intervention

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.

Locations

Country	Name	City	State
Bulgaria	Haematological Hospital SHAT "Joan Pavel"	Sofia
United States	Georgetown University	Washington	District of Columbia

Sponsors (1)

Lead Sponsor	Collaborator
Octapharma

Countries where clinical trial is conducted

United States,  Bulgaria, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Long-term Immunogenicity	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.	up to 3 years
Secondary	To Determine Long-term Efficacy of Human-cl rhFVIII in the Treatment of Bleeding Episodes and in Surgical Prophylaxis	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