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
1 other identifier
interventional
3
2 countries
2
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for phase_3
Started Jun 2011
Shorter than P25 for phase_3
2 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
April 22, 2011
CompletedFirst Posted
Study publicly available on registry
April 26, 2011
CompletedStudy Start
First participant enrolled
June 1, 2011
CompletedPrimary Completion
Last participant's last visit for primary outcome
August 1, 2012
CompletedStudy Completion
Last participant's last visit for all outcomes
August 1, 2012
CompletedResults Posted
Study results publicly available
February 3, 2014
CompletedMarch 17, 2020
March 1, 2020
1.2 years
April 22, 2011
October 11, 2013
March 3, 2020
Conditions
Outcome Measures
Primary Outcomes (1)
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 Outcomes (1)
To Determine Long-term Efficacy of Human-cl rhFVIII in the Treatment of Bleeding Episodes and in Surgical Prophylaxis
up to 3 years
Study Arms (1)
Human-cl rhFVIII
EXPERIMENTALRecombinant FVIII derived from a human cell line.
Interventions
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.
Eligibility Criteria
You may qualify if:
- 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
You may not qualify if:
- 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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- Octapharmalead
Study Sites (2)
Georgetown University
Washington D.C., District of Columbia, 20057, United States
Haematological Hospital SHAT "Joan Pavel"
Sofia, Bulgaria
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Limitations and Caveats
Only 3 patients participated in the study and all 3 patients withdrew consent prematurely. Therefore the results are summarized descriptively only.
Results Point of Contact
- Title
- VP of Clinical Research and Development
- Organization
- Octapharma
Publication Agreements
- PI is Sponsor Employee
- No
- Restriction Type
- OTHER
- Restrictive Agreement
- Yes
Study Design
- Study Type
- interventional
- Phase
- phase 3
- Allocation
- NA
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SINGLE GROUP
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
April 22, 2011
First Posted
April 26, 2011
Study Start
June 1, 2011
Primary Completion
August 1, 2012
Study Completion
August 1, 2012
Last Updated
March 17, 2020
Results First Posted
February 3, 2014
Record last verified: 2020-03