To determine the safety, effectiveness, and effect on the body of GreenGene F in previously treated patients 12 years of age or older who have severe Hemophilia A.
- Conditions
- Hemophilia AMedDRA version: 17.0Level: LLTClassification code 10060613Term: Hemophilia A (Factor VIII)System Organ Class: 100000004850Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
- Registration Number
- EUCTR2012-001445-40-HR
- Lead Sponsor
- Green Cross Corporation
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 124
1.Male or female subjects age = 12 years at time of informed consent
2.Body weight = 35 kg
3.Diagnosed with severe hemophilia A. Subjects must have severe hemophilia A with baseline FVIII < 1%, <0.01 IU/mL
4.Have = 150 previous exposure days to FVIII concentrates, as documented in the subject's medical records
5.Subjects included in the on-demand treatment cohort must have a verifiable record of at least three bleeding episodes per month on average in the last 6 months prior to enrollment
6.Negative assays for FVIII inhibitor at inclusion(<0.6BU Nijmegen assay)
7.Negative assays for FVIII inhibitor in subject files(<0.6BU Nijmegen assay). No history of positive inhibitor is allowed
Are the trial subjects under 18? yes
Number of subjects for this age range: 40
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 64
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 20
1.Presence at Screening of FVIII inhibitor = 0.6 BU as tested with the Nijmegen modification of the Bethesda assay in either central or local laboratory
2.History of FVIII inhibitor of = 0.6 BU as measured using the Nijmegen
modification of the Bethesda assay
3.History of FVIII inhibitor = 1.0 BU if the subject has been tested routinely using the original Bethesda assay, or history of periods with low recovery and no response to Factor VIII treatment
4.Demonstrated an inability to respond to conventional doses of FVIII therapy
5.History of incremental recovery of Factor VIII <1.35% per IU/kg infused
6.Hematological disorders or blood coagulation diseases(e.g., idiopathic thrombocytopenic purpura, von Willebrand disease, etc.) other than haemophilia A
7.Laboratory or clinical evidence of portal vein hypertension including, but not limited to, an INR>1.4, the presence of splenomegaly and/or spider aniomata of physical examination and/or a history of esophageal hemorrhage or documented esophageal varices
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Main Objective: •To demonstrate safety of GreenGene™ F with respect to inhibitor development (neutralizing anti-Factor VIII antibodies) and long-term safety<br>•To evaluate the hemostatic efficacy in prophylaxis (rate of breakthrough bleeding) and on demand treatment in the management of acute bleeding events<br>•To evaluate the physicians and subjects rating of response for on demand treatment of bleeding episodes; and<br>•To evaluate peri-operative hemostatic control of GreenGene™ F during surgical and invasive diagnostic procedures.;Secondary Objective: Please see main objective above.;Primary end point(s): Safety and efficacy; The primary safety objective is to demonstrate a sufficiently low incidence of inhibitor associated with the use GreenGene™ F.;Timepoint(s) of evaluation of this end point: Safety and efficacy; Following a minimum 50 exposure days for both prophylaxis and on demand patients
- Secondary Outcome Measures
Name Time Method Secondary end point(s): Safety and efficacy:<br>• Frequency of breakthrough bleeds (total, trauma and joint);<br>• Subject evaluation of efficacy;<br>• Number of infusions per bleeding episodes;<br>• Units of FVIII per infusion per bleeding episode;<br>• Units of FVIII per bleeding; and<br>• Units of FVIII per prophylaxis treatment.;Timepoint(s) of evaluation of this end point: Safety and efficacy; Following a minimum 50 exposure days for both prophylaxis and on demand patients.