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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.

Phase 1
Conditions
Haemophilia A
MedDRA version: 14.1Level: LLTClassification code 10018937Term: Haemophilia ASystem Organ Class: 100000004850
Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
Registration Number
EUCTR2012-001445-40-GB
Lead Sponsor
Green Cross Corporation
Brief Summary

Not available

Detailed Description

Not available

Recruitment & Eligibility

Status
ot Recruiting
Sex
All
Target Recruitment
124
Inclusion Criteria

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 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 both local and central laboratories 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

8.Normal liver and kidney function.

9.Platelet count = 100,000 µL

10.Normal prothrombin time or International Normalized Ratio (INR) < 1.5

11.Subjects receiving therapy for human immunodeficiency virus (HIV) or hepatitis must be on a stable treatment regimen

12.Subjects must be able to withhold FVIII infusions for approximately 72 h prior to each FVIII activity and inhibitor assay

13.Absolute CD4 lymphocyte cell count = 200 µL

14.Signed the written informed consent form or informed consent was obtained from the subject’s legal guardian

15.Females must not be lactating or pregnant at Screening or Baseline

16.All females will be considered to be of childbearing potential unless they are postmenopausal or have been sterilized surgically

17.Willing and able to comply with all aspects of the protocol
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

Exclusion Criteria

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 hemophilia 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 angiomata on physical examination and/or a history of esophageal hemorrhage or documented esophageal varices

8.Uncontrolled hypertension (diastolic blood pressure >100 mm Hg)

9.Hemoglobin < 10 g.dL

10.HIV disease symptoms regardless of presence of HIV antibodies

11.Routine administration (or planned routine administration during the course of the study), of immunosuppressive or immunomodulating drugs other than anti-retroviral therapy (e.g., steroids, beta-interferon)

12.Severe renal dysfunction (creatinine > 2x upper limit of normal [ULN], total bilirubin > 2x the ULN)

13.Liver disease (alanine aminotransferase [ALT], aspartate aminotransferase [ AST] > 3x the ULN)

14.History of diabetes or other metabolic disease

15.History of hypersensitivity or serious adverse reaction to recombinant or plasma-derived FVIII concentrate

16.History of pretreatment prior to the administration of FVIII products (e.g., of antihistamines)

17.Regular use of antifibrinolytics or medications affecting platelet function

18.Hypersensitivity to hamster-or mouse derived proteins

19.Blood transfusions within 30 days of enrollment into the study

20.Current participation in another investigational drug or device study, or participated in a clinical study involving an investigational drug or device within 30 days of enrollment into the study

21.Unable or unwilling to cooperate with study procedures

22.Females who are pregnant (positive ß-hCG test) or breastfeeding

Study & Design

Study Type
Interventional clinical trial of medicinal product
Study Design
Not specified
Primary Outcome Measures
NameTimeMethod
Secondary Outcome Measures
NameTimeMethod
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