Safety and Efficacy Extension Study of GreenGene™ F in Previously Treated Patients Diagnosed With Severe Hemophilia A

Hemophilia A Clinical Trial

Official title:

An Open Label Safety and Efficacy Extension Study of GreenGene™ F in Previously Treated Patients Diagnosed With Severe Hemophilia A

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT02027779
• Other study ID #: GreenGene™ F_E_P3
• Status: Recruiting
• Phase: Phase 3
• First received: January 2, 2014
• Last updated: January 3, 2014
• Start date: January 2014
• Est. completion date: February 2016

Study information

• Verified date: January 2014
• Source: Green Cross Corporation
• Contact: Chang Hee Lee, M.D.
• Phone: +82 31 260 9729
• Email: chleedr[@]greencross.com
• Is FDA regulated: No
• Health authority: United States: Food and Drug Administration
• Study type: Interventional

Clinical Trial Summary

This study primarily will address the safety and secondarily will assess efficacy of GreenGene™ F in subjects with severe hemophilia A previously treated ≥50 exposure days with a GreenGene™ F, and without presence inhibitor to FVIII (Factor VIII).

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 150
• Est. completion date: February 2016
• Est. primary completion date: December 2015
• Accepts healthy volunteers: No
• Gender: Both
• Age group: N/A and older

Eligibility

Inclusion Criteria:

1. Subjects must have participated in the "GreenGene™ F_P3", (with Eudra CT number 2012-001445-40) or a pediatric study with GreenGene™ F

2. Have =50 previous exposure days to GreenGene™ F, as documented in the subject's medical records.

3. Negative assays for FVIII inhibitor at inclusion (<0.6BU Nijmegen assay), i.e. at the end of study "GreenGene™ F_P3" for patients entering into this extension study immediately after finishing the previous phase III study.

4. Normal liver and kidney function

5. Platelet count = 100,000?

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

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

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

9. Absolute CD4 lymphocyte cell count = 200?

10. Signed the written informed consent form or informed consent was obtained from the subject's legal guardian

11. Females must not be lactating or pregnant at screening or Baseline (as documented by a negative beta-human chorionic gonadotropin [ß-hCG] test with a minimum sensitivity of 25 IU/L or equivalent units of ß-hCG). A test was obtained more than 72 hours before the first dose of study drug

12. All females will be considered to be of childbearing potential unless they are appropriate age group and without other known or suspected cause) or have been sterilized surgically (i.e. bilateral tubal ligation, total hysterectomy or bilateral oophorectomy, all with surgery at least one month before dosing)

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

Exclusion Criteria:

1. Presence at Screening of FVIII inhibitor = 0.6 BU as tested with the Nijmegen modification of the Bethesda assay.

2. 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 of physical examination and/or a history of esophageal hemorrhage or documented esophageal varices

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

4. Hemoglobin < 10 g/dL

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

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

7. History of diabetes or other metabolic disease

8. History of hypersensitivity or serious adverse reaction to recombinant or plasma-derived FVIII concentrates

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

10. Regular use of antifibrinolytics or medications affecting platelet function

11. Hypersensitivity to hamster- or mouse derived proteins

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

13. 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

14. Unable or unwilling to cooperate with study procedures

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

Study Design

Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Hemophilia A

Intervention

Biological:
• GreenGene™ F
Prophylaxis safety and efficacy substudy: intra venous infusion, 30 ± 10 IU/kg infusions 3 times per week with dose escalation to 45 ± 10 IU/kg if appropriate, for 50 exposure days
• GreenGene™ F
On-demand safety and efficacy substudy: minor bleed = 20 ± 10 IU/kg moderate bleed = 30 ± 10 IU/kg major bleed = 30 - 50 IU/kg

Locations

Country	Name	City	State
United States	Arkansas Children's Hospital	Little Rock	Arkansas
United States	Long Island Jewish Medical Center - Hemophilia Treatment Center	New Hyde Park	New York

Sponsors (2)

Lead Sponsor	Collaborator
Green Cross Corporation	Atlantic Research Group

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Number of subjects with development of inhibitors	Development of neutralizing antibodies (inhibitors) will be followed during the regular visits, average of 3 months.	every 3 months, up to 18 months	Yes