Gena-21b

Principal Investigator: Hassan Yaish
Keywords: Prophylaxis , Hemophilia , rhFVIII Department: Pediatric Administration
IRB Number: 00077654 Co Investigator:  
Specialty: Hematology/BMT
Sub Specialties:
Recruitment Status: Recruiting

Contact Information

Tiffanie Hales
tiffanie.hales@hsc.utah.edu
8012133367

Brief Summary

Primary Objective
• To compare the annualised total bleeding rate of individually tailored prophylaxis with the historical bleeding rate observed in patients having received on-demand treatment with Human-cl rhFVIII from study GENA-01
 
Secondary Objectives
1. To compare the annualised spontaneous bleeding rate of individually tailored prophylaxis with the historical bleeding rate observed in patients having received on-demand treatment with Human-cl rhFVIII
2. To compare the annualised total bleeding rate in patients with 2x/week (or less) prophylaxis with the historical bleeding rate observed in patients having received on-demand treatment with Human-cl rhFVIII
3. To assess the median prophylactic dosing interval
4. To assess the PK of Human-cl rhFVIII in terms of FVIII:C
5. To assess the safety of Human-cl rhFVIII
 
Additional Objectives
1. To assess the clinical efficacy of Human-cl rhFVIII in the treatment of breakthrough bleeding episodes (BEs)
2. To assess the clinical efficacy of Human-cl rhFVIII in surgical prophylaxis
3. To assess the correlation of VWF antigen concentration and half-life of Human-cl rhFVIII
4. To assess the association between ABO blood type and half-life of Human-cl
rhFVIII
5. To assess Human-cl rhFVIII consumption data (exploratory)

Inclusion Criteria

Inclusion Criteria
(a) Severe haemophilia A (FVIII:C < 1%) according to medical history
(b) Male patients ≥ 18 years of age
(c) Previous treatment with a FVIII concentrate (regular prophylaxis with good compliance or on-demand treatment) for at least 150 EDs
(d) Good documentation regarding dosing and bleeding frequency in the 6 months preceding study start
(e) Immunocompetence (CD4+ count > 200/μL)
(f) Freely given written informed consent

Exclusion Criteria

Exclusion Criteria
(a) Any coagulation disorder other than haemophilia A
(b) Present or past FVIII inhibitor activity (≥ 0.6 BU) according to medical history
(c) Severe liver or kidney disease (ALT and AST levels > 5 times of upper limit of normal, creatinine > 120 μmol/L)
(d) Treatment with any investigational medicinal product (IMP) except FVIII IMP within 14 days prior to the screening visit