NN7008-3553 Guardian 5

Principal Investigator: Hassan Yaish
Keywords: Turoctocog Alpha , Haemophilia A Department: Pediatric Administration
IRB Number: 00093859 Co Investigator:  
Specialty: Pediatrics, General
Sub Specialties:
Recruitment Status: Recruiting

Contact Information

Rebbecca Hanshew
Rebbecca.Perez@hsc.utah.edu
801-213-9105

Brief Summary

Study Purpose

The research question behind the study is to provide additional documentation of the immunogenicity, and obtain additional clinical data, of turoctocog alfa in the setting of normal clinical practise.

Primary objective
• The primary objective is to assess the incidence rate of FVIII inhibitors (≥0.6 BU for central laboratory analyses, or above the specific local laboratory reference range) during long-term prevention and treatment of bleeds with turoctocog alfa in previously FVIII treated (>150 EDs) patients with severe and moderately severe haemophilia A (FVIII ≤2%).
 
Secondary objective
• To further evaluate the general safety and obtain additional clinical experience of turoctocog alfa in prevention and treatment of bleeds and prevention of bleeds during and after surgery.
 
Endpoint(s)
Primary endpoint
• Incidence rate of FVIII inhibitors (≥0.6 BU for central laboratory analyses, or above the specific local laboratory reference range) represented as the percentage of patients developing inhibitors.
 
Secondary endpoints
Safety endpoints
• Number of adverse reactions (ARs) reported during the study period
• Number of serious adverse reactions (SARs) reported during the study period
 
Efficacy endpoints
• Haemostatic effect of turoctocog alfa in the treatment of bleeds as assessed by the patient or the Physician according to a predefined four point scale: Excellent, Good, Moderate, or None
• Haemostatic effect of turoctocog alfa during surgical procedures as assessed by evaluation according to a predefined four point scale: Excellent, Good, Moderate, or None
• Annualised bleeding rate for patients using turoctocog alfa for preventive treatment
• Annualised bleeding rate for patients using turoctocog alfa for on-demand treatment
• Total consumption of turoctocog alfa per patient (prevention, treatment of bleeds and surgery) per month
• Actual consumption of turoctocog alfa (IU/kg BW/months) for prevention
• Actual consumption of turoctocog alfa per bleed (IU/kg BW/bleeding episode)
• Actual consumption of turoctocog alfa (IU/kg BW) from the day of surgery until the day of return to preventive regimen

Inclusion Criteria

1. Informed consent obtained before any study-related activities. (Study-related activities are any procedure related to recording of data according to the protocol).
2. Previously FVIII treated (>150 EDs at the time of first dosing with turoctocog alfa) male patients with the diagnosis of congenital severe and moderately severe haemophilia A (FVIII ≤2%).
3. The decision to initiate treatment with commercially available turoctocog alfa has been made by the patient/parent and the patient’s treating physician before and independently from the decision to include the patient in this study.
4. Availability of a detailed and reliable patient documentation (patient records, diary, logbook etc.) covering either the last 50 EDs or the last 2 years per patient to confirm treatment modality (i.e. prophylaxis, on-demand or recent surgery) prior to enrolment.
5. A negative FVIII inhibitor test obtained not more than four weeks prior to first dosing with turoctocog alfa.
6. Patients with a history of FVIII inhibitors and who have been immune-tolerized to FVIII through Immune Tolerance Induction treatment must have FVIII plasma recovery level ≥66 % of expected level and a FVIII half- life (T½) of ≥6 h after a 72 h wash-out period (as demonstrated by available medical records).
7. No clinical suspicion of HIV-1 or, if HIV-1 seropositive, viral load <400.000 copies/mL and immunocompetent with CD4+ lymphocyte count ≥200/μL, as assessed during the last 6 months prior to the Baseline visit.

Exclusion Criteria

1. Contraindications for use according to the approved product information text (US Package Insert (PI), European Summary of Product Characteristics (SmPC) or corresponding local prescribing information). This includes known or suspected allergy to turoctocog alfa or related products.
2. Previous participation and/or withdrawal from this study. Participation is defined as having given informed consent in this study.
3. Treatment with any investigational drug within 30 days prior to enrolment into the study
4. Mental incapacity, unwillingness or language barriers precluding adequate understanding or cooperation.
5. Previous participation in any clinical trial with turoctocog alfa.
6. Treatment with other FVIII products after initiation of treatment with turoctocog alfa.