Alleviate 1: NN7170-4213

Principal Investigator: Hassan Yaish
Keywords: Pharmacokinetics , Turoctocog , haemophilia Department: Pediatric Administration
IRB Number: 00096330 Co Investigator:  
Specialty: Pediatric Hematology and Oncology
Sub Specialties: Hemophilia
Recruitment Status: Recruiting

Contact Information

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

Brief Summary

Purpose

  • The purpose of this trial is to evaluate safety (including immunogenicity), local tolerability, pharmacokinetics (PK), and preliminary efficacy of SC N8-GP in previously treated patients with haemophilia A.

 

Primary objective

  • To evaluate the safety of s.c. administration of turoctocog alfa pegol (SC N8-GP) in patients with severe haemophilia A
Secondary objectives
  • To evaluate the pharmacokinetics of SC N8-GP in patients with severe haemophilia A
  • To evaluate the local tolerability of SC N8-GP in patients with severe haemophilia A
  • To evaluate the preliminary efficacy of SC N8-GP in terms of bleeding prevention during three months daily treatment in patients with severe haemophilia A

Inclusion Criteria

Inclusion criterion number 2 differs between part A (18 years and above with body mass index (BMI) ≤ 35.0) and part B (12 years and above, no BMI restrictions). For that reason the inclusion criteria are outlined separately. 
 
Inclusion criteria for part A
 
1. Informed consent obtained before any trial-related activities. Trial-related activities are any procedures that are carried out as part of the trial, including activities to determine suitability
for the trial.
 
2. Male, age above or equal to 18 years at the time of signing informed consent.
 
3. Diagnosis of congenital haemophilia A based on medical records (FVIII activity <1%).
 
4. History of more than 150 exposure days to any Factor VIII containing products, fulfilling the criteria for previously treated patients.
 
5. Body mass index (BMI) ≤ 35.0 kg/m2.
 
 
Inclusion criteria for part B
 
1. Informed consent obtained before any trial-related activities. Trial-related activities are any procedures that are carried out as part of the trial, including activities to determine suitability for the trial.
 
2. Male, age above or equal to 12 years at the time of signing informed consent. The first six patients exposed in part B must be aged above or equal to 18 years
 
3. Diagnosis of congenital haemophilia A based on medical records (FVIII activity <1%).
 
4. History of more than 150 exposure days to any Factor VIII containing products, fulfilling the criteria for previously treated patients.

Exclusion Criteria

The exclusion criteria are identical for both parts of the trial.

Exclusion criteria
 
1. Known or suspected hypersensitivity to trial products or related products.
 
2. Previous participation in this trial. Participation is defined as signed informed consent. (Patients who have completed part A are allowed to also participate in part B. If so, a
separate informed consent covering part B must be signed.)
 
3. Participation in any clinical trial of an approved or non-approved investigational medicinal product within 1 month before screening.
 
4. Any disorder, except for conditions associated with Haemophilia A, which in the investigator’s opinion might jeopardise patient’s safety or compliance with the protocol.
 
5. Mental incapacity, unwillingness to cooperate, or a language barrier precluding adequate understanding and cooperation.
 
6. Immune compromised patients due to HIV infection (defined as viral load ≥400.000 copies/mL and/or CD4+ lymphocyte count ≤200/μL performed at screening or defined by medical records no older than 6 months).
 
7. Any history of FVIII inhibitors (defined by medical records within 8 years ofrandomisation).
 
8. Inhibitors to FVIII (≥ 0.6 BU) at screening, measured by Nijmegen modified Bethesda method at central laboratory.
 
9. Known congenital or acquired coagulation disorders other than haemophilia A.
 
10. Major surgery within one month prior to trial start or planned during the trial.