Safety and Efficacy of turoctocog alfa pegol (N8-GP) in Previously Untreated Patients with Haemophilia A

• Register: EUCTR
• Last refreshed on: 3 November 2014
• Main ID: EUCTR2013-004025-88-ES
• Date of registration: 06/03/2014
• Prospective Registration: Yes
• Primary sponsor: Novo Nordisk A/S
• Public title: Safety and Efficacy of turoctocog alfa pegol (N8-GP) in Previously Untreated Patients with Haemophilia A
• Scientific title: Safety and Efficacy of turoctocog alfa pegol (N8-GP) in Previously Untreated Patients with Haemophilia A An open-label single-arm multicentre non-controlled phase 3a trial investigating safety and efficacy of N8-GP in prophylaxis and treatment of bleeding episodes in previously untreated paediatric patients with severe haemophilia A - pathfinder?6
• Date of first enrolment: 28/04/2014
• Target sample size: 125
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2013-004025-88
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: no Randomised: no Open: no Single blind: no Double blind: no Parallel group: no Cross over: no Other: no If controlled, specify comparator, Other Medicinial Product: no Placebo: no Other: no

Countries of recruitment

Algeria, Argentina, Australia, Austria, Canada, China, European Union, Germany, Greece, Israel, Italy, Japan, Korea, Republic of, Malaysia, Mexico, Serbia, Spain, Taiwan, Thailand, Turkey, Ukraine, United States

Contacts

• Name: Global Clinical Registry (GCR,1452)
• Affiliation: Novo Nordisk A/S

• Name: Global Clinical Registry (GCR,1452)
• Affiliation: Novo Nordisk A/S

Key inclusion & exclusion criteria

Inclusion criteria:
- 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
- Male, age < 6 years at the time of signing informed consent
- Diagnosis of severe haemophilia A (factor VIII activity level < 1%) based on medical records or central laboratory results
- No prior use of purified clotting factor products (5 previous exposure to blood components is acceptable)
Are the trial subjects under 18? yes
Number of subjects for this age range: 125
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion criteria:
- Any history of FVIII inhibitor (defined by medical records)
- Known or suspected hypersensitivity to trial product or related products
- Previous participation in this trial. Participation is defined as administration of trial product
- Receipt of any investigational medicinal product within 30 days before screening
- Congenital or acquired coagulation disorder other than haemophilia A. Any chronic disorder or severe disease which, in the opinion of the Investigator, might jeopardise patient?s safety or compliance with the protocol.
- Patient?s parent(s)/legally acceptable representative(s) mental incapacity, unwillingness to cooperate, or a language barrier precluding adequate understanding and cooperation


Age minimum:
Age maximum:
Gender:
Female: no
Male: yes

Health Condition(s) or Problem(s) studied

Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

Haemophilia A
MedDRA version: 16.1 Level: LLT Classification code 10018938 Term: Haemophilia A (Factor VIII) System Organ Class: 100000004850

Intervention(s)

Product Name: N8-GP rFVIII
Pharmaceutical Form: Powder and solvent for solution for injection
INN or Proposed INN: TUROCTOCOG ALFA PEGOL
Current Sponsor code: NNC129-1003
Concentration unit: U unit(s)
Concentration type: equal
Concentration number: 2000-

Product Name: N8-GP 500 U vial
Pharmaceutical Form: Powder and solvent for solution for injection
INN or Proposed INN: TUROCTOCOG ALFA PEGOL
Current Sponsor code: NNC129-1003
Concentration unit: U unit(s)
Concentration type: equal
Concentration number: 500-

Primary Outcome(s)

Main Objective: To evaluate immunogenicity of N8-GP (turoctocog alfa pegol) in previously untreated patients (PUPs) with severe haemophilia A

Timepoint(s) of evaluation of this end point: All objectives/endpoints will be evaluated when the first 50 PUP have reached at least 50 exposure dates, when the first 100 PUP have reached 100 exposure dates, and at end of trial. End of trial will be up to 4 years after the patient has reached 100 exposure dates.

Secondary Objective: - To evaluate safety other than immunogenicity of N8-GP (turoctocog alfa pegol) in PUPs with severe haemophilia A
- To evaluate efficacy of N8-GP (turoctocog alfa pegol) in PUPs with severe haemophilia A
o in long-term prophylaxis treatment (bleeding preventive effect)
o in the treatment of bleeding episodes

Primary end point(s): Incidence of FVIII inhibitors

Secondary Outcome(s)

Timepoint(s) of evaluation of this end point: All objectives/endpoints will be evaluated when the first 50 PUP have reached at least 50 exposure dates, when the first 100 PUP have reached 100 exposure dates, and at end of trial. End of trial will be up to 4 years after the patient has reached 100 exposure dates.

Secondary end point(s): - Frequency of adverse events including serious adverse events and medical events of special interest.
- Incidence of confirmed high titre inhibitors (defined as inhibitor titre > 5BU).
- Number of breakthrough bleeding episodes during prophylaxis with N8-GP (annualised bleeding rate).
- Haemostatic effect of N8-GP in treatment of bleeding episodes, assessed by a predefined 4-point haemostatic response scale ?excellent?, ?good?, ?moderate? and ?none?).

Secondary ID(s)

2013-004025-88-DE

NN7088-3908

Source(s) of Monetary Support

Novo Nordisk A/S