Safety and efficacy of turoctocog alfa in prevention and treatment of bleeds in previously untreated children with Haemophilia A

• Register: EUCTR
• Last refreshed on: 30 April 2019
• Main ID: EUCTR2011-001033-16-DK
• Date of registration: 23/12/2014
• Prospective Registration: Yes
• Primary sponsor: Novo Nordisk A/S
• Public title: Safety and efficacy of turoctocog alfa in prevention and treatment of bleeds in previously untreated children with Haemophilia A
• Scientific title: Safety and Efficacy of turoctocog alfa in Prevention and Treatment of Bleeds in Paediatric Previously Untreated Patients with Haemophilia A - guardian™4
• Date of first enrolment: 10/03/2015
• Target sample size: 60
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2011-001033-16
• 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
• Phase: Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): no Therapeutic confirmatory - (Phase III): yes Therapeutic use (Phase IV): no

Countries of recruitment

Algeria, Austria, China, Denmark, Greece, Hong Kong, Hungary, Japan, Lithuania, Poland, Portugal, Russian Federation, Serbia, Spain, Turkey, United Kingdom, 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 procedure that would not have been performed during normal management of the patient)
- Male patients diagnosed with congenital severe haemophilia A (FVIII = 1%)
- Age < 6 years
- No prior use of purified clotting FVIII products (previous exposure, equal to or less than 5 ED to blood components, e.g. cryoprecipitate, fresh frozen plasma is accepted) including commercially available NovoEight® /Novoeight®
Are the trial subjects under 18? yes
Number of subjects for this age range: 60
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:
- Known or suspected allergy to hamster protein or intolerance to trial product(s) or related products
- Previous participation in this trial defined as withdrawal after administration of trial product
- Congenital or acquired coagulation disorders other than haemophilia A
- Any history of FVIII inhibitor
- Ongoing treatment or planned treatment during the trial with immunomodulatory agents (e.g. intravenous immunoglobulin (IVIG)), routine systemic corticosteroids)



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: 20.0 Level: LLT Classification code 10018937 Term: Haemophilia A System Organ Class: 100000004850

Intervention(s)

Product Name: turoctocog alfa 2000 IU/vial
Product Code: N8
Pharmaceutical Form: Powder and solvent for solution for injection
INN or Proposed INN: turoctocog alfa
CAS Number: 1192451-26-5
Concentration unit: IU/kg international unit(s)/kilogram
Concentration type: equal
Concentration number: 2000-

Product Name: turoctocog alfa 250 IU/vial
Product Code: N8 250
Pharmaceutical Form: Powder and solvent for solution for injection
INN or Proposed INN: turoctocog alfa
CAS Number: 1192451-26-5
Concentration unit: IU/kg international unit(s)/kilogram
Concentration type: equal
Concentration number: 250 -

Primary Outcome(s)

Main Objective: To evaluate safety of turoctocog alfa in paediatric previously untreated patients (PUP) with haemophilia A

Timepoint(s) of evaluation of this end point: will be evaluated for the main phase of the trial

Secondary Objective: - To evaluate efficacy of turoctocog alfa in treatment of bleeds in paediatric PUP with haemophilia A
- To evaluate preventive effect of turoctocog alfa on bleeds in paediatric PUP with haemophilia A

Primary end point(s): Incidence rate of FVIII inhibitors (=0.6 BU)

Secondary Outcome(s)

Secondary end point(s): - Haemostatic effect of turoctocog alfa on treatment of bleeds assessed on a predefined four point scale: Excellent, Good, Moderate and None
- Annualized bleeding rate
- Incidence rate of FVIII inhibitors (=0.6 BU)

Timepoint(s) of evaluation of this end point: Will be evaluated for the main phase of trial, for the extension phase of trial, and for the combined main and extension phases

Secondary ID(s)

NN7008-3809

2011-001033-16-GB

Source(s) of Monetary Support

Novo Nordisk A/S

Ethics review

Status: Approved
Approval date:
Contact:

Results

• Results available: Yes
• Date Posted: 20/06/2019
• Date Completed: 05/12/2018
• URL: https://www.clinicaltrialsregister.eu/ctr-search/trial/2011-001033-16/results