Safety and efficacy of NNC 0155-0000-0004 in prevention and treatment of bleeds in previously untreated children with Haemophilia A

Safety and Efficacy of NNC 0155-0000-0004 in Prevention and Treatment of Bleeds in Paediatric Previously Untreated Patients with Haemophilia A - guardian™4

Status

Active, not recruiting

Phases

Phase 3

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2011-001033-16-GB

Enrollment

120

Registered

2011-08-17

Start date

Unknown

Completion date

Unknown

Last updated

2017-10-02

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Haemophilia A MedDRA version: 14.0 Level: LLT Classification code 10018937 Term: Haemophilia A System Organ Class: 10010331 - Congenital, familial and genetic disorders

Interventions

Product Code: N8 Pharmaceutical Form: Powder and solvent for solution for injection INN or Proposed INN: Proposed INN: turoctocog alfa CAS Number: 1192451-26-5 Current Sponsor code: NNC 1055-0000-0004

Eligibility

Sex/Gender

Male

Inclusion 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 with congenital severe haemophilia A (baseline level FVIII =65 years) no F.1.3.1 Number of subjects for this age range

Exclusion criteria

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 - FVIII inhibitor (>= 0.6 BU/mL) at screening - Ongoing treatment or planned treatment during the trial with immunomodulatory agents (e.g. intravenous immunoglobulin (IVIG)), routine systemic corticosteroids) - Platelet count <50,000 platelets/µL

Design outcomes

Primary

Measure	Time frame
Main Objective: To evaluate safety of NNC 0155-0000-0004 in paediatric previously untreated patients (PUP) 1 with haemophilia A;Secondary Objective: - To evaluate efficacy of NNC 0155-0000-0004 in treatment of bleeds in paediatric PUP with haemophilia A - To evaluate preventive effect of NNC 0155-0000-0004 on bleeds in paediatric PUP with haemophilia A;Primary end point(s): Incidence rate of FVIII inhibitors (>0.6 BU/mL) will be evaluated from Visit 1 to End of Trial Visit/Visit 7;Timepoint(s) of evaluation of this end point: from Visit 1 to End of Trial Visit/Visit 7	—

Measure

Time frame

Main Objective: To evaluate safety of NNC 0155-0000-0004 in paediatric previously untreated patients (PUP) 1 with haemophilia A;Secondary Objective: - To evaluate efficacy of NNC 0155-0000-0004 in treatment of bleeds in paediatric PUP with haemophilia A - To evaluate preventive effect of NNC 0155-0000-0004 on bleeds in paediatric PUP with haemophilia A;Primary end point(s): Incidence rate of FVIII inhibitors (>0.6 BU/mL) will be evaluated from Visit 1 to End of Trial Visit/Visit 7;Timepoint(s) of evaluation of this end point: from Visit 1 to End of Trial Visit/Visit 7

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Secondary

Measure	Time frame
Secondary end point(s): - Haemostatic effect of NNC 0155-0000-0004 on treatment of bleeds assessed on a predefined four point scale: Excellent, Good, Moderate and None will be evaluated from Visit 2 to End of Trial Visit/Visit 7 - Annualized bleeding rate will be evaluated from Visit 2 to End of Trial Visit/Visit 7;Timepoint(s) of evaluation of this end point: from Visit 2 to End of Trial Visit/Visit 7	—

Measure

Time frame

Secondary end point(s): - Haemostatic effect of NNC 0155-0000-0004 on treatment of bleeds assessed on a predefined four point scale: Excellent, Good, Moderate and None will be evaluated from Visit 2 to End of Trial Visit/Visit 7 - Annualized bleeding rate will be evaluated from Visit 2 to End of Trial Visit/Visit 7;Timepoint(s) of evaluation of this end point: from Visit 2 to End of Trial Visit/Visit 7

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Countries

Austria, Brazil, China, Croatia, Czech Republic, Denmark, Greece, Hong Kong, Hungary, Japan, Korea, Republic of, Lithuania, Malaysia, Poland, Portugal, Romania, Russian Federation, Serbia, Spain, Thailand, Turkey, United Kingdom, United States

Contacts

Public ContactGlobal Clinical Registry (GCR,1452)

Novo Nordisk A/S

clinicaltrials@novonordisk.com

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026