A Multi-national Trial Evaluating Safety and Efficacy, including Pharmacokinetics, of NNC 0129-0000-1003 when Administered for Treatment and Prophylaxis of Bleeding in Patients with Haemophilia A

Phase 1

• Conditions: Haemophilia A; MedDRA version: 14.1Level: LLTClassification code 10018937Term: Haemophilia ASystem Organ Class: 10010331 - Congenital, familial and genetic disorders; Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

• Registration Number: EUCTR2011-001142-15-NO
• Lead Sponsor: ovo Nordisk A/S

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2012-01-05
• Study Completion: 2018-12-10
• Last Update Posted: 10 January 2022

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: Male
• Target Recruitment: 186

Inclusion Criteria

- Male patients with severe congenital haemophilia A (FVIII activity <1%, according to medical records)
- Documented history of at least 150 EDs to other FVIII products
- Age = 12 years and body weight = 35 kg (except for Croatia and The Netherlands where the lower age limit will be 18 years)
Are the trial subjects under 18? yes
Number of subjects for this age range: 15
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 117
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

- Previous participation in this trial defined as withdrawal after administration N8-GP
- Any history of FVIII inhibitors
- FVIII inhibitors = 0.6 BU/mL at screening
- HIV positive, defined by medical records with CD4+ count =200/µL or a viral load of >400000 copies/mL If the data is not available in medical records within last 6 months, CD4+ will be measured at the screening visit
- Congenital or acquired coagulation disorders other than haemophilia A
- Previous significant thromboembolic events (e.g. myocardial infarction, cerebrovascular disease or deep venous thrombosis) as defined by available medical records
- Platelet count < 50,000 platelets/µL (laboratory value at the screening visit)
- ALAT > 3 times the upper limit of normal reference ranges at central laboratory
- Creatinine level = 1.5 times above upper normal limit (according to central laboratory reference ranges)
- Ongoing immune modulating or chemotherapeutic medication

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: - To evaluate the immunogenicity of NNC 0129-0000-1003 (hereafter referred to as N8-GP) in previously treated patients with Haemophilia A<br>- To evaluate the clinical efficacy of N8-GP in bleeding prophylaxis (number of bleeds during prophylaxis);Secondary Objective: - To evaluate the clinical efficacy of N8-GP when treating bleeds in patients with haemophilia A<br>- To evaluate the safety of N8-GP when used for prevention of bleeds and treatment of bleeds in patients with haemophilia A<br>- To evaluate PK properties of N8-GP<br>- To evaluate Patient Reported Outcomes<br>- To evaluate the health economic impact of N8-GP treatment<br>- Generation of a population based PK-model for N8-GP;Primary end point(s): - The Incidence rate of FVIII-inhibitors =0.6 BU<br>- Annualised bleeding rate in the prophylaxis arm;Timepoint(s) of evaluation of this end point: The endpoints will be analysed based on all available information until the end of trial (EOT) visit and up to approximately 19 months.

Secondary Outcome Measures

Name	Time	Method
Secondary end point(s): - Haemostatic effect of N8-GP when used for treatment of bleeds, assessed on a four-point scale for haemostatic response (excellent, good, moderate and none) by counting excellent and good as success and moderate and none as failure.;Timepoint(s) of evaluation of this end point: The endpoints will be analysed based on all available information until the end of trial (EOT) visit and up to approximately 19 months.