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

• Register: EUCTR
• Last refreshed on: 10 January 2022
• Main ID: EUCTR2011-001142-15-NO
• Date of registration: 05/01/2012
• Prospective Registration: Yes
• Primary sponsor: Novo Nordisk A/S
• Public title: 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
• Scientific title: A Multi-national Trial Evaluating Safety and Efficacy, including Pharmacokinetics, of NNC 0129-0000-1003 (N8-GP) when Administered for Treatment and Prophylaxis of Bleeding in Patients with Haemophilia A - pathfinder™ 2
• Date of first enrolment: 27/02/2012
• Target sample size: 132
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2011-001142-15
• 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

Australia, Brazil, Bulgaria, Croatia, Denmark, France, Germany, Hungary, Italy, Japan, Korea, Republic of, Malaysia, Netherlands, Norway, Russian Federation, Spain, Sweden, Switzerland, Taiwan, 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:
- 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


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

Health Condition(s) or Problem(s) studied

Haemophilia A
MedDRA version: 14.1 Level: LLT Classification code 10018937 Term: Haemophilia A System Organ Class: 10010331 - Congenital, familial and genetic disorders

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

Intervention(s)

Product Name: N8-GP rFVIII
Pharmaceutical Form: Powder and solvent for solution for injection
CAS Number: 1309086-46-1
Current Sponsor code: NNC129-1003
Concentration unit: U unit(s)
Concentration type: equal
Concentration number: 2000-

Primary Outcome(s)

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

Main Objective: - To evaluate the immunogenicity of NNC 0129-0000-1003 (hereafter referred to as N8-GP) in previously treated patients with Haemophilia A
- 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
- To evaluate the safety of N8-GP when used for prevention of bleeds and treatment of bleeds in patients with haemophilia A
- To evaluate PK properties of N8-GP
- To evaluate Patient Reported Outcomes
- To evaluate the health economic impact of N8-GP treatment
- Generation of a population based PK-model for N8-GP

Secondary Outcome(s)

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 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.

Secondary ID(s)

2011-001142-15-NL

NN7088-3859

Source(s) of Monetary Support

Novo Nordisk A/S

Ethics review

Status: Approved
Approval date: 27/02/2012
Contact:

Results

• Results available: Yes
• Date Posted: 22/06/2019
• Date Completed: 10/12/2018
• URL: https://www.clinicaltrialsregister.eu/ctr-search/trial/2011-001142-15/results