A Multinational, Open-Label, Non-Controlled Trial on Safety, Efficacy and Pharmacokinetics of NNC 0129-0000-1003 in Previously Treated Paediatric Patients with Severe Haemophilia A

• Register: EUCTR
• Last refreshed on: 23 November 2020
• Main ID: EUCTR2012-001711-23-IT
• Date of registration: 13/12/2012
• Prospective Registration: Yes
• Primary sponsor: NOVO NORDISK
• Public title: A Multinational, Open-Label, Non-Controlled Trial on Safety, Efficacy and Pharmacokinetics of NNC 0129-0000-1003 in Previously Treated Paediatric Patients with Severe Haemophilia A
• Scientific title: A Multinational, Open-Label, Non-Controlled Trial on Safety, Efficacy and Pharmacokinetics of NNC 0129-0000-1003 in Previously Treated Paediatric Patients with Severe Haemophilia A
• Date of first enrolment: 18/01/2013
• Target sample size: 60
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2012-001711-23
• 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: Placebo: Other: Number of treatment arms in the trial: 1
• 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, Canada, European Union, Germany, Greece, Israel, Italy, Japan, Korea, Republic of, Lithuania, Macedonia, the former Yugoslav Republic of, Malaysia, Portugal, Switzerland, Turkey, Ukraine, 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 level
< 1%)
- Age below 12 years
- Weight =10 kg
- Documented history of > 150 ED to FVIII products for patients aged 6-
11 years and > 50 ED to FVIII products for patients aged 0-5 years
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:
Anamnesi di sviluppo di inibitori del FVIII


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 10018938 Term: Haemophilia A (Factor VIII) System Organ Class: 100000004850

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

Intervention(s)

Product Name: N8-GP rFVIII
Product Code: NA
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-

Product Name: N8-GP 500U vial
Product Code: NA
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: 500-

Primary Outcome(s)

Main Objective: To evaluate immunogenicity of NNC 0129-0000-1003 (hereafter referred
to as N8-GP)

Primary end point(s): Incidence of inhibitory antibodies against coagulation factor VIII (FVIII)
=0.6 Bethesda units

Timepoint(s) of evaluation of this end point: During the main phase of the trial (from 0-26 weeks of treatment)

Secondary Objective: To evaluate safety other than immunogenicity of N8-GP
- To evaluate efficacy of N8-GP in prophylaxis and treatment of bleeding
episodes
- To evaluate pharmacokinetic properties of N8-GP and compare to
previous FVIII product

Secondary Outcome(s)

Timepoint(s) of evaluation of this end point: The pharmacokinetic endpoints on previous FVIII product will be based
on assessments performed 2-6 weeks prior to initial dosing with N8-GP
and up to 30 hours after administration of previous FVIII product.
The pharmacokinetic endpoints on N8-GP will be based on assessments
performed from 1 hour prior to and up to 96 hours after initial
administration of N8-GP.
All secondary safety and efficacy endpoints will be analysed and
reported separately for the main phase (from 0-26 weeks of treatment)
and the extension phase of the trial (from 26 weeks to the last patient
has completed the trial).

Secondary end point(s): 1. Frequency of adverse events including serious adverse events
reported during the trial period
2. Haemostatic effect of N8-GP when used for treatment of bleeding
episodes and assessed as: Excellent, Good, Moderate, or None
3. Number of bleeding episodes during prophylactic treatment with N8-
GP (annualised bleeding rate)
4. Consumption of N8-GP per bleeding episode (number of injections and
U/kg)
5. Consumption of N8-GP during prophylaxis (number of injections and
U/kg per month and year)
6. Incremental recovery (defined as the peak level recorded 30 min after
end of injection) evaluated for previous FVIII product and N8-GP
7. Area under the curve evaluated for previous FVIII product and N8-GP
8. Terminal half-life evaluated for previous FVIII product and N8-GP
9. Clearance evaluated for previous FVIII product and N8-GP

Secondary ID(s)

NN7088-3885

2012-001711-23-DE

Source(s) of Monetary Support

Novo Nordisk S.p.A.

Ethics review

Status: Approved
Approval date: 10/12/2012
Contact:

Results

• Results available: Yes
• Date Posted: 12/04/2019
• Date Completed: 28/09/2018
• URL: https://www.clinicaltrialsregister.eu/ctr-search/trial/2012-001711-23/results