Safety, tolerability, and pharmacokinetics study of turoctocog alfa pegol injected under the skin in patients with haemophilia A

• Register: EUCTR
• Last refreshed on: 12 November 2018
• Main ID: EUCTR2016-002396-99-AT
• Date of registration: 09/09/2016
• Prospective Registration: Yes
• Primary sponsor: Novo Nordisk A/S
• Public title: Safety, tolerability, and pharmacokinetics study of turoctocog alfa pegol injected under the skin in patients with haemophilia A
• Scientific title: Safety, tolerability, and pharmacokinetics study of single and multiple subcutaneous doses of turoctocog alfa pegol in patients with haemophilia A - alleviate 1
• Date of first enrolment: 20/10/2016
• Target sample size: 48
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2016-002396-99
• 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:
• Phase: Human pharmacology (Phase I): yes Therapeutic exploratory (Phase II): no Therapeutic confirmatory - (Phase III): no Therapeutic use (Phase IV): no

Countries of recruitment

Austria, Bulgaria, European Union, France, Germany, Japan, Serbia, Turkey, United States

Contacts

• Name: Clinical Disclosure (1452)
• Affiliation: Novo Nordisk A/S

• Name: Clinical Disclosure (1452)
• Affiliation: Novo Nordisk A/S

Key inclusion & exclusion criteria

Inclusion criteria:
1. Male, age above or equal to 18 years at the time of signing informed consent,(part A).
2. Male, age above or equal to 12 years at the time of signing informed consent,(part B).
3. Diagnosis of congenital haemophilia A based on medical records (FVIII activity <1%).
4. History of more than 150 exposure days to any FVIII containing products.

Are the trial subjects under 18? yes
Number of subjects for this age range: 3
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 45
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion criteria:
1. Previous participation in this trial. Participation is defined as signed informed consent. (Patients who have completed part A are allowed to also participate in part B. If so, a separate informed consent covering part B must be signed.)
2. Immune compromised patients due to human immunodeficiency virus (HIV) infection (defined as viral load =400.000 copies/mL and/or cluster of differentiation 4+ (CD4+) lymphocyte count =200/µL performed at screening or defined by medical records no older than 6 months)
3. Any history of FVIII inhibitors (defined by medical records within 8 years of randomisation)
4. Inhibitors to FVIII (= 0.6 Bethesda unit (BU)) at screening, measured by Nijmegen modified Bethesda method at central laboratory.



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

Health Condition(s) or Problem(s) studied

Haemophilia A
MedDRA version: 20.0 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: SC turoctocog alfa pegol A 2000 IU/vial
Pharmaceutical Form: Powder for solution for injection
INN or Proposed INN: turoctocog alfa pegol
CAS Number: *MASKED*
Other descriptive name: TUROCTOCOG ALFA PEGOL
Concentration unit: IU international unit(s)
Concentration type: equal
Concentration number: 2000-
Pharmaceutical form of the placebo: Solution for injection
Route of administration of the placebo: Subcutaneous use

Primary Outcome(s)

Main Objective: To evaluate the safety of s.c. administration of turoctocog alfa pegol (SC N8-GP) in patients with severe haemophilia A

Timepoint(s) of evaluation of this end point: Until 28 days after last exposure

Secondary Objective: To evaluate the pharmakokinetics of SC N8-GP in patients with severe haemophilia A

Primary end point(s): Number of adverse events reported after exposure to SC N8-GP

Secondary Outcome(s)

Secondary end point(s): Pharmacokinetic parameter Cmax (up to 144 hours after dose)

Timepoint(s) of evaluation of this end point: After single dose administration (part A)

Secondary ID(s)

NN7170-4213

Source(s) of Monetary Support

Novo Nordisk A/S