A Multi-Centre, Open-Label, Single-Arm, and Multiple Dosing Trial on Safety of Monthly Replacement Therapy with Recombinant Factor XIII (rFXIII) in Subjects with Congenital Factor XIII Deficiency - Mentor™2

• Register: EUCTR
• Last refreshed on: 7 December 2015
• Main ID: EUCTR2008-007883-41-FI
• Date of registration: 15/06/2009
• Prospective Registration: Yes
• Primary sponsor: Novo Nordisk A/S
• Public title: A Multi-Centre, Open-Label, Single-Arm, and Multiple Dosing Trial on Safety of Monthly Replacement Therapy with Recombinant Factor XIII (rFXIII) in Subjects with Congenital Factor XIII Deficiency - Mentor™2
• Scientific title: A Multi-Centre, Open-Label, Single-Arm, and Multiple Dosing Trial on Safety of Monthly Replacement Therapy with Recombinant Factor XIII (rFXIII) in Subjects with Congenital Factor XIII Deficiency - Mentor™2
• Date of first enrolment: 13/08/2009
• Target sample size: 60
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2008-007883-41
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: yes Randomised: no Open: yes 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: yes Other specify the comparator: Historical

Countries of recruitment

Austria, Finland, France, Germany, Italy, Spain, United Kingdom

Key inclusion & exclusion criteria

Inclusion criteria:
For subjects who participated in F13CD-1725:
1 Informed consent obtained before any trial-related activities. (Trialrelated activities are any procedure that would not have been performed during normal management of the subject).
2. Previous participation (means up to and inclusive Visit 16, (EOT)) in F13CD-1725.
3. If female and of childbearing potential: negative pregnancy test at screening.
For all other subjects:
1. Informed consent obtained before any trial-related activities (Trialrelated activities are any procedure that would not have been performed during normal management of the subject).
2. Diagnosis of congenital FXIII A-subunit deficiency (confirmed by genotyping at screening visit or documented results from previously performed genotyping).
3. Subjects with age = 6 years and a weight =20 kg.
4. If female and of child-bearing potential: negative pregnancy test at screening.
Are the trial subjects under 18? yes
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range

Exclusion criteria:
1. Known neutralizing antibodies (inhibitors) towards FXIII.
2. Any known congenital or acquired coagulation disorder other than congenital FXIII deficiency.
3. Platelet count (thrombocytes) < 50 × 10^9/L. For subjects who participated in F13CD-1725 platelet count from visit 15 in F13CD-1725 must be used for evaluation.
4. Known or suspected allergy to trial product(s) or related products.
5. Treatment with any investigational drug within 30 days of trial enrolment, except pdFXIII and rFXIII
6. Renal insufficiency defined as currently requiring dialysis therapy.
7. Any history of confirmed venous or arterial thrombo-embolic events, including myocardial infarction or stroke
8. Medical, social or psychosocial factors expected to impact compliance or safety.
9. Any disease or condition which, judged by the Investigator, could imply a potential hazard to the subject, interfere with the trial participation or trial outcome.
10. Mental incapacity, unwillingness or language barriers precluding adequate understanding or cooperation in participating in the trial.
11. Females of childbearing potential who are pregnant, breastfeeding or are not using adequate contraceptive methods (adequate contraceptive measures as required by local law or practice) from the time of
enrolment to completion of all follow-up trial visits, if there is any risk of pregnancy in the opinion of the Investigator.


Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

Congenital Factor XIII Deficiency
MedDRA version: 14.1 Level: PT Classification code 10061992 Term: Haemophilia System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 14.1 Level: LLT Classification code 10010432 Term: Congenital deficiency of other clotting factors System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 14.1 Level: HLT Classification code 10009735 Term: Coagulation disorders congenital System Organ Class: 10010331 - Congenital, familial and genetic disorders

Intervention(s)

Product Name: Recombinant factor XIII (rFXIII)
Pharmaceutical Form: Powder and solvent for solution for injection
INN or Proposed INN: Catridecacog
CAS Number: 606138-08-3
Current Sponsor code: NN1841
Other descriptive name: Recombinant factor XIII (rFXIII)
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 5-

Primary Outcome(s)

Primary end point(s): Adverse events (serious and non-serious) occurring from first trial related activity after signing the informed consent to the end of subject’s participation in the trial

Secondary Objective: To evaluate the efficacy of monthly replacement therapy with rFXIII when used for prevention of bleeding episodes and for treatment of breakthrough bleedings in subjects with congenital FXIII deficiency.

Main Objective: To assess long term safety of monthly replacement therapy with recombinant factor XIII (rFXIII) when used for prevention of bleeding episodes and for treatment of breakthrough bleedings in subjects with congenital factor XIII (FXIII) deficiency.

Secondary ID(s)

F13CD-3720(Mentor™2)