3868: Non-Interventional Study in Congenital FXIII Deficiency
Research type
Research Study
Full title
Use of rFXIII in treatment of congenital FXIII deficiency, a prospective multi-centre non-interventional study
IRAS ID
158603
Contact name
Anja Rahbeck Trolle
Contact email
Sponsor organisation
Novo Nordisk A/S
Clinicaltrials.gov Identifier
UTN Number, U1111-1131-1558; EU PAS Number, ENCEPP/SDPP/3687
Duration of Study in the UK
3 years, 4 months, 7 days
Research summary
This post-marketing observational study of recombinant factor XIII (rFXIII) is designed to observe the use of rFXIII in normal clinical practice. The overall objective is to investigate the incidence of specific adverse drug reactions associated with the use of rFXIII in patients with congenital FXIII subunit A-deficiency. Hence, the study will expand our understanding of the safety profile of rFXIII.
This study was requested by the authorities as part of the condition of marketing authorisation to enable further safety data to be collected in real-life clinical practice.
The study will be conducted as a prospective, single-arm, multinational observational Post Authorisation Safety Study (PASS) in patients with congenital FXIII deficiency who are treated with rFXIII. The study will be accessible by all EU countries and other selected non-EU countries, where rFXIII is commercially available.
The study duration is estimated to be 5 years. The number of recruited patients will be limited by the small target population, and additionally the market penetration of rFXIII for the treatment of FXIII deficiency will determine the number of patients eligible for inclusion in the study.All patients enrolled in this study will receive their medication through usual commercial channels and according to normal clinical practice per discretion of their medical doctor. The study product will not be provided free of charge by Novo Nordisk as this is a non-interventional study and participants will already be on the drug.
The study will include patients with congenital FXIII subunit A-deficiency for whom the decision to treat with rFXIII has already been made and who are willing to provide informed consent (or consent of the patient’s legally acceptable representative, if applicable). Due to the multicentre, multinational population selected, the generalisability of the study is evaluated as high.
REC name
North of Scotland Research Ethics Committee 2
REC reference
15/NS/0013
Date of REC Opinion
13 Mar 2015
REC opinion
Favourable Opinion