Status: Ongoing First registered on: 03/07/2018
Last updated on: 16/12/2019
1. Study identification
EU PAS Register NumberEUPAS24718
Official titleObservatory of TTR-related amyloidosis and of patients treated with Vyndaqel(r)
Study title acronymOBSAMYL
Study typeObservational study
Brief description of the studyTTR-related amyloidosis is a systemic trouble in which extracellular transthyretin deposits aggregate in organs and tissues. In hereditary TTR-related amyloidosis, more than 100 gene mutations on transthyretin gene have been identified to be responsible for symptoms such as nerve and neurological peripheral damages. Two main phenotypic forms are encountered: transthyretin familial amyloid polyneuropathy (TTR-FAP) which is an autosomal dominant hereditary disease and transthyretin familial amyloid cardiomyopathy (TTR-FAC), occurring in 80% of patients with TTR-FAP. Prevalence of these two conditions are not well-known: TTR-FAP was estimated at 1/1.106 in general population whereas the French national registry has recorded 482 patients from 1989 to 2014. For TTR-FAC, there is a lack of epidemiological data. TTR-related amyloidosis also includes senile amyloidosis occurring predominantly in men aged over 60 years, in which amyloid deposits are derived from wild transthyretin. Prevalence of WT-TTR in the general population is not known either. Vyndaqel® (tafamidis) is a TTR tetramer stabilizer authorized in the EU market since 2012, for the treatment of TTR-FAP in stage 1 to delay neurologic peripheral impairment and is actually under clinical study for TTR-cardiomyopathy (senile amyloidosis + TTR-FAC). In the Transparency Committee's opinion of 2012, the medical value of Vyndaqel® was considered moderate and added medical value was considered minor. In this report, 200 patients were estimated eligible to receive Vyndaqel®. As part of the five-yearly renewal of tafamidis on the list of reimbursable products, its target population and modalities of use need to be assessed in 'real life’. Thus, Pfizer aims to conduct a real-life study to provide new data regarding TTR-amyloidosis and tafamidis use. This epidemiological study will rely on the French amyloidosis network. fr
Was this study requested by a regulator?Yes: France
Is the study required by a Risk Management Plan (RMP)? Not applicable
Regulatory procedure number (RMP Category 1 and 2 studies only)
Other study registration identification numbers and URLs as applicable
2. Research centres and Investigator details
Coordinating study entity
Centre to which the investigator belongsKappa Santé
Department/Research groupKappa Santé
Organisation/affiliationKappa Santé
Website/Homepagewww.kappasante.com
Details of (Primary) lead investigator
Title Dr
Last name DEMARCQ
First name Olivier
Is this study being carried out with the collaboration of a research network?
No
Other centres where this study is being conducted
Multiple centres

In total how many centres are involved in this Study?30
Countries in which this study is being conducted
National study

France
3. Study timelines: initial administrative steps, progress reports and final report
PlannedActual
Date when funding contract was signed01/07/201801/11/2018
Start date of data collection16/07/201821/08/2018
Start date of data analysis14/12/201814/01/2019
Date of interim report, if expected10/01/201920/02/2019
Date of final study report31/01/2019
4. Sources of funding
Please provide estimates of the percentage of funding by source for this study
Names(s)Approximate % funding
Pharmaceutical companiesPfizer100
Charities
Government body
Research councils
EU funding scheme
5. Contact details for enquiries
Scientific Enquiries
Title Dr
Last name DEMARCQ
First name Olivier
Address line 123-25 Avenue du Dr Lannelongue
Address line 2 
Address line 3 
CityParis 
Postcode75014 
CountryFrance
Phone number (incl. country code)0033144827474 
Alternative phone number 
Fax number (incl. country code) 
Email address olivier.demarcq@pfizer.com
Public Enquiries
Title Mrs 
Last name SABIN 
First name Bénédicte 
Address line 14 rue de Cléry 
Address line 2 
Address line 3 
City75002 
Postcode 
CountryFrance 
Phone number (incl. country code)0614431702 
Alternative phone number 
Fax number (incl. country code) 
Email address benedicte.sabin@kappasante.com 
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