Observatory of ATTR amyloidosis and of patients treated with Vyndaqel® (tafamidis) OBSAMYL study

Study identification

• EU PAS number:
  EUPAS24718
• Study ID:
  42847
• Official title and acronym:
  Observatory of ATTR amyloidosis and of patients treated with Vyndaqel® (tafamidis) OBSAMYL study
• DARWIN EU® study:
  No
• Study countries:
  France
• Study description:
  TTR-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
• Study status:
  Finalised

Research institutions and networks

Institutions

Kappa Santé

France

Multiple centres: 30 centres are involved in the study

Study timelines

Date when funding contract was signed

Planned:01/07/2018

Actual:01/11/2018

Study start date

Planned:16/07/2018

Actual:21/08/2018

Data analysis start date

Planned:14/12/2018

Actual:14/01/2019

Date of interim report, if expected

Planned:10/01/2019

Actual:20/02/2019

Date of final study report

Planned:31/01/2019

Actual:14/10/2020

Sources of funding

Pharmaceutical company and other private sector 

More details on funding

Pfizer

Regulatory

• Was the study required by a regulatory body?:
  Yes
• Is the study required by a Risk Management Plan (RMP)?:
  Not applicable