Background and Importance As a reference centre for the treatment of familial paramyloidosis, our hospital receives patients from all over the country.¹ The emergence of new therapeutic options is essential to ensure treatment and reduce the impact that the disease has on individuals and families.

Clinical Trials (CT) using new molecules such as tafamidis, inotersen and patisiran represent significant advances in the treatment of patients with Hereditary Transthyretin Amyloidosis (hATTR), instead of liver transplant.²

Aim and Objectives Describing the activity of the Pharmacy Department Clinical Trials Unit (PDCTU) in a reference centre for the investigation and treatment of hATTR, between 2006 and 2023.

Material and Methods Retrospective analysis of the participation of the PDCTU of our hospital in the clinical investigation of hATTR. For this analysis, the number of CT started each year, the number of ongoing CT and the number of patients included in CT associated with hATTR were evaluated.

Results Since 2006, our PDCTU has participated in 21 CT. It has made a significant contribution to the approval of emerging therapies, some of which have already been granted Marketing Authorisation, as is the case of transthyretin (TTR) stabilisers and TTR level reducing agents.

In total, since 2006, 327 patients have taken part in hATTR-related CT, 64 of whom are still taking part in a set of 6 CT, all of them of phase 3.

Each trial associated with hATTR had an average participation of eight patients, an average well above the average of patients/trial (two patients/trial) at our centre.

Conclusion and Relevance Since 2015 there has been a growing trend in the inclusion of hospital in new CT. The centre is evaluating various investigational therapies for the treatment of hATTR, including agents that stabilise TTR, antibodies, antisense oligonucleotides and RNAi therapies.

The pharmacists at the PDCTU, contribute to the development and approval of new therapeutics, guidelines and protocols. Since they are responsible for the entire investigational product circuit, they ensure that trials are well conducted.

References and/or Acknowledgements 1. SNS – Portal do SNS. Paramiloidose em Portugal e no mundo. https://www.sns.gov.pt/noticias/2021/04/29/paramiloidose-em-portugal-e-no-mundo/

2. Dispenzieri A. Clinical and genetic profile of patients enrolled in the transthyretin amyloidosis outcomes survey (THAOS): 14-year update. Orphanet Journal of Rare Diseases. 2022; 17. https://doi.org/10.1186/s13023-022-02359-w

Conflict of Interest No conflict of interest.