Alnylam Pregnancy Surveillance Program
Research type
Research Study
Full title
Patisiran-LNP Pregnancy Surveillance Program
IRAS ID
275656
Contact name
Julian Gillmore
Contact email
Sponsor organisation
Alnylam Pharmaceuticals Inc
Duration of Study in the UK
10 years, 0 months, 0 days
Research summary
This is a non-interventional, multi country, multi-center study being conducted in up to 8 countries. The patients for the study will be pregnant women who possess the Hereditary transthyretin-mediated amyloidosis condition and have been exposed to Patisiran-LNP at least 12 weeks before their last menstrual period.
The study is observational and will collect primary data from pregnant women in the form of questionnaires that will be administered to both patients and their healthcare providers throughout a total of expected 8 visits. Follow up visits will also be conducted with the infant's healthcare provider throughout 1 year after birth.
The goal is to develop a better understanding of the safety of Patisiran LNP. The drug is known to result in a reduction of Vitamin A serum levels in fetuses however the impact of such a reduction is unknown.
Patients will be selected based on adherence to the inclusion and exclusion criteria and will be expected to participate for a total of 21 months with the entire duration of the program being approximately 10 years. A coordinating center will be responsible for both patient enrollment and as well as data entry into the electronic case report form (eCRF).
A pregnancy awareness program will be set up in order so that patients not participating in Alnylam studies are aware of the pregnancy registry.REC name
London - Stanmore Research Ethics Committee
REC reference
20/PR/0417
Date of REC Opinion
24 Nov 2020
REC opinion
Further Information Favourable Opinion