Bonaph1de
Research type
Research Study
Full title
BONAPH1DE, A prospective observational study of patients \nwith primary hyperoxaluria type 1 (PH1)
IRAS ID
303235
Contact name
Faidra Veligratli
Contact email
Sponsor organisation
Alnylam Pharmaceuticals Inc
Clinicaltrials.gov Identifier
N/A, N/A
Duration of Study in the UK
5 years, 2 months, 1 days
Research summary
This is a global, prospective, observational, longitudinal study designed to characterize the long-term safety of lumasiran in PH1 patients in the real-world setting. The data collected in this study will also be utilized to further characterize the long-term effectiveness of lumasiran as well as the natural history and real-world clinical management of patients diagnosed with PH1.\nThe study is designed for a prospective study duration of at least 7 years and will enroll PH1 patients regardless of their treatment status. The study aims to enroll all eligible PH1 patients but is targeting to include approximately 100 patients treated with lumasiran. The overall number of PH1 patients is not pre-specified and the number of patients and sites may be adjusted during the study period to meet the enrollment goal to achieve a number of 100 patients treated with lumasiran. The end of study enrollment and the overall study duration will occur upon agreement with regulatory authorities. Patients will be enrolled from multiple sites in Europe, North America, and the Middle East (other regions may be considered).\nIn addition to the prospective data collection, an up to 5-year chart review relative to the time of enrollment in the study will be conducted to collect retrospective data, where available. In cases where diagnosis was more than 5 years prior to enrollment, data will also be collected at the time of diagnosis, if available.
REC name
North East - Tyne & Wear South Research Ethics Committee
REC reference
21/NE/0179
Date of REC Opinion
22 Oct 2021
REC opinion
Further Information Favourable Opinion