Clinical Trials

Overview

About this study

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 natural history and real-world clinical management of patients diagnosed with PH1, as well as the long-term effectiveness of lumasiran. Patients will be managed and treated per routine clinical practice. This protocol does not recommend the use of any specific treatments, visits, or procedures. No medication is provided as part of study participation.

Patients are expected to contribute data for the duration of the study or until study discontinuation (e.g., due to death, withdrawal of consent, loss to follow-up, study termination, enrollment in a clinical trial with an investigational treatment during the study). Study data will be collected at the time of a routine clinical encounter, or by referencing the medical record, and entered into the electronic data capture (EDC) at least once every 12 months using electronic case report forms (eCRFs). If patients attend more than 1 visit per year, sites can enter multiple follow-up visits. In addition to the prospective data collection, a chart review of up to 5 years 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.

Participation eligibility

Participant eligibility includes age, gender, type and stage of disease, and previous treatments or health concerns. Guidelines differ from study to study, and identify who can or cannot participate. There is no guarantee that every individual who qualifies and wants to participate in a trial will be enrolled. Contact the study team to discuss study eligibility and potential participation.

Participating Mayo Clinic locations

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