Hereditary Amyloidosis clinical trials at UCLA
2 research studies open to eligible people
ConTTRibute: A Global Observational Study of Patients With Transthyretin (TTR)-Mediated Amyloidosis (ATTR Amyloidosis)
open to all eligible people
The purpose of this study is to: - Describe epidemiological and clinical characteristics, natural history and real-world clinical management of ATTR amyloidosis patients - Characterize the safety and effectiveness of patisiran and vutrisiran as part of routine clinical practice in the real-world clinical setting - Describe disease emergence/progression in pre-symptomatic carriers of a known disease-causing transthyretin (TTR) variant
Los Angeles 5368361, California 5332921 and other locations
Patients With Transthyretin (ATTR) Amyloidosis
open to eligible people ages 18-130
The MaesTTRo study aims to enroll a global cohort of patients with transthyretin (ATTR) amyloidosis to longitudinally observe the natural course of the disease and describe real-world treatment patterns and outcomes. In addition, information on the effectiveness of ATTR amyloidosis treatments, including eplontersen, which is a ligand-conjugated antisense oligonucleotide gene silencing treatment targeting activity against both the mutant and wild-type TTR protein, will be collected.
Los Angeles 5368361, California 5332921 and other locations
Our lead scientists for Hereditary Amyloidosis research studies include Perry B. Shieh, MD, PhD.
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