Browse Studies — Arctuva

Phase 1

A Phase 1/2 Clinical Study to Evaluate Safety, Tolerability, Biological Activity, and Initial Efficacy of Prime Editing (PM577a) for the Treatment of Wilson Disease (WD) in Adult and Adolescent Participants With at Least One Allele Harboring the p.H1069Q Mutation in ATP7B

The purpose of this study is to evaluate the safety, tolerability, biological activity, and initial efficacy of PM577a, an investigational Prime Editing therap…

Wilson DiseaseWilson's DiseaseWilsons Disease

Estimated

~$10 – $8,750

Prime Medicine, Inc.NCT07748403

Natural History of Wilson Disease: Registry for Patients With Wilson Disease

The purpose of the registry/repository is to provide a mechanism to store data and specimens to support the conduct of future research about Wilson disease (WD…

Wilson Disease

Estimated

~$10 – $750

Yale UniversityNCT03334292

Monitoring of Therapy in Wilson Disease With Off-Treatment Urinary Copper Excretion (OT-UCE): Comparison With Serum Non-Ceruloplasmin Copper (NCC) Assays

This is a prospective study that will determine the optimal timing for 24-hour urinary copper excretion (UCE) measurement after temporary discontinuation of st…

Wilson Disease

Estimated

~$10 – $750

Yale UniversityNCT07301216