A Study of the Safety and Efficacy of Prime Editing (PM577) in Participants With Wilson Disease (WD)
Trial statusNot yet recruiting
Trial phasePhase 1, Phase 2
Trial typeInterventional
Biological sexAll
Age12+
SponsorPrime Medicine, Inc.
About this trial
The purpose of this study is to evaluate the safety, tolerability, biological activity, and initial efficacy of PM577a, an investigational Prime Editing therapy, in adults and adolescents with Wilson disease (WD).
Wilson disease is caused by changes (mutations) in the ATP7B gene that prevent the body from removing excess copper normally. PM577a is designed to precisely correct one of the most common disease-causing ATP7B mutations (p.H1069Q) in liver cells with the goal of restoring normal copper metabolism.
This is the first study of PM577a in people. Participants will receive a single intravenous (IV) infusion of PM577a and will be monitored closely to evaluate safety, how the body responds to treatment, whether copper metabolism improves, and whether treatment may improve signs and symptoms of Wilson disease.
Eligibility criteria
Qualifiers
Confirmed Wilson Disease (WD) diagnosis as determined by medical history consistent with WD
Historical genetic analysis demonstrating biallelic pathogenic, likely pathogenic, or suspected pathogenic ATP7B variants, including at least one p.H1069Q allele.
Treated and stable on standard of care therapy for WD for the past 6 months prior to signing ICF, as documented by a history of adherence to SOC medications (i.e., penicillamine, trientine, and/or zinc) without significant medication or dose/frequency changes, per Investigator judgement.
Demonstrated Adequate Copper Control confirmed at screening
Disqualifiers
Known prior medically significant reactions (e.g., severe hypersensitivity, myocarditis) to an LNP-based or PEG-containing product (e.g., mRNA-based COVID vaccinations, MiraLAX) or any medication required as part of the clinical study protocol
Receipt of any prior gene therapy for WD, including AAV-based therapy
Receipt of any liver-directed LNP gene therapy (including siRNA or ASO therapies) or gene editing treatment.
Unstable neurological conditions within the prior 12 months which may impact participant safety or participation in the study, including ability to complete study requirements or procedures as outlined in the clinical study protocol in the opinion of the Investigator
Trial design
Treatments tested in this trial
- PM577a
Treatment groups
42 Participants
are divided into 1 treatment groupSponsors and collaborators
Source ClinicalTrials.gov