A Study of the Safety and Efficacy of Prime Editing (PM577) in Participants With Wilson Disease (WD)

Part of paid clinical trials in San Antonio, Texas.

Sponsor
Prime Medicine, Inc.
Study ID
NCT07748403
Phase
PHASE1/PHASE2
Status
Recruiting
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Conditions

  • Wilson Disease
  • Wilson's Disease
  • Wilsons Disease

Eligibility Criteria

Sex
ALL
Age
12 Years - N/A
Healthy Volunteers
Not accepted

Interventions

  • PM577a — DRUG
    PM577a is being evaluated in participants with Wilson disease caused by biallelic pathogenic, likely pathogenic, or suspected pathogenic ATP7B variants, including at least one p.H1069Q allele.

Study Details

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.

Key Dates

First listed
Aug 5, 2026
Start date
Sep 30, 2026
Status verified
Sep 2026
Primary completion
Nov 30, 2028
Completion
Dec 31, 2028

Study Design

Enrollment
42 participants (estimated)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: PM577a
    PM577a is a sterile suspension of lipid nanoparticle (LNP)-formulated Prime Editors (PEs) intended for single dose intravenous (IV) infusion for the treatment of Wilson disease (WD).

Primary Outcome Measure

Safety and tolerability of PM577a. Quantified by frequency and severity of treatment emergent adverse events (TEAEs). [ Time Frame: Post-infusion through Week 48 ]

Locations (1)

FacilityCityStateZIPSite coordinators
ARC Texas Liver InstituteSan AntonioTexas78215
Eric Lawitz, MD
[email protected]
210-253-3426
Tolu Okubote
[email protected]
210-890-5851

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By research site
ARC Texas Liver Institute· San Antonio, TX

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