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Clinical trial

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

NCT07748403 is registered in ClinicalTrials.gov with Prime Medicine, Inc. as lead sponsor.

NCT NCT07748403
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Registry record

As filed in ClinicalTrials.gov.

Title
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
Lead sponsor
Prime Medicine, Inc.
Phase
Phase 1/2
Overall status
Recruiting
Start date
September 30, 2026
Completion date
December 31, 2028
Enrollment
42 (estimated)
Sites
4

Conditions

As the registry lists them; a condition with its own page is linked.

wilson disease, wilson's disease, wilsons disease