Clinical Trial

Personalized Antisense Oligonucleotide Therapy for A Single Participant With ASXL3 Gene Mutation

Name: Personalized Antisense Oligonucleotide Therapy for A Single Participant With ASXL3 Gene Mutation — clinical trial record (NCT07197268)
Creator: DataLookout
License: https://creativecommons.org/licenses/by/4.0/

Active, Not Recruiting Phase 1/2

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Summary

This research project entails delivery of a personalized antisense oligonucleotide (ASO) drug designed for a single participant with Bainbridge-Ropers Syndrome (BRPS) due to a pathogenic, de novo nonsense variant in ASXL3

Trial Details

NCT Number	NCT07197268
Lead Sponsor	n-Lorem Foundation Collaborators: University of North Carolina, Chapel Hill
Conditions	Bainbridge-Ropers Syndrome
Enrollment	1 participants
Start Date	2025-05-19
Primary Completion	2027-05 (estimated)
Study Completion	2027-05 (estimated)
Updated on ClinicalTrials.gov	2025-09-29