Clinical Trial

Personalized Antisense Oligonucleotide for A Single Participant With UBTF Gene Mutation

Name: Personalized Antisense Oligonucleotide for A Single Participant With UBTF Gene Mutation — clinical trial record (NCT07588581)
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 Childhood-Onset Neurodegeneration with Brain Atrophy (CONDBA) due to a heterozygous missense gain-of-function mutation in UBTF

Trial Details

NCT Number	NCT07588581
Lead Sponsor	n-Lorem Foundation Collaborators: Massachusetts General Hospital
Conditions	Childhood-Onset Neurodegeneration With Brain Atrophy (CONDBA)
Enrollment	1 participants
Start Date	2025-02-05
Primary Completion	2027-02 (estimated)
Study Completion	2027-02 (estimated)
Updated on ClinicalTrials.gov	2026-05-15