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Personalized Antisense Oligonucleotide for A Single Participant With UBTF Gene Mutation

StatusActive, Not Recruiting
PhasePhase 1/2
Started
View on ClinicalTrials.gov ↗

Record History

  1. Study First Submitted
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
(from sponsor)		 Childhood-Onset Neurodegeneration With Brain Atrophy (CONDBA)
Enrollment 1 participants
Start Date
Primary Completion (estimated)
Study Completion (estimated)
Updated on ClinicalTrials.gov