MedTrace Logo

Personalized Antisense Oligonucleotide for A Single Participant With UBTF Gene Mutation

NCT07588581 · Status: ACTIVE_NOT_RECRUITING · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 1

Last updated 2026-05-15

No results posted yet for this study

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

Conditions

  • Childhood-Onset Neurodegeneration With Brain Atrophy (CONDBA)

Interventions

DRUG

nL-UBTF-001

Personalized antisense oligonucleotide

Sponsors & Collaborators

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2025-02-05
Primary Completion
2027-02-28
Completion
2027-02-28
FDA Drug
Yes

Countries

  • United States

Study Locations

More Related Trials

Entities

Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT07588581 on ClinicalTrials.gov