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Filters: Condition: Childhood-Onset Neurodegeneration With Brain Atrophy (CONDBA) Clear all
Active, not recruiting Phase 1Phase 2 Interventional

Personalized Antisense Oligonucleotide for A Single Participant With UBTF Gene Mutation

NCT07588581
View directory record Official record
Conditions
Childhood-Onset Neurodegeneration With Brain Atrophy (CONDBA)
Interventions
nL-UBTF-001
Drug
Lead sponsor
n-Lorem Foundation
Other
Eligibility
Not listed
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated May 14, 2026 · Synced Jun 10, 2026, 4:05 AM EDT