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Personalized Antisense Oligonucleotide Therapy for A Single Participant With ATN1 Gene Mutation

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

Record History

  1. Primary and study completion dates: Feb 2026 to Feb 2027v2
  2. Study First Submitted
  • Stopped: an ongoing trial was terminated, suspended or withdrawn.
  • Notable: the trial’s plan changed such as a big move in its timeline or size, a new primary endpoint, sponsor or phase, or recruiting opened or resumed.
  • Minor: routine progress and housekeeping, such as sites added or enrollment closed.
This research project entails delivery of a personalized antisense oligonucleotide (ASO) drug designed for a single participant with dentatorubral-pallidoluysian atrophy (DRPLA) due to a heterozygous pathogenic CAG trinucleotide expansion in ATN1
Trial Details
NCT Number NCT06706388
Lead Sponsor n-Lorem Foundation
Collaborators: Columbia University
Conditions
(from sponsor)		 Dentatorubral-Pallidoluysian Atrophy
Related terms Myoclonic Epilepsies, Progressive
Enrollment 1 participants
Start Date
Primary Completion (estimated)
Study Completion (estimated)
Updated on ClinicalTrials.gov