Personalized Antisense Oligonucleotide for A Single Participant With ATN1 Gene Mutation

Running, not enrolling · Phase 1/Phase 2

Conditions studied: Dentatorubral-Pallidoluysian Atrophy

In brief

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

Key facts

Study ID

NCT07084311

Run by

n-Lorem Foundation

People needed

1

Starts

2024-10-24

Expected to finish

2026-11-01

Last updated by the study team

2026-07-13

Who can join

Age: 29 and older, up to 29. Sex: female. Healthy volunteers: not accepted.

You may qualify if…

• Informed consent/assent provided by the participant (when appropriate), and/or participant's parent(s) or legally authorized representative(s).
• Ability to travel to the study site and adhere to study-related follow-up examinations and/or procedures and provide access to participant's medical records.
• Clinical phenotype and neuroimaging consistent with a diagnosis of ATN1 mutation associated Dentatorubral-pallidoluysian atrophy (DRPLA).
• Documented genetic mutation in ATN1.

You may not qualify if…

• Participant has any known contraindication to or unwillingness to undergo lumbar puncture.
• Use of investigational medication within 5 half-lives of the drug at enrollment.
• Participant has any condition that in the opinion of the Site Investigator, would ultimately prevent the completion of study procedures.

Where it is running

• Hawaii Pacific Neuroscience — Honolulu, Hawaii, United States

Full record on ClinicalTrials.gov

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