Personalized Antisense Oligonucleotide Therapy for a Single Participant with ATN1 Gene Mutation

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

ACTIVE_NOT_RECRUITING

Clinical Phase

PHASE1/PHASE2

Total Enrollment

1 participants

Study Classification

INTERVENTIONAL

Study Start Date

2024-02-21

Study Completion Date

2026-02-28

Brief Summary

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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

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Detailed Description

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This is an interventional study to evaluate the safety and efficacy of treatment with an individualized antisense oligonucleotide (ASO) treatment in a single participant with DRPLA due to a heterozygous pathogenic CAG trinucleotide expansion in ATN1

Conditions

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Dentatorubral-Pallidoluysian Atrophy

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Open Label

Group Type EXPERIMENTAL

nL-ATN1-002

Intervention Type DRUG

Personalized antisense oligonucleotide

Interventions

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nL-ATN1-002

Personalized antisense oligonucleotide

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

* 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.
* Genetically confirmed Dentatorubral-pallidoluysian atrophy (DRPLA) due to ATN1 mutation

Exclusion Criteria

* Use of investigational medication within 5 half-lives of the drug at enrolment
* Participant has any condition that in the opinion of the Site Investigator, would ultimately prevent the completion of study procedures.

Minimum Eligible Age

17 Years

Maximum Eligible Age

17 Years

Eligible Sex

MALE

Accepts Healthy Volunteers

No