Personalized Antisense Oligonucleotide for A Single Participant (nL62541) With ATN1 Gene Mutation

n-Lorem Foundation

Status and phase

Not yet enrolling

Phase 2

Phase 1

Conditions

Dentatorubral-Pallidoluysian Atrophy

Treatments

Drug: nL-ATN1-001

Study type

Interventional

Funder types

Other

Identifiers

NCT07221760

STUDY00007386

Details and patient eligibility

About

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

Full description

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

Enrollment

1 estimated patient

Sex

Male

Ages

22+ years old

Volunteers

No Healthy Volunteers

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