Status:
NOT_YET_RECRUITING
Personalized Antisense Oligonucleotide for A Single Participant (nL62541) With ATN1 Gene Mutation
Lead Sponsor:
n-Lorem Foundation
Collaborating Sponsors:
Dell Children's Medical Center of Central Texas
Conditions:
Dentatorubral-Pallidoluysian Atrophy
Eligibility:
MALE
22+ years
Phase:
PHASE1
PHASE2
Brief Summary
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 ...
Detailed 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 heterozygo...
Eligibility Criteria
Inclusion
- 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
- 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
Key Trial Info
Start Date :
November 1 2025
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
November 1 2027
Estimated Enrollment :
1 Patients enrolled
Trial Details
Trial ID
NCT07221760
Start Date
November 1 2025
End Date
November 1 2027
Last Update
October 28 2025
Active Locations (1)
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1
Dell Children's
Austin, Texas, United States, 78723