NCT06816498

Personalized Antisense Oligonucleotide Therapy for A Single Participant With LMNB1 Mutation Associated Autosomal Dominant Leukodystrophy (ADLD)

An Open-label, Single-center, Single-participant Study of an Experimental Antisense Oligonucleotide Treatment for a Patient With LMNB1 Mutation Associated Autosomal Dominant Leukodystrophy (ADLD)

• Status: Active Not Recruiting
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 1 (actual)
• Sponsor: n-Lorem Foundation · Academic / Other
• Sex: Male
• Age: 51 Years – 51 Years
• Healthy volunteers: Not accepted

Summary

This research project entails delivery of a personalized antisense oligonucleotide (ASO) drug designed for a single participant with Autosomal Dominant Leukodystrophy (ADLD) due to LMNB1 mutation

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 Autosomal Dominant Leukodystrophy (ADLD) due to LMNB1 mutation

Conditions

• Autosomal Dominant Leukodystrophy

Interventions

Type	Name	Description
DRUG	nL-LMNB1-001	Personalized antisense oligonucleotide

Timeline

• Start date: 2025-03-17
• Primary completion: 2027-03-01
• Completion: 2027-03-01
• First posted: 2025-02-10
• Last updated: 2025-05-04

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study