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ACTIVE NOT RECRUITING
NCT06816498
PHASE1/PHASE2
Personalized Antisense Oligonucleotide Therapy for A Single Participant With LMNB1 Mutation Associated Autosomal Dominant Leukodystrophy (ADLD)
Sponsor: n-Lorem Foundation
View on ClinicalTrials.gov
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
Official title: 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)
Key Details
Gender
MALE
Age Range
51 Years - 51 Years
Study Type
INTERVENTIONAL
Enrollment
1
Start Date
2025-03-17
Completion Date
2027-03
Last Updated
2025-05-04
Healthy Volunteers
No
Conditions
Interventions
DRUG
nL-LMNB1-001
Personalized antisense oligonucleotide
Locations (1)
Mayo Clinic
Rochester, Minnesota, United States