Clinical Trial

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

Active, Not Recruiting Phase 1/2
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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
Protocol Amendment History 2 amendments
This ClinicalTrials.gov record has been amended 2 times since 2025-02-03; most recent amendment 2026-07-09.
Status change: Enrolling by Invitation → Active, Not Recruiting 2025-04-30
Trial Details
NCT Number NCT06816498
Lead Sponsor n-Lorem Foundation
Collaborators: Mayo Clinic
Conditions Autosomal Dominant Leukodystrophy
Enrollment 1 participants
Start Date 2025-03-17
Primary Completion 2027-03 (estimated)
Study Completion 2027-03 (estimated)
Updated on ClinicalTrials.gov 2026-07-13