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

Running, not enrolling · Phase 1/Phase 2

Conditions studied: Autosomal Dominant Leukodystrophy

In brief

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

Key facts

Study ID

NCT06816498

Run by

n-Lorem Foundation

People needed

1

Starts

2025-03-17

Expected to finish

2027-03-01

Last updated by the study team

2026-07-13

Who can join

Age: 51 and older, up to 51. Sex: male. Healthy volunteers: not accepted.

You may qualify if…

• Informed consent provided by the participant (when appropriate), and/or participant's parent(s) or legally authorized representative(s).
• Autosomal dominant adult-onset leukodystrophy (ADLD) caused by an LMNB1 duplication mutation
• 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.
• Willingness to follow contraceptive guidance during the intervention period and for at least 40 weeks after the last dose of study intervention

You may not qualify if…

• Participant has any condition that in the opinion of the Site Investigator, would ultimately prevent the completion of study procedures

Where it is running

• Mayo Clinic — Rochester, Minnesota, United States

Full record on ClinicalTrials.gov

Trial information comes from ClinicalTrials.gov and is refreshed daily. TrialsForMe does not provide medical care and does not run the studies it lists.