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

NCT06816498 · Status: ACTIVE_NOT_RECRUITING · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 1

Last updated 2025-05-04

No results posted yet for this study

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

Conditions

  • Autosomal Dominant Leukodystrophy

Interventions

DRUG

nL-LMNB1-001

Personalized antisense oligonucleotide

Sponsors & Collaborators

  • Mayo Clinic

    collaborator OTHER

  • n-Lorem Foundation

    lead OTHER

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
51 Years
Max Age
51 Years
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2025-03-17
Primary Completion
2027-03-31
Completion
2027-03-31
FDA Drug
Yes

Countries

  • United States

Study Locations

More Related Trials

Entities

Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT06816498 on ClinicalTrials.gov