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

NCT06816498 | Active Not Recruiting Phase 1 DMC FDA Drug

• 1 other identifier: 23-011942
• Study Type: interventional
• Target: 1
• Locations: 1 country
• Sites: 1

Brief 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

Outcome Measures

Primary Outcomes (5)

• Gait

  Change in gait from baseline at 6-, 12-, 18- and 24-months post nL-LMNB1-001 administration as measured by gait motion analysis

  Baseline to 24 months

• Gait

  Change in gait from baseline at 6-, 12-, 18- and 24-months post nL-LMNB1-001 administration as measured by 6-minute walk test

  Baseline to 24 months

• Gait

  Change in gait from baseline at 6-, 12-, 18- and 24-months post nL-LMNB1-001 administration as measured by 25-feet walk test

  Baseline to 24 months

• Neurological functioning

  Change in neurological functioning results from baseline at 6-, 12-, 18- and 24-months post nL-LMNB1-001 administration as measured by formal neuro-psychological evaluation (abnormalities in cognitive functioning such as memory, visual function, and language function).

  Baseline to 24 months

• Brain atrophy

  Change in degree of brain atrophy from baseline at 6-, 12-, 18- and 24-months post nL-LMNB1-001 administration as measured by brain MRI

  Baseline to 24 months

Secondary Outcomes (8)

• Urodynamics

  Baseline to 24 months

• Autonomic function

  Baseline to 24 months

• Autonomic function

  Baseline to 24 months

• Autonomic function

  Baseline to 24 months

• Autonomic function

  Baseline to 24 months

Study Arms (1)

Open Label

EXPERIMENTAL

Drug: nL-LMNB1-001

Interventions

nL-LMNB1-001 DRUG

Personalized antisense oligonucleotide

Open Label

Eligibility Criteria

• Age: 51 Years - 51 Years
• Sex: male
• Healthy Volunteers: No
• Age Groups: Adult (18-64)

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

Sponsors & Collaborators

• n-Lorem Foundation: lead
• Mayo Clinic: collaborator

Study Sites (1)

Mayo Clinic

Rochester, Minnesota, 55905, United States

Location

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 28, 2025
• First Posted: February 10, 2025
• Study Start: March 17, 2025
• Primary Completion (Estimated): March 1, 2027
• Study Completion (Estimated): March 1, 2027
• Last Updated: May 4, 2025

Record last verified: 2025-04

Locations

US (1)