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Clinical Trials/NCT06816498
NCT06816498Active, not recruitingPhase 1

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)

n-Lorem Foundation1 site in 1 country1 target enrollmentStarted: March 17, 2025Last updated:
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Interventions
Drugs

Trial Snapshot

Phase
Phase 1
Status
Active, not recruiting
Enrollment
1
Locations
1
Primary Endpoint
Gait
Study OverviewStudy DesignEligibility CriteriaArms & InterventionsOutcomesInvestigatorsSitesRecords & IdentifiersSimilar Trials

Study Overview

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

Detailed Description

This is an interventional study to evaluate the safety and efficacy of treatment with an individualized antisense oligonucleotide (ASO) treatment in a single participant with Autosomal Dominant Leukodystrophy (ADLD) due to LMNB1 mutation

Study Design

Study Type
Interventional
Allocation
Na
Intervention Model
Single Group
Primary Purpose
Treatment
Masking
None

Eligibility Criteria

Ages
51 Years to 51 Years (Adult)
Sex
Male
Accepts Healthy Volunteers
No

Inclusion Criteria

  • •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

Exclusion Criteria

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

Arms & Interventions

Open Label

Experimental

Intervention: nL-LMNB1-001 (Drug)

Outcomes

Primary Outcomes

Gait

Time Frame: Baseline to 24 months

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

Neurological functioning

Time Frame: Baseline to 24 months

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).

Brain atrophy

Time Frame: Baseline to 24 months

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

Secondary Outcomes

  • Incidence of Treatment-Emergent abnormalities in physical and neurological exams [Safety and Tolerability](Baseline to 24 months)
  • Urodynamics(Baseline to 24 months)
  • Incidence of Treatment-Emergent abnormalities in safety labs (CSF, chemistry, hematology, coagulation, and urinalysis) [Safety and Tolerability](Baseline to 24 months)
  • Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability](Baseline to 24 months)
  • Autonomic function(Baseline to 24 months)

Investigators

Sponsor Class
Other
Responsible Party
Sponsor

Study Sites (1)

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