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

n-Lorem Foundation

Status and phase: Active, not recruiting; Phase 2; Phase 1

Conditions: Autosomal Dominant Leukodystrophy

Treatments: Drug: nL-LMNB1-001

Study type: Interventional

Funder types: Other

Identifiers

NCT06816498

23-011942

Details and patient eligibility

About

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

Full 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

Enrollment: 1 patient

Sex: Male

Ages: 51 to 51 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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1 participants in 1 patient group

Open Label

Experimental group

Treatment:

Drug: nL-LMNB1-001