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

Study Purpose

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

Recruitment Criteria

Accepts Healthy Volunteers Healthy volunteers are participants who do not have a disease or condition, or related conditions or symptoms	No
Study Type An interventional clinical study is where participants are assigned to receive one or more interventions (or no intervention) so that researchers can evaluate the effects of the interventions on biomedical or health-related outcomes. An observational clinical study is where participants identified as belonging to study groups are assessed for biomedical or health outcomes. Searching Both is inclusive of interventional and observational studies.	Interventional
Eligible Ages	51 Years - 51 Years
Gender	Male

More Inclusion & Exclusion Criteria

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 Details

Trial ID: This trial id was obtained from ClinicalTrials.gov, a service of the U.S. National Institutes of Health, providing information on publicly and privately supported clinical studies of human participants with locations in all 50 States and in 196 countries.	NCT06816498
Phase Phase 1: Studies that emphasize safety and how the drug is metabolized and excreted in humans. Phase 2: Studies that gather preliminary data on effectiveness (whether the drug works in people who have a certain disease or condition) and additional safety data. Phase 3: Studies that gather more information about safety and effectiveness by studying different populations and different dosages and by using the drug in combination with other drugs. Phase 4: Studies occurring after FDA has approved a drug for marketing, efficacy, or optimal use.	Phase 1/Phase 2
Lead Sponsor The sponsor is the organization or person who oversees the clinical study and is responsible for analyzing the study data.	n-Lorem Foundation
Principal Investigator The person who is responsible for the scientific and technical direction of the entire clinical study.	N/A
Principal Investigator Affiliation	N/A
Agency Class Category of organization(s) involved as sponsor (and collaborator) supporting the trial.	Other
Overall Status	Enrolling by invitation
Countries	United States
Conditions The disease, disorder, syndrome, illness, or injury that is being studied.	Autosomal Dominant Leukodystrophy

Additional Details

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

Arms & Interventions

Arms

Experimental: Open Label

Interventions

Drug: - nL-LMNB1-001

Personalized antisense oligonucleotide

Contact a Trial Team

If you are interested in learning more about this trial, find the trial site nearest to your location and contact the site coordinator via email or phone. We also strongly recommend that you consult with your healthcare provider about the trials that may interest you and refer to our terms of service below.

Mayo Clinic, Rochester, Minnesota

Status

Address

Mayo Clinic

Rochester, Minnesota, 55905

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