Study of an Experimental Antisense Oligonucleotide Treatment for a Patient With LMNB1 Mutation Associated Autosomal Dominant Leukodystrophy (ADLD)

Overview

About this study

The purpose of this study is to determine the efficacy and safety of an experimental drug, an anti-sense oligonucleotide (ASO) called nL-LMNB1-001. Currently there is no standard therapy for LMNB1 mutation associated Autosomal Dominant Leukodystrophy (ADLD), a fatal central nervous system white matter demyelinating disorder.

Participation eligibility

Participant eligibility includes age, gender, type and stage of disease, and previous treatments or health concerns. Guidelines differ from study to study, and identify who can or cannot participate. There is no guarantee that every individual who qualifies and wants to participate in a trial will be enrolled. Contact the study team to discuss study eligibility and potential participation.

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.

Exclusion Criteria:

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

Note: Other protocol defined Inclusion/Exclusion Criteria may apply.

Eligibility last updated 9/18/2024. Questions regarding updates should be directed to the study team contact

Participating Mayo Clinic locations

Study statuses change often. Please contact the study team for the most up-to-date information regarding possible participation.

Mayo Clinic Location	Status	Contact
Rochester, Minn. Mayo Clinic principal investigator Brendan Lanpher, M.D.	Closed-enrolling by invitation What is this? (?) "Close" Not open to everyone who meets the eligibility criteria, but only those invited to participate by the study team.	Contact information: Center for Multiple Sclerosis and Autoimmune Neurology DLCMSANResCoor@mayo.edu

Mayo Clinic Location

Status

Contact

Rochester, Minn.

Mayo Clinic principal investigator

Brendan Lanpher, M.D.

Closed-enrolling by invitation

Contact information:

Center for Multiple Sclerosis and Autoimmune Neurology

DLCMSANResCoor@mayo.edu

More information

Publications

Publications are currently not available

Clinical Trials