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

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

ACTIVE_NOT_RECRUITING

Clinical Phase

PHASE1/PHASE2

Total Enrollment

1 participants

Study Classification

INTERVENTIONAL

Study Start Date

2025-03-17

Study Completion Date

2027-03-31

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

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

Related Clinical Trials

Explore similar clinical trials based on study characteristics and research focus.

Personalized Antisense Oligonucleotide Therapy for A Single Participant With TARDBP ALS

NCT07095712

Amyotrophic Lateral Sclerosis

ACTIVE_NOT_RECRUITING PHASE1/PHASE2

Personalized Antisense Oligonucleotide Therapy for A Single Participant With CHCHD10 ALS

NCT06392126

Amyotrophic Lateral Sclerosis

ACTIVE_NOT_RECRUITING PHASE1/PHASE2

A Study to Evaluate the Safety of AB-1003 (Previously LION-101) in Subjects With Genetic Confirmation of LGMD2I/R9 (Part1)

NCT05230459

RECRUITING PHASE1/PHASE2

A Trial to Learn More About an Experimental Gene Therapy Called Bidridistrogene Xeboparvovec (SRP-9003) as a Possible Treatment for Limb Girdle Muscular Dystrophy 2E/R4

NCT06246513

Limb-girdle Muscular Dystrophy

ACTIVE_NOT_RECRUITING PHASE3

Safety of Cultured Allogeneic Adult Umbilical Cord Derived Mesenchymal Stem Cell Intrathecal Injection for ALS

NCT05003921

Amyotrophic Lateral Sclerosis

SUSPENDED PHASE1

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

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

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Autosomal Dominant Leukodystrophy

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

Review each arm or cohort in the study, along with the interventions and objectives associated with them.

Open Label

Group Type EXPERIMENTAL

nL-LMNB1-001

Intervention Type DRUG

Personalized antisense oligonucleotide

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

nL-LMNB1-001

Personalized antisense oligonucleotide

Intervention Type DRUG

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

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