Personalized Antisense Oligonucleotide Therapy for A Single Patient With CHCHD10 ALS (nL18576)

Phase 2Not Yet RecruitingNCT07423494

n-Lorem Foundation1 enrolled

Overview

This research project entails delivery of a personalized antisense oligonucleotide (ASO) drug designed for a single participant with amyotrophic lateral sclerosis (ALS) due to a pathogenic variant in CHCHD10

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 amyotrophic lateral sclerosis (ALS) due to a pathogenic variant in CHCHD10

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Amyotrophic Lateral Sclerosis

Interventions

nL-CHCHD-001DRUG

Personalized antisense oligonucleotide

Eligibility

Sex: ALLMin age: 18 Years

Medical Language ↔ Plain English

Inclusion Criteria: * Informed consent/assent provided by the participant (when appropriate), and/or participant's parent(s) or legally authorized representative(s) * 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 * Genetically confirmed neurological disorder Exclusion Criteria: * Participant has any condition that in the opinion of the Site Investigator, would ultimately prevent the completion of study procedures * Use of an investigational medication within less than 5 half-lives of the drug at enrollment

Locations (1)

Mayo Clinic

Rochester, Minnesota, United States

Outcomes

Primary Outcomes

Clinical Functioning

Change from baseline at 12-months post nL-CHCHD-001 administration in scores on Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R).

Time frame: Baseline to 12 Months

Motor Function

Change from baseline at 12-months post nL-CHCHD-001 administration in Forced Vital Capacity (FVC)