Personalized Antisense Oligonucleotide Therapy for A Single Participant With CHCHD10 ALS

n-Lorem Foundation

Status and phase

Active, not recruiting

Phase 2

Phase 1

Conditions

Amyotrophic Lateral Sclerosis

Treatments

Drug: nL-CHCHD-001

Study type

Interventional

Funder types

Other

Identifiers

NCT06392126

23-011476

Details and patient eligibility

About

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

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

Enrollment

1 patient

Sex

Male

Ages

48+ years old

Volunteers

No Healthy Volunteers

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