Personalized Antisense Oligonucleotide for Participants With CHCHD10 ALS

n-Lorem Foundation

Status and phase: Invitation-only; Phase 2; Phase 1

Conditions: Amyotrophic Lateral Sclerosis

Treatments: Drug: nL-CHCHD-001

Study type: Interventional

Funder types: Other

Identifiers

NCT07095686

AAAV3923

Details and patient eligibility

About

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

Enrollment: 9 estimated patients

Sex: All

Ages: 18+ 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 representatives(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

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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9 participants in 1 patient group

Open Label nL-CHCHD-001

Experimental group

Treatment:

Drug: nL-CHCHD-001