Personalized Antisense Oligonucleotide Therapy for A Single Participant With CHCHD10 ALS

Plain-language summary

This Phase 1/2 study by the n-Lorem Foundation involves delivering nL-CHCHD-001, a personalized antisense oligonucleotide (ASO) drug designed for a single participant with amyotrophic lateral sclerosis (ALS) caused by a pathogenic variant in CHCHD10. Eligibility requires informed consent/assent from the participant and/or their parent(s) or legally authorized representative(s), the ability to travel to the study site, and compliance with follow-up assessments. The trial is active but no longer recruiting.

The summary above is a plain-language rendering of the official record. The original English title is shown for reference.

Trial details

Phase

PHASE1/PHASE2

Status

Active, not recruiting

Sponsor

n-Lorem Foundation

Intervention

nL-CHCHD-001

Start date

4/16/2024

Estimated completion

4/1/2027

Countries

United States

Related genes

Locations

• Jacksonville, United States

Eligibility criteria

Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.

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