Antisense Oligonucleotide Therapy for ALS

Recruiting in Palo Alto (17 mi)

Overseen ByBjörn E. Oskarsson, M.D.

Age: 18+

Sex: Male

Travel: May be covered

Time Reimbursement: Varies

Trial Phase: Phase 1 & 2

Waitlist Available

Sponsor: n-Lorem Foundation

No Placebo Group

Trial Summary

What is the purpose of this trial?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

Eligibility Criteria

This trial is specifically for one person with a rare form of ALS (Lou Gehrig's disease) caused by a CHCHD10 gene mutation. The participant must have this genetic variant and meet other undisclosed criteria.

Inclusion Criteria

My neurological disorder is confirmed through genetic testing.

Participant Groups

The study is testing an individualized drug called nL-CHCHD-001, which is an antisense oligonucleotide designed to target the genetic cause of ALS in this single participant.

1Treatment groups

Experimental Treatment

Group I: Open LabelExperimental Treatment1 Intervention