Custom-Made genetic drug takes aim at rare form of ALS

NCT ID NCT06977451

Ongoing Disease control Sponsor: n-Lorem Foundation Source: ClinicalTrials.gov ↗

First seen Jul 13, 2026 · Last updated Jul 14, 2026 · Updated 1 time

Summary

This trial tests a personalized medicine approach for a single person with amyotrophic lateral sclerosis (ALS) caused by a specific mutation in the CHCHD10 gene. The experimental drug, called an antisense oligonucleotide, is designed to target the genetic root of the disease. Researchers will monitor changes in muscle function, breathing, and thinking skills over 12 months to see if the treatment can slow the disease's progression.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance: nL-CHCHD-001 (personalized antisense oligonucleotide)
What this could lead to: If successful, this approach could point toward a treatment for ALS caused by this specific genetic mutation, potentially slowing disease progression.
What could go wrong: This is an early, single-participant study, so results may not apply to others. The treatment may not work or could cause side effects.

This is an AI summary of the original study and may miss details. Read our disclaimer.

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Conditions

The condition(s) this trial relates to.

amyotrophic lateral sclerosis

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

AMYOTROPHIC LATERAL SCLEROSIS

Contacts and locations

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Locations

Columbia University, Irving Medical Center

New York, New York, 10032, United States

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