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Tailored genetic drug takes aim at a rare form of ALS

NCT ID NCT07095686

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

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

Summary

This study tests a personalized drug called an antisense oligonucleotide, designed specifically for people with ALS caused by a particular change in the CHCHD10 gene. The drug aims to slow or stop the disease by targeting the genetic root cause. The trial involves a small group of participants and will measure changes in muscle strength, breathing, and daily function over 12 months.

What this could mean

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

Active substance
personalized antisense oligonucleotide (nL-CHCHD-001)
What this could lead to
If successful, this approach could slow or halt ALS progression in people with this specific genetic cause, offering a targeted treatment option.
What could go wrong
This is an early-phase, small study (9 participants) focused on safety and initial effectiveness. The treatment may not work for everyone, and side effects are possible.

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

Locations

  • Columbia University, Irving Medical Center

    New York, New York, 10032, United States

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