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

NCT ID NCT06977451

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

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing This study
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

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.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 1/2

Runs two stages together: safety and dose first, then whether the treatment works.

Participants

1 person

The number who actually took part.

Started

Jun 2024

Expected to finish

Jun 2027

An estimate. End dates often move.

Lead sponsor

Other sponsor

The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

63 to 63 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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 stie 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

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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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