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Personalized Antisense Oligonucleotide Therapy for CHCHD10 ALS

This study is testing a personalized medicine called nL-CHCHD-001 for a single patient with Amyotrophic Lateral Sclerosis (ALS) caused by a specific genetic change in the CHCHD10 gene. The goal is to see if nL-CHCHD-001 is safe and effective in improving clinical function and motor skills. To join, you must be an adult (18 or older) with a confirmed genetic neurological disorder, able to travel to the study site, and provide informed consent. The study is currently unclear on its recruitment status, as it's designed for one participant. Researchers will measure changes in your clinical function and motor skills over 12 months to see how well the treatment works.

Study design
This is an interventional study designed for a single participant. It aims to evaluate the safety and effectiveness of the treatment.
What's involved
You would need to travel to the study site for follow-up examinations and procedures. The study will measure your progress for 12 months.
Compensation
Not stated in the trial record.
Follow-up
Participants will be followed for 12 months after the start of the study to assess clinical and motor function.

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.

NCT07423494

Personalized Antisense Oligonucleotide Therapy for A Single Patient With CHCHD10 ALS (nL18576)

Not Yet Recruiting
PHASE1Ages 18+InterventionalTreatment
n-Lorem Foundation
~1 participants
Updated 2026-02-20 on ClinicalTrials.gov
What's tested:nL-CHCHD-001

At a glance

Recruiting sites
0 of 1 listed site is recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Clinical Functioning
Measured over Baseline to 12 Months
+5 more outcomes measured

Conditions

Amyotrophic Lateral Sclerosis

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NCT07423494

Where you'd take part

This study runs at 1 site. They're the same protocol — you choose where, and that choice sets who your contact draft is addressed to.

  • Mayo Clinic

    Rochester, Minnesotano site contact published

Sites open and close at different times, so the status above is per site — it can differ from the study's overall status.

Who to contact

This trial hasn't published a contact. View it on ClinicalTrials.gov

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Eligibility criteria

Inclusion

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

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

What this trial measures

  • Clinical FunctioningBaseline to 12 Months

    Change from baseline at 12-months post nL-CHCHD-001 administration in scores on Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R).

  • Motor FunctionBaseline to 12 months

    Change from baseline at 12-months post nL-CHCHD-001 administration in Forced Vital Capacity (FVC)

  • Motor FunctionBaseline to 12 months

    Change from baseline at 12-months post nL-CHCHD-001 administration in muscle strength as quantified by Handheld Dynamometry (HHD)

  • Clinical FunctioningBaseline to 12 months

    Change from baseline at 12-months post nL-CHCHD-001 administration in Edinburgh Cognitive and Behavioral ALS Screen (ECAS) score

  • Clinical FunctioningBaseline to 12 months

    Change from baseline at 12-months post nL-CHCHD-001 administration in scores on ALS Cognitive Behavioral Screen (ALS-CBS)

  • Clinical FunctioningBaseline to 12 months

    Change from baseline at 12-months post nL-CHCHD-001 administration in scores on Amyotrophic Lateral Sclerosis Assessment Questionnaire 5 (ALSAQ-5)