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NCT07095686

Personalized Antisense Oligonucleotide for Participants With CHCHD10 ALS

Enrolling by Invitation
PHASE1Ages 18+InterventionalTreatment
n-Lorem Foundation
~9 participants
Updated 2026-07-13 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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Where it's being run

1 sites across 1 states
New York1

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 representatives(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 Slow Vital Capacity (SVC)

  • 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)