Personalized Antisense Oligonucleotide Therapy for A Single Participant With CHCHD10 ALS

July 9, 2026 updated by: n-Lorem Foundation

An Open-label Study of an Experimental Antisense Oligonucleotide Treatment for a Single Participant With Amyotrophic Lateral Sclerosis (ALS) Due to a Pathogenic Variant in CHCHD10

This research project entails delivery of a personalized antisense oligonucleotide (ASO) drug designed for a single participant with amyotrophic lateral sclerosis (ALS) due to a pathogenic variant in CHCHD10

Study Overview

Status

Active, not recruiting

Conditions

Intervention / Treatment

Detailed Description

This is an interventional study to evaluate the safety and efficacy of treatment with an individualized antisense oligonucleotide (ASO) treatment in a single participant with amyotrophic lateral sclerosis (ALS) due to a pathogenic variant in CHCHD10

Study Type

Interventional

Enrollment (Actual)

1

Phase

  • Phase 2
  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • Florida
      • Jacksonville, Florida, United States, 32224
        • Mayo Clinic

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

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 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 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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Open Label
Drug: nL-CHCHD-001
Personalized antisense oligonucleotide

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Clinical Functioning
Time Frame: Baseline 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).
Baseline to 12 months
Clinical Functioning
Time Frame: Baseline to 12 months
Change from baseline at 12-months post nL-CHCHD-001 administration in scores on ALS Cognitive Behavioral Screen (ALS-CBS)
Baseline to 12 months
Clinical Functioning
Time Frame: Baseline 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).
Baseline to 12 months
Clinical Functioning
Time Frame: Baseline to 12 months
Change from baseline at 12-months post nL-CHCHD-001 administration in scores on vital capacity performance.
Baseline to 12 months
Disease biomarkers
Time Frame: Baseline to 12 months
Change from baseline at 12-months post nL-CHCHD-001 administration in serum/plasma and CSF neurofilament light chain levels
Baseline to 12 months
Survival
Time Frame: Baseline to 12 months
Change from baseline at 12-months post nL-CHCHD-001 administration in survival status
Baseline to 12 months

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Safety and tolerability
Time Frame: Baseline to 12 months
Incidence and severity of adverse events
Baseline to 12 months
Safety and tolerability
Time Frame: Baseline to 12 months
Emergent abnormalities in physical exam
Baseline to 12 months
Safety and tolerability
Time Frame: Baseline to 12 months
Emergent abnormalities in neurological exam
Baseline to 12 months
Safety and tolerability
Time Frame: Baseline to 12 months
Emergent abnormalities in safety labs (CSF, chemistry, hematology, coagulation, and urinalysis)
Baseline to 12 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

n-Lorem Foundation

Collaborators

Mayo Clinic

Investigators

  • Principal Investigator: Björn E. Oskarsson, M.D., Mayo Clinic

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

April 16, 2024

Primary Completion (Estimated)

April 1, 2027

Study Completion (Estimated)

April 1, 2027

Study Registration Dates

First Submitted

April 24, 2024

First Submitted That Met QC Criteria

April 26, 2024

First Posted (Actual)

April 30, 2024

Study Record Updates

Last Update Posted (Actual)

July 13, 2026

Last Update Submitted That Met QC Criteria

July 9, 2026

Last Verified

July 1, 2026

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 23-011476

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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