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

NCT07423494 | Not Yet Recruiting Phase 1 DMC FDA Drug

• 1 other identifier: 25-010790
• Study Type: interventional
• Target: 1
• Locations: 1 country
• Sites: 1

Brief Summary

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

Conditions

Amyotrophic Lateral Sclerosis

Outcome Measures

Primary Outcomes (6)

• Clinical Functioning

  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

• Motor Function

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

  Baseline to 12 months

• Motor Function

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

  Baseline to 12 months

• Clinical Functioning

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

  Baseline to 12 months

• Clinical Functioning

  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

  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

Secondary Outcomes (5)

• Disease Biomarkers

  Baseline to 12 months

• Safety and Tolerability

  Baseline to 12 months

• Safety and Tolerability

  Baseline to 12 months

• Safety and Tolerability

  Baseline to 12 months

• Safety and Tolerability

  Baseline to 12 months

Study Arms (1)

Open Label

EXPERIMENTAL

Drug: nL-CHCHD-001

Interventions

nL-CHCHD-001 DRUG

Personalized antisense oligonucleotide

Open Label

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Mayo Clinic: collaborator
• n-Lorem Foundation: lead

Study Sites (1)

Mayo Clinic

Rochester, Minnesota, 55905, United States

Location

MeSH Terms

Conditions

Amyotrophic Lateral Sclerosis

Condition Hierarchy (Ancestors)

Spinal Cord Diseases; Central Nervous System Diseases; Nervous System Diseases; Motor Neuron Disease; Neurodegenerative Diseases; TDP-43 Proteinopathies; Neuromuscular Diseases; Proteostasis Deficiencies; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 13, 2026
• First Posted: February 20, 2026
• Study Start: March 1, 2026
• Primary Completion (Estimated): March 1, 2027
• Study Completion (Estimated): March 1, 2027
• Last Updated: February 20, 2026

Record last verified: 2026-02

Locations

US (1)