Personalized Antisense Oligonucleotide Therapy for A Single Participant With CHCHD10 ALS

NCT06392126 | Active Not Recruiting Phase 1 FDA Drug

• 1 other identifier: 23-011476
• 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

• 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

• Clinical Functioning

  Change from baseline at 12-months post nL-CHCHD-001 administration in scores on vital capacity performance.

  Baseline to 12 months

• Disease biomarkers

  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

  Change from baseline at 12-months post nL-CHCHD-001 administration in survival status

  Baseline to 12 months

Secondary Outcomes (4)

• 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: 48 Years+
• Sex: male
• 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

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

Study Sites (1)

Mayo Clinic

Jacksonville, Florida, 32224, 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 Officials

• Björn E. Oskarsson, M.D.

  Mayo Clinic

  PRINCIPAL INVESTIGATOR

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: April 24, 2024
• First Posted: April 30, 2024
• Study Start: April 16, 2024
• Primary Completion: April 1, 2026
• Study Completion: April 1, 2026
• Last Updated: August 5, 2025

Record last verified: 2025-05

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)