Personalized Antisense Oligonucleotide for a Single Participant With CHCHD10 ALS

NCT06977451 | Active Not Recruiting Phase 1 FDA Drug

• 1 other identifier: AAAV1774
• 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). A maximum score of 48 represents normal functioning, and the minimum score is 0.

  Baseline to 12 months

• Motor Functioning

  Change from baseline at 12-months post nL-CHCHD-001 administration in scores on Slow Vital Capacity (SVC) Performance.

  Baseline to 12 months

• Motor Functioning

  Change from baseline at 12-months post nL-CHCHD-001 administration in scores on Handheld Dynamometry (HHD).

  Baseline to 12 months

• Clinical Functioning

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

  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 Efficacy

  Baseline to 12 months

• Safety and Efficacy

  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: 63 Years - 63 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64)

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 stie 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
• Columbia University: collaborator

Study Sites (1)

Columbia University, Irving Medical Center

New York, New York, 10032, 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: May 9, 2025
• First Posted: May 18, 2025
• Study Start: June 24, 2024
• Primary Completion (Estimated): June 1, 2026
• Study Completion (Estimated): June 1, 2026
• Last Updated: July 25, 2025

Record last verified: 2025-05

Locations

US (1)