Personalized Antisense Oligonucleotide Therapy for A Single Participant With CHCHD10 ALS

Phase 1

Active, not recruiting

• Conditions: Amyotrophic Lateral Sclerosis
• Interventions: Drug: nL-CHCHD-001

• Registration Number: NCT06392126
• Lead Sponsor: n-Lorem Foundation

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

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 Timeline

• Study Start: 2024-04-16
• Study First Submitted: 2024-04-24
• Study First Submitted QC: 2024-04-26
• Study First Posted: 2024-04-30
• Status Verified: 2025-05
• Last Update Submitted: 2025-05-09
• Last Update Posted: 2025-05-14
• Primary Completion: 2026-04
• Study Completion: 2026-04

Recruitment & Eligibility

• Status: ACTIVE_NOT_RECRUITING
• Sex: Male
• Target Recruitment: 1

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 & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Open Label	nL-CHCHD-001	-

Primary Outcome Measures

Name	Time	Method
Clinical Functioning	Baseline to 12 months	Change from baseline at 12-months post nL-CHCHD-001 administration in scores on vital capacity performance.

Secondary Outcome Measures

Name	Time	Method
Disease biomarkers	Baseline to 12 months	Change from baseline at 12-months post nL-CHCHD-001 administration in serum/plasma and CSF neurofilament light chain levels
Safety and tolerability	Baseline to 12 months	Emergent abnormalities in safety labs (CSF, chemistry, hematology, coagulation, and urinalysis)

Trial Locations

Locations (1)

Mayo Clinic; 🇺🇸 Jacksonville, Florida, United States