Personalized Antisense Oligonucleotide for a Single Participant With CHCHD10 ALS

An Open-Label Single Center, Single Participant Study of an Experimental Antisense Oligonucleotide Treatment for Amyotrophic Lateral Sclerosis (ALS) Due to an Arg15Leu Pathogenic Variant in CHCHD10

Status

Active, not recruiting

Phases

Phase 1Phase 2

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT06977451

Enrollment

Registered

2025-05-18

Start date

2024-06-24

Completion date

2027-06-01

Last updated

2026-07-13

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Amyotrophic Lateral Sclerosis

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

Interventions

DRUGnL-CHCHD-001

Personalized Antisense Oligonucleotide

Study design

Allocation

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Masking

NONE

Eligibility

Sex/Gender

ALL

Age

63 Years to 63 Years

Healthy volunteers

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 stie 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

Design outcomes

Primary

Measure	Time frame	Description
Clinical Functioning	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). A maximum score of 48 represents normal functioning, and the minimum score is 0.
Motor Functioning	Baseline to 12 months	Change from baseline at 12-months post nL-CHCHD-001 administration in scores on Slow Vital Capacity (SVC) Performance.

Secondary

Measure	Time frame	Description
Disease Biomarkers	Baseline to 12 months	Change from baseline at 12-months post nL-CHCHD-001 administration in serum and cerebrospinal fluid neurofilament light chain levels
Safety and Tolerability	Baseline to 12 months	Incidence and Severity of Adverse Events
Safety and Efficacy	Baseline to 12 months	Emergent abnormalities in neurological exam

Countries

United States

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Jul 14, 2026