Personalized Antisense Oligonucleotide Therapy for A Single Participant With CHCHD10 ALS

Amyotrophic Lateral Sclerosis Clinical Trial

Official title:

An Open-label Study of an Experimental Antisense Oligonucleotide Treatment for a Single Participant With Amyotrophic Lateral Sclerosis (ALS) Due to a Pathogenic Variant in CHCHD10

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT06392126
• Other study ID #: 23-011476
• Status: Active, not recruiting
• Phase: Phase 1/Phase 2
• Start date: April 16, 2024
• Est. completion date: April 2025

Study information

• Verified date: April 2024
• Source: n-Lorem Foundation
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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

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

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 1
• Est. completion date: April 2025
• Est. primary completion date: April 2025
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 48 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Amyotrophic Lateral Sclerosis
• Motor Neuron Disease
• Sclerosis

Intervention

Drug:
• nL-CHCHD-001
Personalized antisense oligonucleotide

Locations

Country	Name	City	State
United States	Mayo Clinic	Jacksonville	Florida

Sponsors (2)

Lead Sponsor	Collaborator
n-Lorem Foundation	Mayo Clinic

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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
Primary	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
Primary	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
Primary	Clinical Functioning	Change from baseline at 12-months post nL-CHCHD-001 administration in scores on vital capacity performance.	Baseline to 12 months
Secondary	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
Secondary	Safety and tolerability	Incidence and severity of adverse events	Baseline to 12 months
Secondary	Safety and tolerability	Emergent abnormalities in physical exam	Baseline to 12 months
Secondary	Safety and tolerability	Emergent abnormalities in neurological exam	Baseline to 12 months
Secondary	Safety and tolerability	Emergent abnormalities in safety labs (CSF, chemistry, hematology, coagulation, and urinalysis)	Baseline to 12 months