Menu
Not yet recruiting NCT07423494

Personalized Antisense Oligonucleotide Therapy for A Single Patient With CHCHD10 ALS (nL18576)

Phase I / Phase II Interventional Amyotrophic Lateral Sclerosis

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: nL-CHCHD-001.
Who it may be relevant to
Registry conditions: Amyotrophic Lateral Sclerosis. Basic parameters: from 18 years · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
United States
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Unsure about the terms? Read our patient guide →

Official title

An Open-label Single Center, Single Participant Study of an Experimental Antisense Oligonucleotide Treatment for Amyotrophic Lateral Sclerosis (ALS) Due to CHCHD10 Gene Mutation

Overview

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

Interventions

  • Drug nL-CHCHD-001
    Personalized antisense oligonucleotide

Primary outcome measures

  • Clinical Functioning [Time frame: Baseline to 12 Months]
  • Motor Function [Time frame: Baseline to 12 months]
  • Motor Function [Time frame: Baseline to 12 months]
  • Clinical Functioning [Time frame: Baseline to 12 months]
  • Clinical Functioning [Time frame: Baseline to 12 months]
  • Clinical Functioning [Time frame: Baseline to 12 months]
Secondary outcome measures (5)
  • Disease Biomarkers [Time frame: Baseline to 12 months]
  • Safety and Tolerability [Time frame: Baseline to 12 months]
  • Safety and Tolerability [Time frame: Baseline to 12 months]
  • Safety and Tolerability [Time frame: Baseline to 12 months]
  • Safety and Tolerability [Time frame: Baseline to 12 months]

Eligibility criteria

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

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation
N/A
Model
Single group
Masking
Open label
Primary purpose
Treatment

Study locations

United States · 1 center
  • Mayo Clinic — Rochester

Identifiers

NCT: NCT07423494 · 25-010790

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗