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Personalized Antisense Oligonucleotide for Participants With CHCHD10 ALS

Personalized Antisense Oligonucleotide for Participants With CHCHD10 ALS

Enrolling By Invitation
18 years and older
All
Phase 1/2

Overview

This research project entails delivery of a personalized antisense oligonucleotide (ASO) drug designed for individual participants with amyotrophic lateral sclerosis (ALS) due to a pathogenic variant in CHCHD10

Eligibility

Inclusion Criteria:

  • Informed consent/assent provided by the participant (when appropriate), and/or participant's parent(s) or legally authorized representatives(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

Print
Study details
    Amyotrophic Lateral Sclerosis

NCT07095686

n-Lorem Foundation

9 January 2026

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FAQs

Learn more about clinical trials

What is a clinical trial?

A clinical trial is a study designed to test specific interventions or treatments' effectiveness and safety, paving the way for new, innovative healthcare solutions.

Why should I take part in a clinical trial?

Participating in a clinical trial provides early access to potentially effective treatments and directly contributes to the healthcare advancements that benefit us all.

How long does a clinical trial take place?

The duration of clinical trials varies. Some trials last weeks, some years, depending on the phase and intention of the trial.

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Compensation varies per trial. Some offer payment or reimbursement for time and travel, while others may not.

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Clinical trials follow strict ethical guidelines and protocols to safeguard participants' health. They are closely monitored and safety reviewed regularly.
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