← Back to Search

Antisense Oligonucleotide

Open Label for Amyotrophic Lateral Sclerosis

Phase 1 & 2

Waitlist Available

Led By Björn E. Oskarsson, M.D.

Research Sponsored by n-Lorem Foundation

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Genetically confirmed neurological disorder

Be older than 18 years old

Timeline

Screening 3 weeks

Treatment Varies

Follow Up baseline to 12 months

Awards & highlights

Study Summary

"This trial involves testing a personalized drug called antisense oligonucleotide (ASO) on one person with amyotrophic lateral sclerosis (ALS) caused by a specific genetic mutation in the CHCH

Who is the study for?

This trial is specifically for one person with a rare form of ALS (Lou Gehrig's disease) caused by a CHCHD10 gene mutation. The participant must have this genetic variant and meet other undisclosed criteria.Check my eligibility

What is being tested?

The study is testing an individualized drug called nL-CHCHD-001, which is an antisense oligonucleotide designed to target the genetic cause of ALS in this single participant.See study design

What are the potential side effects?

Potential side effects are not listed, but ASO therapies can sometimes cause injection site reactions, flu-like symptoms, or changes in liver enzymes.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

Select...

My neurological disorder is confirmed through genetic testing.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ baseline to 12 months

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~baseline to 12 months

This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline to 12 months for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Clinical Functioning

Secondary outcome measures

Disease biomarkers

Safety and tolerability

Trial Design

1Treatment groups

Experimental Treatment

Group I: Open LabelExperimental Treatment1 Intervention

0 / 1Eligibility criteria met

Find a Location

Who is running the clinical trial?

n-Lorem FoundationLead Sponsor

2 Previous Clinical Trials

2 Total Patients Enrolled

Mayo ClinicOTHER

3,227 Previous Clinical Trials

3,771,667 Total Patients Enrolled

10 Trials studying Amyotrophic Lateral Sclerosis

5,692 Patients Enrolled for Amyotrophic Lateral Sclerosis

Björn E. Oskarsson, M.D.Principal InvestigatorMayo Clinic

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Are there any opportunities for eligible individuals to participate in this ongoing research study?

"As per clinicaltrials.gov, the current status of this trial does not involve patient recruitment. The study was first listed on 4/16/2024 and last modified on 4/26/2024. Although this specific trial is not enrolling participants at present, there are approximately 531 other trials actively seeking candidates."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

Personalized Antisense Oligonucleotide Therapy for A Single Participant With CHCHD10 ALS

2024. "Personalized Antisense Oligonucleotide Therapy for A Single Participant With CHCHD10 ALS". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT06392126.

All > Lou Gehrigs Disease > Als