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Antisense Oligonucleotide

Antisense Oligonucleotide Therapy for ALS

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
No Placebo-Only Group

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.
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.
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 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

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Clinical Functioning
Secondary study objectives
Disease biomarkers
Safety and tolerability

Awards & Highlights

No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups
Experimental Treatment
Group I: Open LabelExperimental Treatment1 Intervention

Find a Location

Who is running the clinical trial?

n-Lorem FoundationLead Sponsor
3 Previous Clinical Trials
3 Total Patients Enrolled
Mayo ClinicOTHER
3,353 Previous Clinical Trials
3,061,018 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
~0 spots leftby Apr 2025
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