Personalized Antisense Oligonucleotide Therapy for A Single Patient With CHCHD10 ALS (nL18576)
Age: 18+
Sex: Any
Trial Phase: Phase 1 & 2
Sponsor: n-Lorem Foundation
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)
What You Need to Know Before You Apply
What is the purpose of this trial?
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
Are You a Good Fit for This Trial?
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
What Are the Treatments Tested in This Trial?
How Is the Trial Designed?
1
Treatment groups
Experimental Treatment
Group I: Open LabelExperimental Treatment1 Intervention
Find a Clinic Near You
Who Is Running the Clinical Trial?
n-Lorem Foundation
Lead Sponsor
Trials
5
Recruited
5+
Mayo Clinic
Collaborator
Trials
3,427
Recruited
3,221,000+
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