Antisense Oligonucleotide Therapy for ALS
Age: 18+
Sex: Male
Trial Phase: Phase 1 & 2
Sponsor: n-Lorem Foundation
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)
Trial Summary
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
Research Team
BE
Björn E. Oskarsson, M.D.
Principal Investigator
Mayo Clinic
Eligibility Criteria
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.Inclusion Criteria
Informed consent/assent provided by the participant (when appropriate), and/or participant's parent(s) or legally authorized representative(s)
My neurological disorder is confirmed through genetic testing.
I can travel to the study location and follow the study's requirements.
Exclusion Criteria
Participant has any condition that in the opinion of the Site Investigator, would ultimately prevent the completion of study procedures
I haven't taken any experimental drugs recently.
Timeline
Screening
Participants are screened for eligibility to participate in the trial
2-4 weeks
Treatment
Administration of personalized antisense oligonucleotide (ASO) treatment
12 months
Follow-up
Participants are monitored for safety and effectiveness after treatment
4 weeks
Treatment Details
Interventions
- nL-CHCHD-001
Trial Overview 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.
Participant Groups
1Treatment 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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