← Back to all trials
Enrolling By Invitation
NCT07703462
Personalized Antisense Oligonucleotide Therapy for a Participant With TARDBP ALS
Conditions: Amyotrophic Lateral Sclerosis (ALS)
Sex: All
Healthy volunteers: No
Phase: PHASE1, PHASE2
Enrollment: 1
Sponsor: n-Lorem Foundation
Location: Jefferson Health Philadelphia Pennsylvania
Summary
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 TARDBP.
Eligibility Criteria
Inclusion Criteria:
* Informed consent/assent provided by the participant (when appropriate), and/or participants 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
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
Source: ClinicalTrials.gov (NCT07703462). StuddyBuddy aggregates publicly available trial information.
