Personalized Antisense Oligonucleotide Therapy for A Single Participant With TARDBP ALS
NCT ID: NCT07095712
Last Updated: 2025-07-31
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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ACTIVE_NOT_RECRUITING
PHASE1/PHASE2
1 participants
INTERVENTIONAL
2024-11-25
2025-11-30
Brief Summary
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Detailed Description
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Conditions
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Study Design
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NA
SINGLE_GROUP
TREATMENT
NONE
Study Groups
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Open Label
nL-TARD-001
Personalized antisense oligonucleotide
Interventions
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nL-TARD-001
Personalized antisense oligonucleotide
Eligibility Criteria
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Inclusion Criteria
* 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
* Use of an investigational medication within less than 5 half-lives of the drug at enrollment
49 Years
49 Years
FEMALE
No
Sponsors
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The Methodist Hospital Research Institute
OTHER
Columbia University
OTHER
n-Lorem Foundation
OTHER
Responsible Party
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Locations
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Columbia University, Irving Medical Center
New York, New York, United States
Houston Methodist
Houston, Texas, United States
Countries
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Other Identifiers
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PRO00038978
Identifier Type: -
Identifier Source: org_study_id
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