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| Name | Class |
|---|---|
| The Methodist Hospital Research Institute | OTHER |
| Columbia University | OTHER |
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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.
This is an interventional study to evaluate the safety and efficacy of treatment with an individualized antisense oligonucleotide (ASO) treatment in a single participant with amyotrophic lateral sclerosis (ALS) due to a pathogenic variant in TARDBP
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Open Label | Experimental |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| nL-TARD-001 | Drug | Personalized antisense oligonucleotide |
|
| Measure | Description | Time Frame |
|---|---|---|
| Clinical Functioning | Change from baseline at 12-months post nL-TARD-001 administration in scores on Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R). | Baseline to 12 months |
| Clinical Functioning | Change from baseline at 12-months post nL-TARD-001 administration in Slow Vital Capacity (SVC) | Baseline to 12 months |
| Clinical Functioning | Change from baseline at 12-months post nL-TARD-001 administration in Edinburgh Cognitive and Behavioral ALS Screen (ECAS) score | Baseline to 12 months |
| Clinical Functioning | Change from baseline at 12-months post nL-TARD-001 administration in ALS Cognitive Behavioral Screen (ALS-CBS) score | Baseline to 12 months |
| Clinical Functioning | Change from baseline at 12-months post nL-TARD-001 administration in Amyotrophic Lateral Sclerosis Assessment Questionnaire 5 (ALSAQ-5) | Baseline to 12 months |
| Measure | Description | Time Frame |
|---|---|---|
| Disease Biomarkers | Change from baseline at 12-months post nL-TARD-001 administration in serum/plasma and CSF neurofilament light chain levels | Baseline to 12 months |
| Safety and Tolerability | Incidence and severity of adverse events |
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Inclusion Criteria:
Exclusion Criteria:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Columbia University, Irving Medical Center | New York | New York | 10032 | United States | ||
| Houston Methodist |
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| ID | Term |
|---|---|
| D000690 | Amyotrophic Lateral Sclerosis |
| ID | Term |
|---|---|
| D013118 | Spinal Cord Diseases |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
| D016472 | Motor Neuron Disease |
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| Baseline to 12 months |
| Safety and Tolerability | Emergent abnormalities in physical exam (changes in appearance, skin, neck, ears, nose, throat, heart/lungs, abdomen, lymph nodes, and extremities compared to baseline) | Baseline to 12 months |
| Safety and Tolerability | Emergent abnormalities in neurological exam (changes in mental status, gait, cerebellar, cranial nerve, motor, reflex, and sensations compared to baseline) | Baseline to 12 months |
| Safety and Tolerability | Emergent abnormalities in laboratory analyses (results outside of normal range for CSF, chemistry, hematology, coagulation, and urinalysis) | Baseline to 12 months |
| Houston |
| Texas |
| 77030 |
| United States |
| D019636 | Neurodegenerative Diseases |
| D057177 | TDP-43 Proteinopathies |
| D009468 | Neuromuscular Diseases |
| D057165 | Proteostasis Deficiencies |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |