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| Name | Class |
|---|---|
| Thomas Jefferson 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-TARDB-002 | Drug | Personalized antisense oligonucleotide |
|
| Measure | Description | Time Frame |
|---|---|---|
| Clinical Functioning | Change from baseline at 12-months post nL-TARDB-002 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-TARDB-002 administration in Slow Vital Capacity (SVC) | Baseline to 12 months |
| Clinical Functioning | Change from baseline at 12-months post nL-TARDB-002 administration in Handheld Dynamometry (HHD) | Baseline to 12 months |
| Clinical Functioning | Change from baseline at 12-months post nL-TARDB-002 administration in scores on Edinburgh Cognitive and Behavioral ALS Screen (ECAS). | Baseline to 12 months |
| Clinical Functioning | Change from baseline at 12-months post nL-TARDB-002 administration in scores on ALS Cognitive Behavioral Screen (ALS-CBS). | Baseline to 12 months |
| Clinical Functioning | Change from baseline at 12-months post nL-TARDB-002 administration in scores on Amyotrophic Lateral Sclerosis Assessment Questionnaire 5 (ALSAQ-5). | Baseline to 12 months |
| Survival | Change from baseline at 12-months post nL-TARDB-002 administration in survival status | Baseline to 12 months |
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| Measure | Description | Time Frame |
|---|---|---|
| Safety and Tolerability | Incidence and severity of adverse events | Baseline to 12 months |
| Safety and Tolerability | Emergent abnormalities in physical exam |
Inclusion Criteria:
Exclusion Criteria:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Jefferson Health | Philadelphia | Pennsylvania | 19107 | United States |
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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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| Disease Biomarkers | Change from baseline at 12-months post nL-TARDB-002 administration in serum/plasma and CSF neurofilament light chain levels | Baseline to 12 months |
| Baseline to 12 months |
| Safety and Tolerability | Emergent abnormalities in neurological exam | Baseline to 12 months |
| Safety and Tolerability | Emergent abnormalities in safety labs (CSF, chemistry, hematology, coagulation, and urinalysis) | Baseline to 12 months |
| Safety and Tolerability | Emergent abnormalities in electrocardiogram (ECG) | Baseline to 12 months |
| D019636 | Neurodegenerative Diseases |
| D057177 | TDP-43 Proteinopathies |
| D009468 | Neuromuscular Diseases |
| D057165 | Proteostasis Deficiencies |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |