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| Name | Class |
|---|---|
| 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 CHCHD10
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Open Label | Experimental |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| nL-CHCHD-001 | Drug | Personalized Antisense Oligonucleotide |
|
| Measure | Description | Time Frame |
|---|---|---|
| Clinical Functioning | Change from baseline at 12-months post nL-CHCHD-001 administration in scores on Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R). A maximum score of 48 represents normal functioning, and the minimum score is 0. | Baseline to 12 months |
| Motor Functioning | Change from baseline at 12-months post nL-CHCHD-001 administration in scores on Slow Vital Capacity (SVC) Performance. | Baseline to 12 months |
| Motor Functioning | Change from baseline at 12-months post nL-CHCHD-001 administration in scores on Handheld Dynamometry (HHD). | Baseline to 12 months |
| Clinical Functioning | Change from baseline at 12-months post nL-CHCHD-001 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-CHCHD-001 administration in scores on ALS Cognitive Behavioral Screen (ALS-CBS) | Baseline to 12 months |
| Clinical Functioning | Change from baseline at 12-months post nL-CHCHD-001 administration in scores on 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-CHCHD-001 administration in serum and cerebrospinal fluid neurofilament light chain levels | Baseline to 12 months |
| Safety and Tolerability |
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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 |
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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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Incidence and Severity of Adverse Events
| Baseline to 12 months |
| Safety and Tolerability | Emergent abnormalities in physical exam | Baseline to 12 months |
| Safety and Efficacy | Emergent abnormalities in neurological exam | Baseline to 12 months |
| Safety and Efficacy | Emergent abnormalities in safety labs (CSF, chemistry, hematology, coagulation, and urinalysis) | 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 |