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The purpose of this study is to evaluate the safety and tolerability, and pharmacokinetics of DT-216P2 administered multiple ascending doses in patients with FA.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| DT-216P2 | Experimental |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| DT-216P2 | Drug | Active |
|
| Measure | Description | Time Frame |
|---|---|---|
| Frequency of treatment-emergent adverse events (TEAE) | Up to Week 12 |
| Measure | Description | Time Frame |
|---|---|---|
| Area under the curve (AUClast, AUCinf) | Up to Week 12 | |
| Maximum concentration (Cmax) | Up to Week 12 | |
| Time to Cmax (Tmax) |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Varlli Scott | Contact | +613 8341 6228 | varlli.scott@mcri.edu.au |
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Scientia Clinical Research Ltd | Recruiting | Sydney | New South Wales | 2031 | Australia |
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| ID | Term |
|---|---|
| D005621 | Friedreich Ataxia |
| ID | Term |
|---|---|
| D013132 | Spinocerebellar Degenerations |
| D002526 | Cerebellar Diseases |
| D001927 | Brain Diseases |
| D002493 | Central Nervous System Diseases |
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| Up to Week 12 |
| Half-life (t1/2) | Up to Week 12 |
| Frataxin expression at baseline and after treatment will be measured | Up to Week 12 |
| Doherty Clinical Trials | Recruiting | East Melbourne | Victoria | 3002 | Australia |
|
| Nucleus Network | Recruiting | Melbourne | Victoria | 3004 | Australia |
|
| D009422 | Nervous System Diseases |
| D013118 | Spinal Cord Diseases |
| D020271 | Heredodegenerative Disorders, Nervous System |
| D019636 | Neurodegenerative Diseases |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D028361 | Mitochondrial Diseases |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |