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The purpose of this study is to characterize the safety, tolerability, and pharmacokinetics of EDK060 as compared to placebo in adult patients with CMT1A.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| EDK060 | Experimental | Doses A; B; C; D |
|
| Placebo | Placebo Comparator | Matching placebo |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| EDK060, dose A | Drug | EDK060, dose A, Single dose, IV infusion |
| |
| Measure | Description | Time Frame |
|---|---|---|
| Incidence and severity of Adverse Events (AEs) and Serious Adverse Events (SAEs) | Incidence of AEs and SAEs by treatment group, including changes in vital signs, laboratory measures, neurological examinations and electrophysiology measures qualifying and reported as AEs. | From baseline up to day 140 |
| Measure | Description | Time Frame |
|---|---|---|
| EDK060 Plasma PK parameters - Cmax | The maximum (peak) observed EDK060 plasma concentration after single dose administration (mass x volume-1) | From baseline up to day 140 |
| EDK060 Plasma PK parameters - Tmax |
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Inclusion Criteria:
Exclusion Criteria
Other protocol-defined inclusion/exclusion criteria may apply
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Novartis Pharmaceuticals | Contact | 1-888-669-6682 | novartis.email@novartis.com | |
| Novartis Pharmaceuticals | Contact | +41613241111 |
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| The Ottawa Hospital | Recruiting | Ottawa | Ontario | K1H 8L6 | Canada |
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| EDK060, dose B |
| Drug |
EDK060, dose B, Single dose, IV infusion |
|
| EDK060, dose C | Drug | EDK060, dose C, Single dose, IV infusion |
|
| EDK060, dose D | Drug | EDK060, dose D, Single dose, IV infusion |
|
| Placebo | Other | Placebo |
|
The time to reach maximum (peak) plasma, EDK060 concentration after single dose administration (time)
| From baseline up to day 140 |
| EDK060 Plasma PK parameters - AUClast | The area under the concentration-time curve of EDK060 from time zero to the last measurable concentration sampling time (tlast) (mass x time x volume-1) | From baseline up to day 140 |
| Montreal Neurological Institute | Recruiting | Montreal | Quebec | H3A 2B4 | Canada |
|
| CIUSS de l´Estrie-CHUS- Hôpital Fleurimont | Recruiting | Sherbrooke | Quebec | J1H 5N4 | Canada |
|
| ID | Term |
|---|---|
| D002607 | Charcot-Marie-Tooth Disease |
| ID | Term |
|---|---|
| D015417 | Hereditary Sensory and Motor Neuropathy |
| D009421 | Nervous System Malformations |
| D009422 | Nervous System Diseases |
| D020271 | Heredodegenerative Disorders, Nervous System |
| D019636 | Neurodegenerative Diseases |
| D011115 | Polyneuropathies |
| D010523 | Peripheral Nervous System Diseases |
| D009468 | Neuromuscular Diseases |
| D000013 | Congenital Abnormalities |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D030342 | Genetic Diseases, Inborn |
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