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| ID | Type | Description | Link |
|---|---|---|---|
| TDR12768 | Other Identifier | Sanofi |
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To assess in healthy adult subjects:
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Genz-682452 | Experimental | This study will include three cohorts for doses of Genz-682452: Dose 1, Dose 2, Dose 3. Each cohort will include 12 participants, 9 of whom will be administered Genz-682452 and 3 will be administered placebo. |
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| Placebo | Placebo Comparator | Placebo comparator taken by participants randomized to the placebo arm in each of the three cohorts. Each cohort will include 12 participants, 9 of whom will be administered Genz-682452 and 3 will be administered placebo. |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Genz-682452 | Biological | Capsules for oral administration. |
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| Measure | Description | Time Frame |
|---|---|---|
| Participants with Treatment-Emergent Adverse Events (TEAEs) | Day 1 through Day 14 |
| Measure | Description | Time Frame |
|---|---|---|
| Pharmacokinetics as measure by plasma parameters | Maximum plasma concentration (Cmax), Plasma Concentration Observed Just Before Treatment Administration During Repeated Dosing (Ctrough), First time to reach Cmax (Tmax), Area Under the Plasma Concentration Versus Time Curve Over the Dosing Interval 0-23 hours (AUC0-24), Terminal Half-Life (t1/2z), Apparent Total Body Clearance of Genz-682452 at steady state (CLss/F), plasma 4 beta-hydroxycholesterol (4β-HC) |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Medical Monitor | Genzyme, a Sanofi Company | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Austin | Texas | United States |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 32851809 | Derived | Peterschmitt MJ, Crawford NPS, Gaemers SJM, Ji AJ, Sharma J, Pham TT. Pharmacokinetics, Pharmacodynamics, Safety, and Tolerability of Oral Venglustat in Healthy Volunteers. Clin Pharmacol Drug Dev. 2021 Jan;10(1):86-98. doi: 10.1002/cpdd.865. Epub 2020 Aug 26. |
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| ID | Term |
|---|---|
| C000608118 | venglustat |
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| Placebo |
| Biological |
Placebo capsules matching the Genz-682452 capsules. |
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| Day 1 through Day 14 |
| Pharmacodynamics as measure by Plasma concentrations of Glucosylceramide (GL-1), Globotriaosylceramide (GL-3) and GM3 ganglioside (GM3) | Day 1, through Day 14 |
| Pharmacokinetics Urine parameter as measure by assessment of Genz-682452 in Urine, Ae0-t, fe0-t (Exploratory) | Day 1 through Day 14 |