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This study is a Phase I single ascending dose clinical trial in which healthy adult volunteers were administered single oral doses of GenSci144 tablets at different levels. It was conducted using a randomized, double-blind, placebo-controlled design, primarily to evaluate the drug's safety and tolerability, while also exploring its pharmacokinetic and pharmacodynamic characteristics in the body, providing a basis for subsequent clinical use in the treatment of phenylketonuria.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| GenSci144 single-dose group. | Experimental |
| |
| Matched placebo single-dose group. | Placebo Comparator |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| GenSci144 tablets | Drug | SLC6A19 transporter small molecule inhibitor, oral tablets in 25mg and 75mg, given as a single dose on an empty stomach, used to explore treatment for phenylketonuria. |
| Measure | Description | Time Frame |
|---|---|---|
| Adverse events and clinical safety indicators after a single dose. | Sign informed consent until Day3 discharge. | |
| Change in QT/QTc interval correction ΔΔQTc after dosing. | Dynamic ECG monitoring from before to 24 hours after medication. |
| Measure | Description | Time Frame |
|---|---|---|
| AUC0-t, AUC0-∞, Cmax, Tmax, t1/2, CL/F, Vz/F. | From before dosing to 48 hours after dosing. |
| Measure | Description | Time Frame |
|---|---|---|
| Urinary Hartnup amino acids, urinary phenylalanine and urinary glutamine. | 24 hours before administration, 0-8 hours after administration, 8-24 hours after administration. | |
| Plasma phenylalanine levels and plasma amino acid levels. | Day-1 baseline, 1 hours, 2 hours, 3 hours, 4 hours, 24 hours after dosing. |
Inclusion Criteria:
Exclusion Criteria:
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Wei Zhao | Contact | +86 15131190710 | zhao4wei2@hotmail.com | |
| Yanlin Chen | Contact | +86 18621909391 | chenyanlin01@genscigroup.com |
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| The First Affiliated Hospital of Shandong First Medical University (Shandong Qianfoshan Hospital). | Recruiting | Jinan | Shandong | 250014 | China |
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| ID | Term |
|---|---|
| D010661 | Phenylketonurias |
| ID | Term |
|---|---|
| D020739 | Brain Diseases, Metabolic, Inborn |
| D001928 | Brain Diseases, Metabolic |
| D001927 | Brain Diseases |
| D002493 | Central Nervous System Diseases |
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| GenSci144 placebo tablets | Drug | No active ingredients, appearance, excipients, and specifications are exactly the same as GenSci144. |
|
| Urine sodium and potassium levels. | 0-8 hours and 8-24 hours after dosing. |
| urine output. | - 24 to -16 hours, -16 to 0 hours before dosing, and 0-8 hours, 8-24 hours after dosing. |
| D009422 | Nervous System Diseases |
| D000592 | Amino Acid Metabolism, Inborn Errors |
| D008661 | Metabolism, Inborn Errors |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |