GenSci144 Tablets Phase I Clinical Trial

NCT07685210 — Phase 1, Recruiting, 48 participants, Changchun GeneScience Pharmaceutical Co., Ltd.

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A Single-center, Randomized, Double-blind, Placebo-controlled Phase I Clinical Trial in Healthy Adult Participants in China to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Single Ascending Oral Doses of GenSci144

Registry	NCT07685210
Status	Recruiting
Phase	Phase 1
Study type	Interventional; randomized, parallel, masking: quadruple, other
Enrollment	48 (Estimated)
Sponsor	Changchun GeneScience Pharmaceutical Co., Ltd. (Industry)
Conditions	Phenylketonuria
Interventions	GenSci144 tablets, GenSci144 placebo tablets (Drug)
Dates	start 2026-06-22, primary completion 2026-09-01 (estimated), completion 2026-12-01 (estimated), first posted 2026-07-06, last updated 2026-07-06
Locations	1 site in China

-Summary

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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+Arms and interventions

Arm	Type	Interventions	Description
GenSci144 single-dose group.	Experimental	Drug: GenSci144 tablets
Matched placebo single-dose group.	Placebo comparator	Drug: GenSci144 placebo tablets

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.
GenSci144 placebo tablets (Drug): No active ingredients, appearance, excipients, and specifications are exactly the same as GenSci144.

+Outcome measures

Primary outcomes

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.]

Secondary outcomes

AUC0-t, AUC0-∞, Cmax, Tmax, t1/2, CL/F, Vz/F. [From before dosing to 48 hours after dosing.]

Other outcomes

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.]
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.]

+Eligibility

Sex: All, ages: 18 Years to 45 Years, accepts healthy volunteers

Inclusion Criteria:

* Healthy men and women aged 18 to 45, with at least 3 people of the same gender in each group.
* BMI 19\~26 kg/m², men weigh ≥50 kg, women ≥45 kg.
* Voluntarily sign the informed consent form, fully aware of the research risks and procedures.
* Use reliable contraception for 3 months after the last dose, no plans for pregnancy or sperm/egg donation.

Exclusion Criteria:

* A family history of sudden death and serious neuropsychiatric disorders.
* Major underlying conditions like gastrointestinal absorption problems, chronic liver, kidney, heart, and endocrine diseases.
* Liver function, kidney function with eGFR<90, abnormal QTc interval, positive for various infectious diseases.
* Drug allergies, drug abuse, recent participation in other clinical trials.
* Drinking alcohol, smoking, taking grapefruit/amino acid supplements, and having a chaotic routine during the screening period.
* Pregnant or breastfeeding women, people who can't tolerate blood draws or get faint from needles.

+Locations (1)

Facility	City	Country	Status
The First Affiliated Hospital of Shandong First Medical University (Shandong Qianfoshan Hospital).	Jinan, Shandong	China	Recruiting