GenSci144 Tablets Phase I Clinical Trial

NCT07685210 | Recruiting Phase 1

• 1 other identifier: GenSci144-101
• Study Type: interventional
• Target: 48
• Locations: 1 country
• Sites: 1

Brief 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.

Conditions

Phenylketonuria

Outcome Measures

Primary Outcomes (2)

• 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 (1)

• AUC0-t, AUC0-∞, Cmax, Tmax, t1/2, CL/F, Vz/F.

  From before dosing to 48 hours after dosing.

Other Outcomes (4)

• 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.

Study Arms (2)

GenSci144 single-dose group.

EXPERIMENTAL

Drug: GenSci144 tablets

Matched placebo single-dose group.

PLACEBO COMPARATOR

Drug: GenSci144 placebo tablets

Interventions

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 single-dose group.

GenSci144 placebo tablets DRUG

No active ingredients, appearance, excipients, and specifications are exactly the same as GenSci144.

Matched placebo single-dose group.

Eligibility Criteria

• Age: 18 Years - 45 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Changchun GeneScience Pharmaceutical Co., Ltd.: lead

Study Sites (1)

The First Affiliated Hospital of Shandong First Medical University (Shandong Qianfoshan Hospital).

Jinan, Shandong, 250014, China

RECRUITING

MeSH Terms

Conditions

Phenylketonurias

Condition Hierarchy (Ancestors)

Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Amino Acid Metabolism, Inborn Errors; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Metabolic Diseases; Nutritional and Metabolic Diseases

Central Study Contacts

Wei Zhao

CONTACT

+86 15131190710; zhao4wei2@hotmail.com

Yanlin Chen

CONTACT

+86 18621909391; chenyanlin01@genscigroup.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: OTHER
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 23, 2026
• First Posted: July 6, 2026
• Study Start: June 22, 2026
• Primary Completion (Estimated): September 1, 2026
• Study Completion (Estimated): December 1, 2026
• Last Updated: July 6, 2026

Record last verified: 2026-06

Locations

CN (1)