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NCT Number: NCT07685210

GenSci144 Tablets Phase I Clinical Trial

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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Key information

Conditions

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

Age range

18 year–45 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1

Primary location

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

Jinan, Shandong, 250014, China

Location status: Recruiting

Location contact

Wei Zhao

CONTACT

[email protected]

+86 15131190710

Who can participate

Healthy volunteers accepted: Yes

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

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.

Primary outcomes

  1. Adverse events and clinical safety indicators after a single dose.

    Time frame: Sign informed consent until Day3 discharge.

  2. Change in QT/QTc interval correction ΔΔQTc after dosing.

    Time frame: Dynamic ECG monitoring from before to 24 hours after medication.

Secondary outcomes

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

    Time frame: From before dosing to 48 hours after dosing.

Other outcomes

  1. Urinary Hartnup amino acids, urinary phenylalanine and urinary glutamine.

    Time frame: 24 hours before administration, 0-8 hours after administration, 8-24 hours after administration.

  2. Plasma phenylalanine levels and plasma amino acid levels.

    Time frame: Day-1 baseline, 1 hours, 2 hours, 3 hours, 4 hours, 24 hours after dosing.

  3. Urine sodium and potassium levels.

    Time frame: 0-8 hours and 8-24 hours after dosing.

  4. urine output.

    Time frame: - 24 to -16 hours, -16 to 0 hours before dosing, and 0-8 hours, 8-24 hours after dosing.

Study contacts

Contact information is provided by the study sponsor or research team.

Wei Zhao

CONTACT

[email protected]

+86 15131190710

Yanlin Chen

CONTACT

[email protected]

+86 18621909391

Sponsors and collaborators

Lead sponsor

Changchun GeneScience Pharmaceutical Co., Ltd.

Industry

Registry information

Official study title

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

Important dates

Study start
2026
Primary completion
2026
Study completion
2026
First posted
Jul 6, 2026
Registry last updated
Jul 6, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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