GenSci144 Tablets Phase I Clinical Trial
ConditionPhenylketonuria
Trial statusRecruiting
Trial phasePhase 1
Trial typeInterventional
Biological sexAll
Age18-45
SponsorChangchun GeneScience Pharmaceutical Co., Ltd.
About this 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.
Eligibility criteria
This trial accepts healthy volunteersQualifiers
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.
Disqualifiers
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.
Trial design
Design model
Parallel
Treatments tested in this trial
GenSci144 tablets
DrugSLC6A19 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
DrugNo active ingredients, appearance, excipients, and specifications are exactly the same as GenSci144.
Treatment groups
48 Participants
are divided into 2 treatment groupsGroup A: GenSci144 single-dose group.Experimental treatment 1 intervention
Group B: Matched placebo single-dose group.Placebo comparator 1 intervention
Trial outcomes
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.Sponsors and contacts
Click on the lead sponsor to view all of their trials.
Source ClinicalTrials.gov