Phenylketonuria
Conditions
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.
Interventions
DRUGGenSci144 tablets
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.
DRUGGenSci144 placebo tablets
No active ingredients, appearance, excipients, and specifications are exactly the same as GenSci144.
Sponsors
Changchun GeneScience Pharmaceutical Co., Ltd.
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
OTHER
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
18 Years to 45 Years
Healthy volunteers
Yes
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.
Design outcomes
Primary
| Measure | 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. |
Secondary
| Measure | Time frame |
|---|---|
| AUC0-t, AUC0-∞, Cmax, Tmax, t1/2, CL/F, Vz/F. | From before dosing to 48 hours after dosing. |
Countries
China
Contacts
CONTACTWei Zhao
CONTACTYanlin Chen
Outcome results
None listed