GenSci144 tablets early-dose study for phenylketonuria treatment
Official 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
Run by Changchun GeneScience Pharmaceutical Co., Ltd. · NCT07685210 · Data updated 2026-11-12
This early-phase trial tests a single dose of GenSci144 tablets in healthy adults to see if it is safe and tolerable, while also looking at how the body processes the drug.
Quick facts
| Phase | Phase 1 |
|---|---|
| Study type | Interventional |
| Ages | 18 Years to 45 Years |
| Sex | All |
| Enrollment | 48 (estimated) |
| Sponsor | Changchun GeneScience Pharmaceutical Co., Ltd. Industry-sponsored |
| Locations | 1 site (Jinan, Shandong) |
| Status | Recruiting |
| Started | June 22, 2026 |
| Main results expected | September 2026 |
| Study ends | December 2026 |
| Registry record updated | July 6, 2026 |
| Trial ID | NCT07685210 on ClinicalTrials.gov |
Is this trial a fit for you?
✓Likely a good fit
Healthy men and women aged 18-45 with BMI 19-26 kg/m² who meet consent and contraception requirements.
✕Probably not a fit
Patients with phenylketonuria or other medical conditions may not directly benefit from this Phase I study.
A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.
What this trial studies
This is a Phase I study that uses a randomized, double-blind, placebo-controlled design to assess safety and tolerability of a single ascending dose of GenSci144 tablets. The trial also gathers pharmacokinetic and pharmacodynamic data to understand how the drug behaves in the body. Participants are healthy adults aged 18-45, and the study aims to inform future development for phenylketonuria. Results from similar early trials with related approaches have informed dose selection and safety monitoring in subsequent phases.
Why it matters
Potential benefit: If successful, the study could support future development of GenSci144 as a treatment option for phenylketonuria.
How similar studies have gone: Early-phase, placebo-controlled trials of similar compounds have demonstrated acceptable safety and tolerability in healthy volunteers, guiding subsequent trials.
Who can join: the full rules
Show full inclusion / exclusion criteria
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.
Where it's running
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The First Affiliated Hospital of Shandong First Medical University (Shandong Qianfoshan Hospital).
Jinan, Shandong, China Recruiting
Study contacts
- Study coordinator: Wei Zhao
- Email: zhao4wei2@hotmail.com
- Phone: +86 15131190710
Similar studies
Other studies for the same condition or treatment. Recruiting studies are listed first.
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Impact of a Phenylalanine-Restricted Diet on Gut Health in Children with PKU
This study looks at how a Phe-restricted diet, with or without sapropterin or sepiapterin, might affect gut health in kids with PKU.
-
How Repinatrabit is processed in adults with liver or kidney impairment versus normal function
This study tests how Repinatrabit is handled by the body in adults with moderate liver impairment or severe kidney impairment compared to adults with normal liver function.
-
Eating disorders in people with phenylketonuria
This study looks at whether eating disorders and BMI issues are more common in people with phenylketonuria who are followed at a French hospital.
-
Immune Modulation During Palynziq Treatment in Adults
This study tests whether adding low-dose methotrexate can help people with PKU tolerate Palynziq better and achieve better blood levels of phenylalanine.
-
AG-181 safety and tolerability in people with phenylketonuria
This trial will test AG-181 for safety and tolerability in people with PKU who have high blood phenylalanine and at least one R408W PAH mutation.
-
JNT-517 treatment for adults with phenylketonuria (PKU)
This study will test whether taking JNT-517 pills twice daily lowers high blood phenylalanine and is safe for adults with PKU.
How to take part
- Review the eligibility criteria above with your treating physician.
- Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
- Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.