PhenylketonuriaPhase 1NCT07685210
GenSci144 Tablets Phase I Clinical Trial
A Phase 1 study testing GenSci144 tablets in people with Phenylketonuria, run by Changchun GeneScience Pharmaceutical Co., Ltd. at 1 site in China.
Trial 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… Read more about this study
- Who is it for?
- Ages 18 to 45 yearsHealthy volunteers can take part
- Conditions the study is for
- Phenylketonuria
- What is being tested?
- GenSci144 tablets
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 48 participants
- Where
- 1 siteChina
- Study dates
- 22 Jun 2026to Dec 2026
- Phase
- Phase 1
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 45 years
- All sexes
- Healthy volunteers accepted
- Adult
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site, 1 recruiting
- Recruiting
The First Affiliated Hospital of Shandong First Medical University (Shandong Qianfoshan Hospital).
Jinan, Shandong, 250014
Wei Zhao, +86 15131190710, zhao4wei2@hotmail.com
About this study
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
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
- Being tested
- GenSci144 tablets, GenSci144 placebo tablets
- Medical subject headings
- Phenylketonurias
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
GenSci144 tabletsDrug
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 tabletsDrug
No active ingredients, appearance, excipients, and specifications are exactly the same as GenSci144.
| Study group | Role | What they receive |
|---|---|---|
| GenSci144 single-dose group. | Experimental | Not described Receives: Drug: GenSci144 tablets |
| Matched placebo single-dose group. | Placebo comparator | Not described Receives: Drug: GenSci144 placebo tablets |
What is being measured
The primary outcome is the main question the study is designed to answer.
Adverse events and clinical safety indicators after a single dose.
Measured: Sign informed consent until Day3 discharge.
Change in QT/QTc interval correction ΔΔQTc after dosing.
Measured: Dynamic ECG monitoring from before to 24 hours after medication.
Secondary outcomes (1)
AUC0-t, AUC0-∞, Cmax, Tmax, t1/2, CL/F, Vz/F.
Measured: From before dosing to 48 hours after dosing.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Jul 2026
- Study start22 Jun 2026
- Primary completionSep 2026 (estimated)
- Study completionDec 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for PhenylketonuriaStudies of the same condition, open and under way.
- More trials run by Changchun GeneScience Pharmaceutical Co., Ltd.The sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Other
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 48 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Changchun GeneScience Pharmaceutical Co., Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07685210
- Sponsor's protocol ID
- GenSci144-101
- Status last verified
- Jun 2026
- First submitted
- 23 Jun 2026
- Last updated
- 6 Jul 2026
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 6 Jul 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT07685210
- Acronym / sponsor ID
- GenSci144-101
- Conditions
- Phenylketonuria
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 48 participants (estimated)
- Lead sponsor
- Changchun GeneScience Pharmaceutical Co., Ltd.Industry (pharma, biotech, devices)
- Study start
- 22 Jun 2026 (actual)
- Primary completion
- Sep 2026 (estimated)
- Study completion
- Dec 2026 (estimated)
- First posted
- 6 Jul 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Other
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| GenSci144 single-dose group. | Experimental | Drug: GenSci144 tablets |
| Matched placebo single-dose group. | Placebo comparator | Drug: GenSci144 placebo tablets |
Who may qualify
- Ages
- 18 to 45 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult
Key inclusion criteria (4 listed)
- 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.
Key exclusion criteria (6 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| GenSci144 tabletsSLC6A19 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. | Drug | GenSci144 single-dose group. |
| GenSci144 placebo tabletsNo active ingredients, appearance, excipients, and specifications are exactly the same as GenSci144. | Drug | Matched placebo single-dose group. |
Primary outcome
Adverse events and clinical safety indicators after a single dose.
Time frame: Sign informed consent until Day3 discharge.
Change in QT/QTc interval correction ΔΔQTc after dosing.
Time frame: Dynamic ECG monitoring from before to 24 hours after medication.
1 secondary outcome
AUC0-t, AUC0-∞, Cmax, Tmax, t1/2, CL/F, Vz/F.
Time frame: From before dosing to 48 hours after dosing.
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 Jul 2026.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing GenSci144 tablets in people with Phenylketonuria, run by Changchun GeneScience Pharmaceutical Co., Ltd. at 1 site in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 6 Jul 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Wei Zhao (+86 15131190710, zhao4wei2@hotmail.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Adverse events and clinical safety indicators after a single dose. (time frame: Sign informed consent until Day3 discharge.). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 6 Jul 2026; AzTrials last refreshed its copy on 26 Oct 2026.