Phenylketonuria (PKU)Early Phase 1NCT07318909
To Evaluate the Safety and Efficacy of GS1168 Injection in Adult Phenylketonuria
A Early Phase 1 study testing GS1168 injection in people with Phenylketonuria (PKU), run by Gritgen Therapeutics Co., Ltd. at 1 site in China.
Trial summary
This study is a single-arm, open-label, dose-escalation, exploratory study to evaluate the safety, tolerability, and efficacy of a single administration of GS1168 Injection in Chinese adult phenylketonuria (PKU) with PAH mutation. Read more about this study
- Who is it for?
- Ages 18 to 55 years
- Conditions the study is for
- Phenylketonuria (PKU)
- What is being tested?
- GS1168 injection
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 9 participants
- Where
- 1 siteChina
- Study dates
- 31 Dec 2027to 31 Dec 2032
- Phase
- Early Phase 1
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 55 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- Understand the purpose and risks of the study and voluntarily sign Informed Consent Forms (ICF).
- Male or female, aged 18-55 years.
- Diagnosis of PKU due to PAH mutation, and be inadequately controlled with current therapy (e.g., dietary management) in the judgement of the investigator.
- Intolerant of unresponsive to available treatment such as Kuvan or Palynziq, and have come off the medication for at least 28 days prior to signing the ICF.
- Ability and willingness to maintain dietary protein intake consistent with baseline
- Male and female subjects of childbearing potential must agree to use effective contraception methods from signing ICF until at least 52 weeks after GS1168 infusion. Female subjects must not be breastfeeding.
- Agree to avoid alcohol consumption from 30 days prior to GS1168 infusion until at least 52 weeks after infusion.
- Ability to communicate well with the Investigator and to understand and comply with the requirements of the study.
Who cannot join
- Diagnosis of PKU due to other gene mutations.
- Body weight ≥100 kg or Body Mass Index (BMI) ≥30 kg/m² at screening.
- Presence of anti-AAV8 neutralizing antibody.
- Abnormal liver function tests at screening:
- ALT or AST >1.25 × Upper Limit of Normal (ULN)
- GGT >1.25 × ULN
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- Total Bilirubin >1.5 × ULN
- Abnormal laboratory findings at screening:
- Hemoglobin <9 g/dL
- Neutrophil count <1.0 × 10⁹/L
- Platelet count <100 × 10⁹/L
- Serum Creatinine ≥1.5 mg/dL (133 μmol/L)
- Glycated Hemoglobin (HbA1c) >6% or Fasting Blood Glucose >6.1 mmol/L
- Active or occult hepatitis B virus (HBV) infection, active hepatitis C virus (HCV) infection, positive Human Immunodeficiency Virus (HIV) antibodies and positive syphilis test.
- Any severe conditions of respiratory, cardiovascular, gastrointestinal, renal, neurological, endocrine, immunological, hematological, psychiatric, etc.
- History of malignancy.
- Any other condition that, in the opinion of the Investigator, may affect subject compliance or render the subject unsuitable for participation in this trial.
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
The First Affiliated Hospital of Anhui Medical University
Hefei, Anhui, 230022
Huan Zhou, PhD, +8613665527160, zhouhuanbest@vip.163.com
Datong Deng, MD, +8613855134251, 13855134251@163.com
About this study
Official title: A Clinical Exploration Study of Adeno-Associated Virus Vector Expressing Human Phenylalanine Hydroxylase Gene in Adult Classical Phenylketonuria
This study is a single-arm, open-label, dose-escalation, exploratory study to evaluate the safety, tolerability, and efficacy of a single administration of GS1168 Injection in Chinese adult phenylketonuria (PKU) with PAH mutation.
- Conditions
- Phenylketonuria (PKU)
- Being tested
- GS1168 injection
- Medical subject headings
- Phenylketonurias
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
GS1168 injectionDrug
The dose levels are as follows:
- A single intravenous administration of GS1168 injection at dose level 1;
- A single intravenous administration of GS1168 injection at dose level 2;
- A single intravenous administration of GS1168 injection at dose level 3.
| Study group | Role | What they receive |
|---|---|---|
| Treatment group Arm of GS1168 | Experimental | Dose level 1, level 2 and level 3 will be administered Receives: Drug: GS1168 injection |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of adverse event (AE), including serious adverse event (SAE) and adverse event of special interest (AESI)
Incidence of adverse event (AE), including serious adverse event (SAE) and adverse event of special interest (AESI) as assessed by CTCAE V5.0, from baseline to week 52
Measured: Week 52
Secondary outcomes (3)
Plasma Phe levels
Change from baseline in plasma level of Phe following GS1168 injection administration
Measured: Week 52
Total protein intake
Change from baseline in total protein intake following GS1168 injection administration
Measured: Week 52
Phenylketonuria Quality of Life (PKU-QOL) scores
Change in Phenylketonuria Quality of Life Questionnaire (PKU-QOL, Score range: 0-100, higher scores indicate worse outcomes.)
Measured: Week 52
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Jan 2026
- Study start31 Dec 2027 (estimated)
- Primary completion31 Dec 2028 (estimated)
- Study completion31 Dec 2032 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Phenylketonuria (PKU)Studies of the same condition, open and under way.
- 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
- Early Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 9 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Gritgen Therapeutics Co., Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07318909
- Sponsor's protocol ID
- GS1168-201
- Status last verified
- Sep 2026
- First submitted
- 1 Dec 2025
- Last updated
- 10 Sep 2026
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No