ClinicalTrials.gov record NCT07318909

To Evaluate the Safety and Efficacy of GS1168 Injection in Adult Phenylketonuria

Record copied from ClinicalTrials.gov: Oct 10, 2026AI summaries generated: Sep 6, 2026

A Clinical Exploration Study of Adeno-Associated Virus Vector Expressing Human Phenylalanine Hydroxylase Gene in Adult Classical Phenylketonuria

StatusNot yet recruitingThe study has been registered but has not started enrolling. Sites and contacts may not be listed yet.
Study typeInterventionalA study in which researchers assign participants to one or more interventions, such as a drug, procedure, device, behavioural programme or other approach, to measure their effects.
PhaseEarly phase 1A very early study, usually in a small number of people, to learn how the body handles a treatment. It is not designed to show whether the treatment works.
Age18 years old to 55 years old
SexAll
LocationChina
AI summaryWhat this study is about, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

This is a single-arm, open-label, dose‑escalation study that gives one intravenous dose of GS1168 and follows participants for safety and other measures for up to 52 weeks. It is for adults (18–55) with classical PKU caused by PAH mutations whose condition is judged inadequately controlled with current therapy. The study will test three escalating dose levels of GS1168 given as a single infusion.

What it is studying. The study tests the safety, tolerability, and effects of a single intravenous GS1168 administration — an adeno-associated virus (AAV) gene therapy that delivers the PAH gene — on blood phenylalanine levels, total protein intake, and PKU-related quality of life in adults with PKU due to PAH mutations.

What the researchers hope to learn. To evaluate the safety and tolerability of a single GS1168 infusion and to measure its effects on plasma phenylalanine, total protein intake, and PKU quality-of-life scores.

What this study is about

The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.

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.

Interventions being studied

Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.

Drug
  • GS1168 injection

Eligibility

The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.

Inclusion criteria

  • 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.

Exclusion criteria

  • 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
  • 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.

Study description and what participation involves

The registry record has no detailed description beyond the summary above.

AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

After screening (including blood tests for liver/kidney function, blood counts, viral and antibody tests such as anti‑AAV8), eligible participants receive a single intravenous infusion of GS1168 at one of three dose levels. They will be followed and monitored for safety up to 52 weeks with repeated blood tests (including plasma phenylalanine), recording of protein intake, and completion of PKU quality‑of‑life questionnaires. Participants must avoid alcohol from 30 days before the infusion until 52 weeks after and follow contraception and breastfeeding restrictions. The study team will assess adverse events and laboratory results throughout the follow‑up period.

How the study is structured. Interventional, early-phase study with a single treatment group and no masking. Participants receive one IV infusion of GS1168 at one of three escalating dose levels and are followed (including safety monitoring and lab tests) through week 52.

Questions you could ask

These questions are suggestions for a conversation with the study team or your own clinician, not advice.

  • This is an early-phase study, mainly about safety and dose. What is already known about this treatment?
  • The registry says recruitment has not started. When is it expected to open, and can I register interest now?
  • The listed sites are in China. Is any of them reachable for me, and does the study help with travel?
  • Which of the eligibility criteria would need to be checked with tests before I could join?

More on phenylketonuria

Clinical trials38 trials listed for phenylketonuriaEvery trial the registry lists for phenylketonuria, with recruitment status, phase and sites, updated from ClinicalTrials.gov.See all trials →Research updatesWhat changed in phenylketonuria researchLatest monthly update: September 2026. Includes: Diet supply gaps. New research summarized each month in plain language, with links to every source paper.Read the latest update →

Important dates

Study start
Dec 31, 2027 (estimated)
Primary completion
Dec 31, 2028 (estimated)
Study completion
Dec 31, 2032 (estimated)
Target enrollment
9 participants (estimated)
Last updated on registry
Sep 10, 2026
Data verified
Oct 10, 2026

Sponsor and research team

The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.

Lead sponsor
Gritgen Therapeutics Co., Ltd.

Central study contact

If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.

Participating sites (1)

Terms used on this page · explanations by MedicalUpdates.ai

single-arm
All participants receive the same treatment or follow the same plan. There is no comparison group within the study.
open-label
No masking is used, so the assigned intervention is not deliberately hidden from participants or study staff.
dose-escalation
Groups of participants receive increasing doses of a treatment, one group after another, to find a dose that can be given safely.
Informed Consent
The process of explaining a study, including its risks and what participation involves, so that a person can decide freely whether to take part. Consent is usually documented in writing, and a participant can choose to leave the study later.
baseline
Measurements taken at the start of the study, before any study treatment, so that later results can be compared with them.
childbearing potential
Studies often ask participants who could become pregnant, or could father a child, to use contraception during the study and for a period afterwards, because the effect of the treatment on a pregnancy is unknown.
at screening
The checks done before enrolment to see whether a person qualifies, such as tests, scans or a review of medical history.
Upper Limit of Normal
The highest value of a blood test that a laboratory considers normal. Criteria such as '≤ 3 × ULN' mean the result must be no more than three times that value.
Neutrophil count
The number of neutrophils, a type of white blood cell that fights infection, in a blood sample. Studies often require it to be above a set level.
Inclusion Criteria
The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
Exclusion Criteria
The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.

The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.

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