ClinicalTrials.gov record NCT06687733

Safety and Efficacy Study of NGGT002 in Adult Patients With Phenylketonuria

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

A Phase I/II Study for the Safety and Efficacy of Intravenous Infusion With NGGT002 in Adults Patients With Phenylketonuria

StatusRecruitingThe study is currently enrolling participants. Only the study team can say whether a particular person can join.
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.
PhasePhase 1/2A study that combines safety and dose-finding (phase 1) with a first look at whether the treatment has an effect (phase 2).
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 Phase 1/2, open-label study gives a single intravenous dose of NGGT002 (a rAAV8 vector carrying the human PAH gene) to adults with classic phenylketonuria. The study uses dose escalation across three cohorts followed by cohort expansion. Participants are followed for safety and efficacy assessments for up to 5 years.

What it is studying. The study is testing the safety of a single IV administration of NGGT002 gene therapy and its effects on blood phenylalanine (Phe) levels in adults with classic phenylketonuria.

What the researchers hope to learn. To evaluate the incidence and severity of adverse events after a single NGGT002 dose and to measure changes from baseline in mean plasma phenylalanine at Weeks 12, 28, and 52. Secondary goals include the proportion of participants reaching predefined Phe targets, time to and duration of Phe target achievement, changes in daily Phe and natural protein intake, and changes in 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 is a Phase 1/2, open-label, multiple-center, dose escalation and cohort expansion study to evaluate the safety and efficacy of NGGT002 in adult subjects with classic Phenylketonuria (PKU). NGGT002 is a rAAV8 based vector carrying a functional copy of the human PAH gene. Participants will receive a single administration of NGGT002 and will be followed for safety and efficacy for 5 years.

Interventions being studied

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

Genetic
  • NGGT002

Eligibility

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

Inclusion criteria

  • Voluntarily participating in the study and signing the informed consent form;
  • Gender is not limited; patients must carry biallelic pathogenic or likely pathogenic variants in the PAH gene;
  • Adult patients aged 18 to 55 years;
  • In the past 24 months, at least two blood Phe concentrations have been ≥600 μmol/L (10 mg/dL), with at least one of these measurements taken within 6 months prior to the screening period;
  • Willing and able to manage their diet;
  • According to the investigator's opinion, willing and able to comply with the study procedures and requirements;
  • Women of childbearing potential must have a negative serum HCG test within 7 days before dosing. Participants must agree to use highly effective contraceptive measures for at least one year after receiving NGGT002.

Exclusion criteria

  • Presence of anti-AAV8 neutralizing antibodies（≥1:5）
  • Participants whose disease is well-controlled with existing therapies, such as those currently receiving medications like Sapropterin Dihydrochloride tablets, Pegvaliase-pqpz, etc. (excluding participants who have previously received the above-mentioned drug therapies but did not respond, and have been off treatment for more than 6 weeks);
  • Before dosing, the patient's hematological laboratory tests exceed any of the following limits:
  • Alanine Transaminase (ALT) > 1.5×ULN and/or Aspartate Aminotransferase (AST) > 1.5×ULN
  • Alkaline Phosphatase (ALP) > 1.5×ULN
  • Total Bilirubin (TBil) > 1.5×ULN, Direct Bilirubin > 1.5×ULN
  • International Normalized Ratio (INR) > 1.5
  • Serum Creatinine (Scr) > 1.5×ULN
  • Hematological values outside the normal range (Hemoglobin: <110 g/L for males, <100 g/L for females, White Blood Cells <3.0×10\^9/L, Neutrophils <1.5×10\^9/L, Platelets <100×10\^9/L)
  • Glycated Hemoglobin (HbA1c) > 6% or Fasting Blood Glucose > 6.1 mmol/L
  • At screening, clinically significant abnormal vital signs, physical examination, laboratory test results, or other relevant findings that, in the investigator's opinion, make the subject unsuitable for inclusion;
  • In the investigator's assessment, the participant has contraindications to corticosteroid use or conditions that could lead to a worsening of the condition;
  • Hepatitis A virus infection, active or occult hepatitis B virus infection, active hepatitis C virus infection, positive for Human Immunodeficiency Virus (HIV) antibodies, positive syphilis test, active or latent tuberculosis (TB) infection;
  • A significant history of liver disease, such as steatosis, fibrosis, non-alcoholic steatohepatitis, and cirrhosis, biliary diseases, within 6 months prior to signing the informed consent form, except for Gilbert's syndrome;
  • History of malignant tumors;
  • Imaging (liver ultrasound) evidence of severe liver diseases such as hepatic fibrosis or cirrhosis;
  • In the investigator's assessment, the subject has a history of serious cardiovascular, respiratory, gastrointestinal, endocrine, renal, hematological, neurological, psychiatric, or other systemic diseases before screening;
  • History of allergy to human serum albumin;
  • Participants with a history of substance abuse (e.g., alcohol, heroin, amphetamines, etc.);
  • Participants who have received gene therapy at any time in the past.
  • Participants who have participated in other non-gene therapy drug clinical trials and received the investigational drug within 3 months (or 5 half-lives of the other investigational drug) prior to screening;
  • Participants with elevated Alpha-fetoprotein (AFP);
  • Other conditions that, in the investigator's opinion, make the subject unsuitable for inclusion, such as severe comorbidities associated with PKU (e.g., renal insufficiency or renal failure, osteoporosis, anemia, gastroesophageal reflux or peptic ulcer, major depressive disorder, epilepsy, etc.);
  • Participants whose daily diet includes excessive natural protein intake (>2 g/kg/day).

Study description and what participation involves

The study's detailed description, as published on ClinicalTrials.gov.

Detailed description from the registry

This study will evaluate the safety and efficacy of NGGT002 gene therapy with three dose cohorts in adult subjects with a diagnosis of classic PKU, a condition characterized by severe PAH deficiency with no residual enzyme activity. NGGT002 will be administered through intravenous infusion.

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.

Participants are placed into one of three dose cohorts and receive a single intravenous infusion of NGGT002 (a gene therapy). They will have blood tests to measure plasma phenylalanine at baseline and at Weeks 12, 28, and 52, and safety monitoring for adverse events from baseline through Week 52. Participants will complete PKU quality-of-life questionnaires and record dietary intake to assess daily phenylalanine and natural protein intake (measured at baseline, Week 28, and Week 52). After the first year, participants continue to be followed for safety and efficacy for a total of up to 5 years.

How the study is structured. Open-label, single-group Phase 1/2 study with dose escalation and cohort expansion. Six to eighteen participants will be enrolled across three dose levels. Each participant receives one IV infusion of NGGT002 and then attends follow-up visits with safety and efficacy assessments through Week 52 and continued follow-up up to 5 years. There is no masking (everyone and the study team know the treatment given).

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 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
Jul 16, 2024
Primary completion
Aug 11, 2027 (estimated)
Study completion
Jun 11, 2030 (estimated)
Target enrollment
18 participants (estimated)
Last updated on registry
Aug 13, 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
NGGT (Suzhou) Biotechnology 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 (2)

  • First Affiliated Hospital of Bengbu Medical CollegeRecruiting

    Bengbu, Anhui · China

  • Xinhua Hospital Affifiated to Shanghai Jiao Tong University School of MedicineRecruiting

    Shanghai · China

    • Wenjuan Qiu, PhD
    • Wenjuan Qiu, PhD

Terms used on this page · explanations by MedicalUpdates.ai

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.
cohort
A group of people who share a characteristic, such as a diagnosis, and are studied together. 'Prospective' means they are followed forward from enrolment; 'retrospective' means existing records are reviewed.
informed consent form
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.
screening period
The checks done before enrolment to see whether a person qualifies, such as tests, scans or a review of medical history.
Women of 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.
ULN
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.
contraindications
A reason a particular test or treatment should not be used for a person, for example a metal implant that rules out an MRI scan.
corticosteroid
Steroid medicines, such as prednisone, that reduce inflammation. Studies often require a stable dose at entry or measure whether the study treatment allows the dose to be reduced.
investigational drug
A treatment that is being tested and has not been approved for this use. It may be approved for a different condition. Being studied does not mean it works or is available outside the study.
5 half-lives
The time it takes for the amount of a drug in the body to fall by half. 'Five half-lives' is often used as the point at which a previous drug has essentially left the body.
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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