PhenylketonuriasPhase 1/2NCT06687733
Safety and Efficacy Study of NGGT002 in Adult Patients With Phenylketonuria
A Phase 1/2 study testing NGGT002 in people with Phenylketonurias, run by NGGT (Suzhou) Biotechnology Co., Ltd. at 2 sites in China.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 18 to 55 years
- Conditions the study is for
- Phenylketonurias
- What is being tested?
- NGGT002
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 18 participants
- Where
- 2 sitesChina
- Study dates
- 16 Jul 2024to 11 Jun 2030
- Phase
- Phase 1/2
- 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 55 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- 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.
Who cannot join
- 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
Show all 24Show fewer
- 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).
Trial locations and contacts
2 sites in 1 country. Contact the study team or a site near you to ask about taking part.
China2 sites, 2 recruiting
- Recruiting
First Affiliated Hospital of Bengbu Medical College
Bengbu, Anhui
Huan Zhou, PhD, 8613665527160, zhouhuanbest@vip.163.com
- Recruiting
Xinhua Hospital Affifiated to Shanghai Jiao Tong University School of Medicine
Shanghai
Wenjuan Qiu, PhD
Wenjuan Qiu, PhD
About this study
Official title: A Phase I/II Study for the Safety and Efficacy of Intravenous Infusion With NGGT002 in Adults Patients With Phenylketonuria
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.
Read the detailed description
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.
- Conditions
- Phenylketonurias
- Being tested
- NGGT002
- Medical subject headings
- Phenylketonurias
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
NGGT002Genetic
adeno-associated viral vector with human phenylalanine hydroxylase gene
| Study group | Role | What they receive |
|---|---|---|
| NGGT002 | Experimental | Six to eighteen patients will be enrolled into three cohorts at three dose levels. Receives: Genetic: NGGT002 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence and severity of Adverse Events (AEs)
Incidence and severity of AEs, including serious AEs (SAEs) as assessed by CTCAE v5.0 of a single administration of NGGT002.
Measured: Baseline to Week 52
Change from baseline in average Plasma Phe Concentration at Weeks 12, 28 and 52.
To evaluate the efficacy of intravenous infusion of NGGT002 in adults with classic PKU, as assessed by the change from baseline in mean plasma Phe concentration at Weeks 12, 28, and 52.
Measured: Baseline, Week 12, Week 28, Week 52
Secondary outcomes (4)
Incidence of participants in each dose cohort with a plasma Phe concentration ≤360 μmol/L (6 mg/dL) at Weeks 12, 28, and 52 following NGGT002 administration.
Participants achieving a sustained plasma Phe concentration ≤360 μmol/L (6 mg/dL) at Week 12, Week 28, Week 52 post dose.
Measured: Week 12, Week 28, Week 52
Time to first achievement of a plasma Phe concentration ≤360 μmol/L and duration of maintenance of plasma Phe concentration at ≤360 μmol/L in each dose cohort following NGGT002 administration.
Time (weeks) to first achievement of a blood Phe ≤ 360 μmol/L and the duration(weeks) of Phe ≤ 360 μmol/L in each dose group following NGGT002 administration
Measured: Week 52
Change from baseline in daily Phe intake (mg/day) and total natural protein intake (g/day) at Weeks 28 and 52.
Participants achieving a change from baseline in Phe and nature protein intake at Week 28, Week 52 post dose.
Measured: Baseline, Week 28, Week 52
Change from baseline in Phenylketonuria Quality of Life Questionnaire (PKU-QOL) score at Weeks 28 and 52.
Change from baseline in PKU-QOL score at Week 28, Week 52 following dosing.
Measured: Baseline, Week 28, Week 52
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Nov 2024
- Study start16 Jul 2024
- Primary completion11 Aug 2027 (estimated)
- Study completion11 Jun 2030 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for PhenylketonuriasStudies 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
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 18 participants (estimated)
Sponsor and investigators
- Lead sponsor
- NGGT (Suzhou) Biotechnology Co., Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Jianping Weng, PhD, Principal investigator, The First Affiliated Hospital of Bengbu Medical University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06687733
- Sponsor's protocol ID
- NGGT002-P-2302
- Status last verified
- Aug 2026
- First submitted
- 11 Nov 2024
- Last updated
- 13 Aug 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 13 Aug 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase I/II Study for the Safety and Efficacy of Intravenous Infusion With NGGT002 in Adults Patients With Phenylketonuria
- ClinicalTrials.gov ID
- NCT06687733
- Acronym / sponsor ID
- NGGT002-P-2302
- Conditions
- Phenylketonurias
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 18 participants (estimated)
- Lead sponsor
- NGGT (Suzhou) Biotechnology Co., Ltd.Industry (pharma, biotech, devices)
- Study start
- 16 Jul 2024 (actual)
- Primary completion
- 11 Aug 2027 (estimated)
- Study completion
- 11 Jun 2030 (estimated)
- First posted
- 14 Nov 2024 (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 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.
Detailed description
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| NGGT002 | Experimental | Genetic: NGGT002Six to eighteen patients will be enrolled into three cohorts at three dose levels. |
Who may qualify
- Ages
- 18 to 55 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (7 listed)
- 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;
Key exclusion criteria (24 listed)
- 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:
- 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;
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 |
|---|---|---|
| NGGT002adeno-associated viral vector with human phenylalanine hydroxylase gene | Genetic | NGGT002 |
Primary outcome
Incidence and severity of Adverse Events (AEs)
Time frame: Baseline to Week 52
Incidence and severity of AEs, including serious AEs (SAEs) as assessed by CTCAE v5.0 of a single administration of NGGT002.
Change from baseline in average Plasma Phe Concentration at Weeks 12, 28 and 52.
Time frame: Baseline, Week 12, Week 28, Week 52
To evaluate the efficacy of intravenous infusion of NGGT002 in adults with classic PKU, as assessed by the change from baseline in mean plasma Phe concentration at Weeks 12, 28, and 52.
4 secondary outcomes
Incidence of participants in each dose cohort with a plasma Phe concentration ≤360 μmol/L (6 mg/dL) at Weeks 12, 28, and 52 following NGGT002 administration.
Time frame: Week 12, Week 28, Week 52
Time to first achievement of a plasma Phe concentration ≤360 μmol/L and duration of maintenance of plasma Phe concentration at ≤360 μmol/L in each dose cohort following NGGT002 administration.
Time frame: Week 52
Change from baseline in daily Phe intake (mg/day) and total natural protein intake (g/day) at Weeks 28 and 52.
Time frame: Baseline, Week 28, Week 52
Change from baseline in Phenylketonuria Quality of Life Questionnaire (PKU-QOL) score at Weeks 28 and 52.
Time frame: Baseline, Week 28, Week 52
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 13 Aug 2026.
- Individual participant dataSharing planned: No
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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.
Jianping Weng, PhD principal investigator
The First Affiliated Hospital of Bengbu Medical University
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing NGGT002 in people with Phenylketonurias, run by NGGT (Suzhou) Biotechnology Co., Ltd. at 2 sites 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 7 inclusion and 24 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 13 Aug 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites 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. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined 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.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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 Huan Zhou, PhD (8613665527160, zhouhuanbest@vip.163.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence and severity of Adverse Events (AEs) (time frame: Baseline to Week 52). 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 13 Aug 2026.