PhenylketonuriasEarly Phase 1NCT06061614

Safety and Efficacy Study of NGGT002 in PKU Adult Subjects

A Early Phase 1 study testing NGGT002 Injection in people with Phenylketonurias, run by The First Affiliated Hospital of Bengbu Medical University at 1 site in China.

Active, not recruiting

Under way, not looking for participants. Everyone needed has enrolled. The study is still running.

Official record

Search all trials

Trial summary

This is a single-center, open-label, non-randomized, dose escalation study to evaluate the safety, tolerability and efficacy of NGGT002 in adult Phenylketonuria (PKU) subjects. All subjects will receive a single administration of NGGT002 and will be followed for safety and efficacy for 5 years. Read more about this study

Who is it for?: Ages 18 years and older
Conditions the study is for: Phenylketonurias
What is being tested?: NGGT002 Injection
Type of treatment: Injection or infusion
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 15 participants
Where: 1 siteChina
Study dates: 30 Mar 2023to 30 Dec 2028
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 years and older
All sexes
No healthy volunteers
Adult, Older adult

Who may be able to join

Voluntarily sign informed consent form;
Male and female subjects with diagnosis of PKU caused by confirmed phenylalanine hydroxylase(PAH) mutation according to the "Clinical Practice Guidelines for Phenylketonuria, 2020 Edition";
Age ≥ 18 years;
Blood phenylalanine (Phe) concentration ≥ 600 μmol/L at least once within 2 years prior to screening, with one measurement confirmed within six months of enrollment;
Subjects are able to maintain their baseline diet throughout the study (regardless of dietary phenylalanine restriction), and willingness to follow the instruction of investigators to manage the diet for the duration of the trial;
Subjects are required to obtain approval from the investigator prior to the use of any concomitant medications during the study period;
Willingness and capable per Investigator opinion to comply with study procedures and requirements;
Female participants of childbearing potential must have abstained from unprotected sexual intercourse for at least 14 days prior to dosing, and must have a documented negative serum hCG test between Day -7 and Day 0. All participants must be willing to use a highly effective method of contraception for at least 12 months following NGGT002.

Who cannot join

Anti-AAV8 neutralizing antibody>1:10
Prior gene therapy
Positive hepatitis B virus surface antigen, hepatitis C virus antibody, anti-human immunodeficiency virus antibody or treponema pallidum-specific antibody
Hepatic function abnormal: alanine aminotransferase (ALT) or aspartate aminotransferase (AST) > 1.5 × ULN; alkaline phosphatase (ALP) > 1.5 × ULN; total bilirubin (TBil) > 1.5 × ULN; international normalized ratio (INR) > 1.3
Serum creatinine(Scr)> 1.5 × ULN
Hematology values outside of the normal range (Hemoglobin < 110 g/L (male), < 100 g/L (female), white blood cells < 3.0 × 10^9/L, neutrophils < 1.5 × 10^9/L, platelet counts < 100 × 10^9/L;

Show all 15Show fewer

Hemoglobin A1c > 6%, or fasting glucose > 6.1 mmol/L;
Clinically significant abnormalities in vital signs, physical examination findings, laboratory tests, or other assessments that, in the Investigator's judgment, are deemed unsuitable for study enrollment;
Any contraindications to corticosteroid use or conditions potentially worsened by corticosteroids, as assessed by the Investigator, including but not limited to hypersensitivity to glucocorticoids, epilepsy, recent or unresolved bone fractures, ongoing wound healing, uncontrolled infections, or clinically significant osteoporosis;
Subjects with a history of allergy to human serum albumin;
All types of past and current malignancy;
Severe diseases in the cardiovascular, respiratory, digestive tract, endocrine, kidney, blood, nervous, mental and other systems before screening;
Subjects with history of live diseases, such as hepatitis, liver cirrhosis, liver cancer or other serious liver diseases;
Subjects who participated in other clinical trails and took drugs within 3 months before screening;
Other conditions that the Investigators deemed inappropriate for enrollment.

Trial locations and contacts

1 site in 1 country.

China1 site

The First Affiliated Hospital of Bengbu Medical College
Bengbu, Anhui, 233000

Other trials in China

About this study

Official title: A Clinical Study for the Safety and Efficacy of IV Infusion of NGGT002 in the Treatment of Phenylketonuria

Read the detailed description

This study is designed to evaluate the safety and efficacy of NGGT002 gene therapy in adult subjects diagnosis with PKU due to confirmed PAH gene mutations indicative of PAH deficiency. NGGT002 will be administered via intravenous infusion. The study will begin with Dose Level 1, followed by a stepwise dose escalation. After evaluating the safety and efficacy data, a decision will be made to either expand the current cohort or proceed to the next dose level. The same process will be followed for subsequent dose cohorts.

Conditions: Phenylketonurias
Being tested: NGGT002 Injection
Keywords: Phenylalanine hydroxylase (PAH) Deficiency
Medical subject headings: Phenylketonurias

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

NGGT002 InjectionGenetic
Dose escalation will proceed sequentially from Dose Level 1 to Dose Level 5 in a single-dose administration of NGGT002.

Study groups in NCT06061614
Study group	Role	What they receive
Dose level 1	Experimental	Dose level 1 will be administered Receives: Genetic: NGGT002 Injection
Dose level 2	Experimental	Dose level 2 will be administered Receives: Genetic: NGGT002 Injection
Dose level 3	Experimental	Dose level 3 will be administered Receives: Genetic: NGGT002 Injection
Dose level 4	Experimental	Dose level 4 will be administered Receives: Genetic: NGGT002 Injection
Dose level 5	Experimental	Dose level 5 will be administered Receives: Genetic: NGGT002 Injection

What is being measured

The primary outcome is the main question the study is designed to answer.

Incidence and severity of adverse events (AEs) and serious adverse events (SAEs)
Incidence and severity of adverse events (AEs) and serious adverse events (SAEs) from baseline to week 52 (W52)
Measured: Week 52

Secondary outcomes (3)

Plasma Phe levels
Changes from baseline in plasma Phe levels
Measured: Baseline Week 12, Week 28, Week 52
Nature protein intake
Changes from baseline in nature protein intake (g/kg body weight [BW]/day)
Measured: Baseline, Week 12, Week 28, and Week 52
Food Phe intake
Changes from baseline in food Phe intake (mg/kg body weight [BW]/day)
Measured: Baseline, Week 12, Week 28, and Week 52

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

30 Mar 202330 Dec 2028

First posted29 Sep 2023
Study start30 Mar 2023
Primary completion30 Dec 2028 (estimated)
Study completion30 Dec 2028 (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: Early Phase 1
Primary purpose: Treatment
Allocation: Non-randomised
Intervention model: Sequential
Model description: Single group
Masking: None (open label)
Enrolment: 15 participants (estimated)

IDs, dates and publications

ClinicalTrials.gov ID: NCT06061614
Sponsor's protocol ID: NGGT002-P-2202
Status last verified: Feb 2026
First submitted: 25 Sep 2023
Last updated: 6 Aug 2026
Results posted: No
Data monitoring committee: No
FDA-regulated drug: No
FDA-regulated device: No
Individual data sharing: No

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

RecruitingPhase 3
A Study of Sepiapterin in Participants With Phenylketonuria (PKU)
Phenylketonuria
RecruitingPhase 2
A Study of MZE782 in Adults With PKU
Phenylketonuria (PKU)
Recruiting
Impact of Phenylalanine Elevations on Brain and Cognition in Adult PKU Carriers
Carrier of Phenylketonuria, Healthy
RecruitingPhase 3
A Study of JNT-517 in Participants With Phenylketonuria (PKU)
Phenylketonuria
Not yet recruiting
Market Research of a Slow Release Protein Substitute for the Dietary Management of PKU
Phenylketonuria (PKU) and Hyperphenylalaninemia, Brain Diseases, Metabolic, Inborn

All Phenylketonurias trials