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Tests treatment safety and results for Phenylketonurias
Official title Safety and Efficacy Study of NGGT002 in PKU Adult Subjects
ClinicalTrials.gov ID: NCT06061614
What this study is testing
What is NGGT002 Injection?
NGGT002 Injection is an investigational medicine, given as an infusion into a vein, being studied as a potential treatment for phenylketonurias.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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.
- Phase 1: an early, usually small safety study
- Which group you join is decided by chance.
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 18 and older
You may be able to join if
- Voluntarily sign informed consent form;
- Male and female people with diagnosis of PKU caused by confirmed phenylalanine hydroxylase(PAH) mutation according to the "Clinical Practice...
- 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...
- people are able to maintain their baseline diet throughout the study (regardless of dietary phenylalanine restriction), and willingness to follow the...
You likely can't join if
- 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...
- Hepatic function abnormal: alanine aminotransferase (ALT) or aspartate aminotransferase (AST) \> 1.5 × ULN; alkaline phosphatase (ALP) \> 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 \<...
- Hemoglobin A1c \> 6%, or fasting glucose \> 6.1 mmol/L;
See the full eligibility criteria
- Voluntarily sign informed consent form;
- Male and female people 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;
- people 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...
- people 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...
- 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...
- 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;
- 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...
- people 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;
- people with history of live diseases, such as hepatitis, liver cirrhosis, liver cancer or other serious liver diseases;
- people who participated in other clinical trails and took drugs within 3 months before screening;
- Other conditions that the Investigators deemed inappropriate for enrollment.
The study team makes the final eligibility decision.
Where it's taking place
- Bengbu, Anhui, China
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 18 years and older. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Bengbu, Anhui, China. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.