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Tests treatment safety and results for Phenylketonurias
Official title Safety and Efficacy Study of NGGT002 in cPKU Adult Subjects
ClinicalTrials.gov ID: NCT06687733
What this study is testing
What is NGGT002?
NGGT002 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 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.
- Phase 2: a mid-size study of how well it works
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 18 to 55
You may be able to join if
- 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...
- Willing and able to manage their diet;
You likely can't join if
- Presence of anti-AAV8 neutralizing antibodies（≥1:5）
- people whose disease is well-controlled with existing therapies, such as those currently receiving medications like Sapropterin Dihydrochloride...
- 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
See the full eligibility 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.
- Presence of anti-AAV8 neutralizing antibodies（≥1:5）
- people whose disease is well-controlled with existing therapies, such as those currently receiving medications like Sapropterin Dihydrochloride tablets, Pegvaliase-pqpz, etc.;
- 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 subject 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...
- 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...
- 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...
- History of allergy to human serum albumin;
- people with a history of substance abuse (e.g., alcohol, heroin, amphetamines, etc.);
- people who have received gene therapy at any time in the past.
- people 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;
- people 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...
- people weighing more than 100 kg;
- people whose daily diet includes excessive natural protein intake (\>2 g/kg/day).
The study team makes the final eligibility decision.
Where it's taking place
- Bengbu, Anhui, China
- Shanghai, 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 to 55 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Bengbu, Anhui, China; Shanghai, 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.