NCT06061614 Safety and Efficacy Study of NGGT002 in PKU Adult Subjects
| NCT ID | NCT06061614 |
| Status | Recruiting |
| Phase | EARLY_Phase 1 |
| Sponsor | The First Affiliated Hospital of Bengbu Medical University |
| Condition | Phenylketonurias |
| Study Type | INTERVENTIONAL |
| Enrollment | 15 participants |
| Start Date | 2023-03-30 |
| Primary Completion | 2028-12-30 |
Eligibility & Interventions
Eligibility Fast-Check
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What to Expect as a Participant
You will actively receive the study intervention — which may be a drug, biologic, device, or procedure.
Phase 1 is the earliest stage of human testing — safety and dosage are the primary focus. Visits are frequent and medical supervision is intensive. You will be among the first people to receive this treatment.
This trial targets 15 participants in total. It began in 2023-03-30 with a primary completion date of 2028-12-30.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief 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.
Eligibility Criteria
Inclusion Criteria: * 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. Exclusion Criteria: * 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 * 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 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.
Contact & Investigator
Frequently Asked Questions
Who can join the NCT06061614 clinical trial?
This trial is open to participants of all sexes, aged 18 Years or older, studying Phenylketonurias. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
What phase is the NCT06061614 trial and what does that mean for participants?
Phase 1 trials are the first stage of human testing. The primary goal is to assess safety and determine appropriate dosage levels. Participants are closely monitored. These trials typically involve a small number of volunteers.
Is NCT06061614 currently recruiting?
Yes, NCT06061614 is actively recruiting participants. Contact the research team at zhouhuanbest@vip.163.com for enrollment information.
Where is the NCT06061614 trial being conducted?
This trial is being conducted at Bengbu, China.
Who is sponsoring the NCT06061614 clinical trial?
NCT06061614 is sponsored by The First Affiliated Hospital of Bengbu Medical University. The trial plans to enroll 15 participants.