NCT06687733 Safety and Efficacy Study of NGGT002 in cPKU Adult Subjects
| NCT ID | NCT06687733 |
| Status | Recruiting |
| Phase | Phase 1, Phase 2 |
| Sponsor | NGGT (Suzhou) Biotechnology Co., Ltd. |
| Condition | Phenylketonurias |
| Study Type | INTERVENTIONAL |
| Enrollment | 18 participants |
| Start Date | 2024-07-25 |
| Primary Completion | 2026-07-30 |
Trial Parameters
Eligibility Fast-Check
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Brief 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. Participants will receive a single administration of NGGT002 and will be followed for safety and efficacy for 5 years.
Eligibility Criteria
Inclusion Criteria: 1. Voluntarily participating in the study and signing the informed consent form; 2. Gender is not limited; patients must carry biallelic pathogenic or likely pathogenic variants in the PAH gene; 3. Adult patients aged 18 to 55 years; 4. 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; 5. Willing and able to manage their diet; 6. According to the investigator's opinion, willing and able to comply with the study procedures and requirements; 7. 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. Exclusion Criteria: 1. Presence of anti-AAV8 neutralizing antibodies（≥1:5） 2. Subjects whose disease is well-controlled with existing therapies, such as those currently receiving m