Safety and Efficacy Study of NGGT002 in cPKU Adult Subjects
ConditionPhenylketonurias
Trial statusRecruiting
Trial phasePhase 1, Phase 2
Trial typeInterventional
Biological sexAll
Age18-55
SponsorNGGT (Suzhou) Biotechnology Co., Ltd.
About this trial
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
Qualifiers
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;
Disqualifiers
Presence of anti-AAV8 neutralizing antibodies（≥1:5）
Subjects whose disease is well-controlled with existing therapies, such as those currently receiving medications like Sapropterin Dihydrochloride tablets, Pegvaliase-pqpz, etc.;
Alanine Transaminase (ALT) > 1.5×ULN and/or Aspartate Aminotransferase (AST) > 1.5×ULN
Alkaline Phosphatase (ALP) > 1.5×ULN
Trial design
Treatments tested in this trial
- NGGT002
Treatment groups
18 Participants
are divided into 1 treatment groupLocations
2First Affiliated Hospital of Bengbu Medical College BengbuAnhui, China
Xinhua Hospital Affifiated to Shanghai Jiao Tong University School of Medicine Shanghai China
Sponsors and collaborators
NGGT (Suzhou) Biotechnology Co., Ltd.
Lead sponsor
Source ClinicalTrials.gov