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 groupSponsors and collaborators
Source ClinicalTrials.gov