Health TrialsbyHealthPanel

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

This trial does not accept healthy volunteers

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

Design model

Single group

Treatments tested in this trial

  • NGGT002

    Genetic

    adeno-associated viral vector with human phenylalanine hydroxylase gene

Treatment groups

18 Participants
are divided into 1 treatment group
Group A: NGGT002Experimental treatment 1 intervention

Trial outcomes

Primary outcomes

1

Incidence and severity of Adverse Events (AEs)

Incidence and severity of AEs, including serious AEs (SAEs) as assessed by CTCAE v5.0 of a single administration of NGGT002.

Time frame
Baseline to Week 52
2

Change from baseline in average Plasma Phe Concentration

To evaluate the efficacy in change of average plasma Phe concentration of IV infusion of NGGT002 in adults with classic PKU at Week 12, Week 28, Week 52

Time frame
Week 12, Week 28, Week 52

Secondary outcomes

1

Incidence of sustained plasma Phe concentration of ≤360 μmol/L (6 mg/dL) at Week 12, Week 28, Week 52 post dose

Subjects achieving a sustained plasma Phe concentration ≤360 μmol/L (6 mg/dL) at Week 12, Week 28, Week 52 post dose.

Time frame
Week 12, Week 28, Week 52
2

Occurred Day to first reach Phe ≤ 360 μmol/L and the duration（days） of Phe ≤ 360 μmol/L in each dose group following NGGT002 administration

Time（days） to first reach Phe ≤ 360 μmol/L and the duration（days） of Phe ≤ 360 μmol/L in each dose group following NGGT002 administration

Time frame
Week 52
3

Change from baseline in Phe and total protein intake at Week 28, Week 52 post dose

Subject achieving a change from baseline in Phe and total protein intake at Week 28, Week 52 post dose.

Time frame
Week 28, Week 52
4

Score change in Phenylketonuria Quality of Life Questionnaire (PKU-QOL)

Score change in PKU-QOL at Week 28, Week 52 post dose

Time frame
Week 28, Week 52

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

NGGT (Suzhou) Biotechnology Co., Ltd.

Lead sponsor

Source ClinicalTrials.gov
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