This study is a single-arm, open-label, dose-escalation, exploratory study to evaluate the safety, tolerability, and efficacy of a single administration of GS1168 Injection in Chinese adult phenylketonuria (PKU) with PAH mutation.
Understand the purpose and risks of the study and voluntarily sign Informed Consent Forms (ICF).
Male or female, aged 18-55 years.
Diagnosis of PKU due to PAH mutation, and be inadequately controlled with current therapy (e.g., dietary management) in the judgement of the investigator.
Intolerant of unresponsive to available treatment such as Kuvan or Palynziq, and have come off the medication for at least 28 days prior to signing the ICF.
Diagnosis of PKU due to other gene mutations.
Body weight ≥100 kg or Body Mass Index (BMI) ≥30 kg/m² at screening.
Presence of anti-AAV8 neutralizing antibody.
ALT or AST >1.25 × Upper Limit of Normal (ULN)
Single group
GS1168 injection
DrugThe dose levels are as follows: 1. A single intravenous administration of GS1168 injection at dose level 1; 2. A single intravenous administration of GS1168 injection at dose level 2; 3. A single intravenous administration of GS1168 injection at dose level 3.
Incidence of adverse event (AE), including serious adverse event (SAE) and adverse event of special interest (AESI)
Incidence of adverse event (AE), including serious adverse event (SAE) and adverse event of special interest (AESI) as assessed by CTCAE V5.0, from baseline to week 52
Plasma Phe levels
Change from baseline in plasma level of Phe following GS1168 injection administration
Total protein intake
Change from baseline in total protein intake following GS1168 injection administration
Phenylketonuria Quality of Life (PKU-QOL) scores
Change in Phenylketonuria Quality of Life Questionnaire (PKU-QOL, Score range: 0-100, higher scores indicate worse outcomes.)
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This trial is not recruiting at the moment. You can still explore other options:
