To Evaluate the Safety and Efficacy of GS1168 Injection in Adult Phenylketonuria

NCT07318909 | Not Yet Recruiting Early Phase 1

• 1 other identifier: GS1168-201
• Study Type: interventional
• Target: 9
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Phenylketonuria (PKU)

Outcome Measures

Primary Outcomes (1)

• 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

  Week 52

Secondary Outcomes (3)

• Plasma Phe levels

  Week 52

• Total protein intake

  Week 52

• Phenylketonuria Quality of Life (PKU-QOL) scores

  Week 52

Study Arms (1)

Treatment group Arm of GS1168

EXPERIMENTAL

Dose level 1, level 2 and level 3 will be administered

Drug: GS1168 injection

Interventions

GS1168 injection DRUG

The 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.

Treatment group Arm of GS1168

Eligibility Criteria

• Age: 18 Years - 55 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64)

You may qualify if:

• 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.
• Ability and willingness to maintain dietary protein intake consistent with baseline
• Male and female subjects of childbearing potential must agree to use effective contraception methods from signing ICF until at least 52 weeks after GS1168 infusion. Female subjects must not be breastfeeding.
• Agree to avoid alcohol consumption from 30 days prior to GS1168 infusion until at least 52 weeks after infusion.
• Ability to communicate well with the Investigator and to understand and comply with the requirements of the study.

You may not qualify if:

• 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.
• Abnormal liver function tests at screening:
• ALT or AST \>1.25 × Upper Limit of Normal (ULN)
• GGT \>1.25 × ULN
• Total Bilirubin \>1.5 × ULN
• Abnormal laboratory findings at screening:
• Hemoglobin \<9 g/dL
• Neutrophil count \<1.0 × 10⁹/L
• Platelet count \<100 × 10⁹/L
• Serum Creatinine ≥1.5 mg/dL (133 μmol/L)
• Glycated Hemoglobin (HbA1c) \>6% or Fasting Blood Glucose \>6.1 mmol/L
• Active or occult hepatitis B virus (HBV) infection, active hepatitis C virus (HCV) infection, positive Human Immunodeficiency Virus (HIV) antibodies and positive syphilis test.
• Any severe conditions of respiratory, cardiovascular, gastrointestinal, renal, neurological, endocrine, immunological, hematological, psychiatric, etc.

Sponsors & Collaborators

• Gritgen Therapeutics Co., Ltd.: lead

Study Sites (1)

The First Affiliated Hospital of Anhui Medical University

Hefei, Anhui, 230022, China

Location

MeSH Terms

Conditions

Phenylketonurias

Condition Hierarchy (Ancestors)

Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Amino Acid Metabolism, Inborn Errors; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Metabolic Diseases; Nutritional and Metabolic Diseases

Central Study Contacts

Yuntao Gong, MD

CONTACT

+8618910178979; yuntao.gong@gritgen.com

Sihui Pan

CONTACT

+8618105158856; sihui.pan@gritgen.com

Study Design

• Study Type: interventional
• Phase: early phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 1, 2025
• First Posted: January 6, 2026
• Study Start: December 31, 2025
• Primary Completion (Estimated): August 30, 2027
• Study Completion (Estimated): August 30, 2031
• Last Updated: January 6, 2026

Record last verified: 2025-12

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)