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

NCT ID: NCT07318909

Last Updated: 2026-01-06

Basic Information

• Recruitment Status: NOT_YET_RECRUITING
• Clinical Phase: EARLY_PHASE1
• Total Enrollment: 9 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2025-12-31
• Study Completion Date: 2031-08-30

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)

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Treatment group Arm of GS1168

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

Group Type: EXPERIMENTAL

GS1168 injection

Intervention Type: 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.

Interventions

GS1168 injection

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.

Intervention Type: DRUG

Eligibility Criteria

Inclusion Criteria

1. Understand the purpose and risks of the study and voluntarily sign Informed Consent Forms (ICF).

2. Male or female, aged 18-55 years.

3. Diagnosis of PKU due to PAH mutation, and be inadequately controlled with current therapy (e.g., dietary management) in the judgement of the investigator.

4. 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.

5. Ability and willingness to maintain dietary protein intake consistent with baseline

6. 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.

7. Agree to avoid alcohol consumption from 30 days prior to GS1168 infusion until at least 52 weeks after infusion.

8. Ability to communicate well with the Investigator and to understand and comply with the requirements of the study.

Exclusion Criteria

1. Diagnosis of PKU due to other gene mutations.

2. Body weight ≥100 kg or Body Mass Index (BMI) ≥30 kg/m² at screening.

3. Presence of anti-AAV8 neutralizing antibody.

4. 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

5. 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

6. Active or occult hepatitis B virus (HBV) infection, active hepatitis C virus (HCV) infection, positive Human Immunodeficiency Virus (HIV) antibodies and positive syphilis test.

7. Any severe conditions of respiratory, cardiovascular, gastrointestinal, renal, neurological, endocrine, immunological, hematological, psychiatric, etc.

8. History of malignancy.

9. Any other condition that, in the opinion of the Investigator, may affect subject compliance or render the subject unsuitable for participation in this trial.

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 55 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Gritgen Therapeutics Co., Ltd.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

The First Affiliated Hospital of Anhui Medical University

Hefei, Anhui, China

Countries

China

Central Contacts

Yuntao Gong, MD

Role: CONTACT

yuntao.gong@gritgen.com

+8618910178979

Sihui Pan

Role: CONTACT

sihui.pan@gritgen.com

+8618105158856

Facility Contacts

Huan Zhou, PhD

Role: primary

zhouhuanbest@vip.163.com

+8613665527160

Datong Deng, MD

Role: backup

13855134251@163.com

+8613855134251

Other Identifiers

GS1168-201

Identifier Type: -

Identifier Source: org_study_id