Back to resultsAAV8 Gene Therapy in PKU Adult Subjects
Primary Purpose
Phenylketonurias
Status
Recruiting
Phase
Early Phase 1
Locations
China
Study Type
Interventional
Intervention
NGGT002
Sponsored by
About this trial
This is an interventional treatment trial for Phenylketonurias focused on measuring PAH Deficiency
Eligibility Criteria
Inclusion Criteria: Voluntarily sign informed consent form; Male and female subjects with diagnosis of PKU caused by confirmed PAH mutation as per the "Clinical Practice Guidelines for Phenylketonuria 2020"; Age ≥ 18 years; Phe concentration ≥ 600 μmol/L at screening and ≥ 600 μmol/L at least once within 2 years prior to screening; Subjects who are willing and able to maintain their baseline diet throughout the study, regardless of phenylalanine restriction, except at the investigator's request; Exclusion Criteria: Presence of anti-AAV8 neutralizing antibody Prior gene therapy Positive hepatitis B virus surface antigen, hepatitis C virus antibody, anti-human immunodeficiency virus antibody or treponema pallidum-specific antibody Hepatic function abnormal: alanine aminotransferase (ALT) or aspartate aminotransferase (AST) > 1.5 × ULN; alkaline phosphatase (ALP) > 1.5 × ULN; total bilirubin (TBil) > 1.5 × ULN; international normalized ratio (INR) > 1.3
Sites / Locations
- The First Affiliated Hospital of Bengbu Medical CollegeRecruiting
Arms of the Study
Arm 1
Arm 2
Arm 3
Arm Type
Experimental
Experimental
Experimental
Arm Label
Dose level 1
Dose level 2
Dose level 3
Arm Description
Dose level 1 will be administered
Dose level 2 will be administered
Dose level 3 will be administered
Outcomes
Primary Outcome Measures
Incidence and severity of adverse events (AEs) and serious adverse events (SAEs)
Incidence and severity of adverse events (AEs) and serious adverse events (SAEs) from baseline to week 52 (W52)
Secondary Outcome Measures
plasma Phe levels
Change from baseline in mean plasma Phe levels
Full Information
NCT ID
NCT06061614
First Posted
September 25, 2023
Last Updated
September 25, 2023
Sponsor
First Affiliated Hospital Bengbu Medical College
1. Study Identification
Unique Protocol Identification Number
NCT06061614
Brief Title
AAV8 Gene Therapy in PKU Adult Subjects
Official Title
A Clinical Study for the Safety and Efficacy of IV Infusion of NGGT002 in the Treatment of Phenylketonuria
Study Type
Interventional
2. Study Status
Record Verification Date
August 2023
Overall Recruitment Status
Recruiting
Study Start Date
March 30, 2023 (Actual)
Primary Completion Date
December 30, 2028 (Anticipated)
Study Completion Date
December 30, 2028 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
First Affiliated Hospital Bengbu Medical College
4. Oversight
Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No
5. Study Description
Brief Summary
This is a open-label, dose escalation study in adult PKU particpants.
Detailed Description
This is a investigator initated trial which is an open-label, dose escalation study to evaluate the safety, tolerability of NGGT002 in adult PKU subjects with PAH deficiency. All Participants will only receive one administration of NGGT002 and will be followed for safety and efficacy up to five years.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Phenylketonurias
Keywords
PAH Deficiency
7. Study Design
Primary Purpose
Treatment
Study Phase
Early Phase 1
Interventional Study Model
Sequential Assignment
Model Description
single group
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
9 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
Dose level 1
Arm Type
Experimental
Arm Description
Dose level 1 will be administered
Arm Title
Dose level 2
Arm Type
Experimental
Arm Description
Dose level 2 will be administered
Arm Title
Dose level 3
Arm Type
Experimental
Arm Description
Dose level 3 will be administered
Intervention Type
Genetic
Intervention Name(s)
NGGT002
Intervention Description
NGGT002
Primary Outcome Measure Information:
Title
Incidence and severity of adverse events (AEs) and serious adverse events (SAEs)
Description
Incidence and severity of adverse events (AEs) and serious adverse events (SAEs) from baseline to week 52 (W52)
Time Frame
Week 52
Secondary Outcome Measure Information:
Title
plasma Phe levels
Description
Change from baseline in mean plasma Phe levels
Time Frame
W12, W28, W52
Other Pre-specified Outcome Measures:
Title
total protein intake
Description
Change from baseline in total protein intake
Time Frame
W12, W28, W52
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Voluntarily sign informed consent form;
Male and female subjects with diagnosis of PKU caused by confirmed PAH mutation as per the "Clinical Practice Guidelines for Phenylketonuria 2020";
Age ≥ 18 years;
Phe concentration ≥ 600 μmol/L at screening and ≥ 600 μmol/L at least once within 2 years prior to screening;
Subjects who are willing and able to maintain their baseline diet throughout the study, regardless of phenylalanine restriction, except at the investigator's request;
Exclusion Criteria:
Presence of anti-AAV8 neutralizing antibody
Prior gene therapy
Positive hepatitis B virus surface antigen, hepatitis C virus antibody, anti-human immunodeficiency virus antibody or treponema pallidum-specific antibody
Hepatic function abnormal: alanine aminotransferase (ALT) or aspartate aminotransferase (AST) > 1.5 × ULN; alkaline phosphatase (ALP) > 1.5 × ULN; total bilirubin (TBil) > 1.5 × ULN; international normalized ratio (INR) > 1.3
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Huan Zhou, Doctor
Phone
008613665527160
Email
zhouhuanbest@vip.163.com
First Name & Middle Initial & Last Name or Official Title & Degree
Xiaoli Li, Master
Phone
008615215520890
Email
158169847@qq.com
Facility Information:
Facility Name
The First Affiliated Hospital of Bengbu Medical College
City
Bengbu
State/Province
Anhui
ZIP/Postal Code
233000
Country
China
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Huan Zhou
Phone
+8613665527160
Email
zhouhuanbest@vip.163.com
First Name & Middle Initial & Last Name & Degree
Xiaoli Li
Phone
+8615215520890
Email
158169847@qq.com
12. IPD Sharing Statement
Plan to Share IPD
No
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AAV8 Gene Therapy in PKU Adult Subjects
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