Back to resultsEvaluation of the Safety and Efficacy of Infantile-onset Pompe Disease Gene Therapy Drug
Primary Purpose
Pompe Disease Infantile-Onset
Status
Recruiting
Phase
Phase 1
Locations
China
Study Type
Interventional
Intervention
GC301
Sponsored by
About this trial
This is an interventional treatment trial for Pompe Disease Infantile-Onset
Eligibility Criteria
Inclusion Criteria: Age < 6 months Patient has diagnosis of infantile onset Pompe disease The patient's legal guardian(s) must be able to understand the purpose and risks of the study and voluntarily provide signed and dated informed consent prior to any study-related procedures being performed. Exclusion Criteria: Left ventricle ejection fraction (LVEF) < 40%; Patient who has AAV9 neutralizing antibody titer ≥ 1:100； Patient who has received enzyme replacement therapy (ERT) more than twice; Patient who has respiratory dysfunction before enrollment, including the blood oxygen (O2) saturation level < 90%, or the partial pressure of carbon dioxide (PCO2) in venous blood > 55 mmHg, or PCO2 in arterial blood > 40 mmHg; Patient who has laboratory abnormalities of: creatinine > Upper Limit of Normal (ULN), hemoglobin < 90 g/L; Patient with congenital organ absence; Patient with a history of glucocorticoid allergy; Patient who is positive for human immunodeficiency (HIV) antibody, hepatitis B surface antigen, hepatitis C antibody, or treponema pallidum antibody; Patient who has participated in a previous gene therapy research trial; Patient who has any concurrent clinically significant major disease or any other condition that, in the opinion of the investigator, makes the subject unsuitable for participation in the study.
Sites / Locations
- Peking Union Medical CollegeRecruiting
- 301 Chinese PLA General Hospital
- Central South University, Xiangya Hospital
- Zhejiang University, School of Medicine, The Children's HospitalRecruiting
- The First Affiliated Hospital of Zhengzhou University
Arms of the Study
Arm 1
Arm 2
Arm 3
Arm Type
Experimental
Experimental
Experimental
Arm Label
Cohort 1
Cohort 2
Cohort 3
Arm Description
Single intravenous administration of GC301 at a dose of 8.0 x 10^13 vector genomes per kilogram body weight
Single intravenous administration of GC301 at a dose of 1.2 x 10^14 vector genomes per kilogram body weight
Single intravenous administration of GC301 at a dose of 1.8 x 10^14 vector genomes per kilogram body weight
Outcomes
Primary Outcome Measures
Safety and tolerability over time
Frequency of adverse events (AEs), serious adverse events (SAEs), and changes from baseline in relevant clinical laboratory tests
Proportion of patients treated with GC301 who are alive
Secondary Outcome Measures
Proportion of patients treated w/ GC301 who were alive and free of ventilator support
Changes from baseline Left Ventricular Mass (LVM) annd LVMI (LVM index)
Changes from baseline creatine kinase (CK), CK-MB, Troponin I, B-Type Natriuretic Peptide (BNP)
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT05793307
Brief Title
Evaluation of the Safety and Efficacy of Infantile-onset Pompe Disease Gene Therapy Drug
Official Title
A Multi-centered, Single Arm, Open Labeled, Study to Evaluate the Safety and Efficacy of an Adeno-associated Virus Vector Expressing the Human Acid Alpha-glucosidase (GAA) Transgene Intravenous Injection in Patients With Infantile-onset Pompe Disease
Study Type
Interventional
2. Study Status
Record Verification Date
June 2023
Overall Recruitment Status
Recruiting
Study Start Date
June 2, 2023 (Actual)
Primary Completion Date
December 2024 (Anticipated)
Study Completion Date
December 2024 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
GeneCradle Inc
4. Oversight
Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes
5. Study Description
Brief Summary
This study is being conducted to evaluate the safety and effectiveness of GC301 adeno-associated virus vector expressing codon-optimized human acid alpha-glucosidase (GAA) as potential gene therapy for Pompe disease. Patients diagnosed with infantile-onset Pompe disease who are younger than 6 months old will be studied.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Pompe Disease Infantile-Onset
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 1, Phase 2
Interventional Study Model
Sequential Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
16 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
Cohort 1
Arm Type
Experimental
Arm Description
Single intravenous administration of GC301 at a dose of 8.0 x 10^13 vector genomes per kilogram body weight
Arm Title
Cohort 2
Arm Type
Experimental
Arm Description
Single intravenous administration of GC301 at a dose of 1.2 x 10^14 vector genomes per kilogram body weight
Arm Title
Cohort 3
Arm Type
Experimental
Arm Description
Single intravenous administration of GC301 at a dose of 1.8 x 10^14 vector genomes per kilogram body weight
Intervention Type
Genetic
Intervention Name(s)
GC301
Intervention Description
GC301, is an adeno-associated virus 9 (AAV9) vector delivering a functional copy of the human GAA gene
Primary Outcome Measure Information:
Title
Safety and tolerability over time
Description
Frequency of adverse events (AEs), serious adverse events (SAEs), and changes from baseline in relevant clinical laboratory tests
Time Frame
52 weeks
Title
Proportion of patients treated with GC301 who are alive
Time Frame
52 weeks
Secondary Outcome Measure Information:
Title
Proportion of patients treated w/ GC301 who were alive and free of ventilator support
Time Frame
52 weeks
Title
Changes from baseline Left Ventricular Mass (LVM) annd LVMI (LVM index)
Time Frame
26 and 52 weeks
Title
Changes from baseline creatine kinase (CK), CK-MB, Troponin I, B-Type Natriuretic Peptide (BNP)
Time Frame
26 and 52 weeks
Other Pre-specified Outcome Measures:
Title
Change from baseline glycogen content in muscle tissue
Time Frame
26 and 52 weeks
Title
Change from baseline acid alpha-glucosidase (GAA) enzyme in muscle and blood
Time Frame
26 and 52 weeks
Title
Change in patient's motor function
Description
To evaluate the changes in patient's mobility and physical ability using Hammersmith Infant Neurological Examination (HINE) scores
Time Frame
52 weeks
Title
The viral load of adeno-associated virus (AAV) vector
Description
To assess the change of AAV vector copy numbers within 52 weeks after administration.
Time Frame
At multiple time points from pre-dose through up to 1 years post-dose
10. Eligibility
Sex
All
Maximum Age & Unit of Time
6 Months
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Age < 6 months
Patient has diagnosis of infantile onset Pompe disease
The patient's legal guardian(s) must be able to understand the purpose and risks of the study and voluntarily provide signed and dated informed consent prior to any study-related procedures being performed.
Exclusion Criteria:
Left ventricle ejection fraction (LVEF) < 40%;
Patient who has AAV9 neutralizing antibody titer ≥ 1:100；
Patient who has received enzyme replacement therapy (ERT) more than twice;
Patient who has respiratory dysfunction before enrollment, including the blood oxygen (O2) saturation level < 90%, or the partial pressure of carbon dioxide (PCO2) in venous blood > 55 mmHg, or PCO2 in arterial blood > 40 mmHg;
Patient who has laboratory abnormalities of: creatinine > Upper Limit of Normal (ULN), hemoglobin < 90 g/L;
Patient with congenital organ absence;
Patient with a history of glucocorticoid allergy;
Patient who is positive for human immunodeficiency (HIV) antibody, hepatitis B surface antigen, hepatitis C antibody, or treponema pallidum antibody;
Patient who has participated in a previous gene therapy research trial;
Patient who has any concurrent clinically significant major disease or any other condition that, in the opinion of the investigator, makes the subject unsuitable for participation in the study.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
GeneCradle, Inc. China
Phone
86-13501380583
Email
ind@bj-genecradle.com
Facility Information:
Facility Name
Peking Union Medical College
City
Beijing
ZIP/Postal Code
100005
Country
China
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Juan Ding
First Name & Middle Initial & Last Name & Degree
Zhengqing Qiu
Facility Name
301 Chinese PLA General Hospital
City
Beijing
Country
China
Individual Site Status
Not yet recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Xinting Liu
First Name & Middle Initial & Last Name & Degree
Guang Yang
Facility Name
Central South University, Xiangya Hospital
City
Changsha
Country
China
Individual Site Status
Not yet recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Fang Zhou
First Name & Middle Initial & Last Name & Degree
Jing Peng
Facility Name
Zhejiang University, School of Medicine, The Children's Hospital
City
Hangzhou
Country
China
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Yujia Wang
First Name & Middle Initial & Last Name & Degree
Yiying Zhang
Facility Name
The First Affiliated Hospital of Zhengzhou University
City
Zhengzhou
Country
China
Individual Site Status
Not yet recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Jianjiang Zhang
12. IPD Sharing Statement
Learn more about this trial
Evaluation of the Safety and Efficacy of Infantile-onset Pompe Disease Gene Therapy Drug
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