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.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
16
GC301, is an adeno-associated virus 9 (AAV9) vector delivering a functional copy of the human GAA gene
Peking Union Medical College
Beijing, China
301 Chinese PLA General Hospital
Beijing, China
Central South University, Xiangya Hospital
Changsha, China
Zhejiang University, School of Medicine, The Children's Hospital
Hangzhou, China
Safety and tolerability over time
Frequency of adverse events (AEs), serious adverse events (SAEs), and changes from baseline in relevant clinical laboratory tests
Time frame: 52 weeks
Proportion of patients treated with GC301 who are alive
Time frame: 52 weeks
Proportion of patients treated w/ GC301 who were alive and free of ventilator support
Time frame: 52 weeks
Changes from baseline Left Ventricular Mass (LVM) annd LVMI (LVM index)
Time frame: 26 and 52 weeks
Changes from baseline creatine kinase (CK), CK-MB, Troponin I, B-Type Natriuretic Peptide (BNP)
Time frame: 26 and 52 weeks
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The First Affiliated Hospital of Zhengzhou University
Zhengzhou, China