Pompe disease
Conditions
Interventions
Experimental group:GC801 intravenous injection
Sponsors
The Seventh Medical Center of Chinese PLA General Hospital
Eligibility
Sex/Gender
All
Age
No minimum to 8 Years
Inclusion criteria
Inclusion criteria: 1. Patients with Pompe disease aged <=8 years, regardless of gender; 2. Serum anti-AAV9 binding antibody titer: 1:1000 to 1:10000 (including the cutoff values); 3. Immunoglobulin G (IgG) level within the normal range at the time of treatment; 4. The subject’s guardian is able to understand and comply with the requirements and procedures of the study protocol, and voluntarily participates and provides written informed consent.
Exclusion criteria
Exclusion criteria: 1. History or family history of primary or secondary immunodeficiency. 2. Hypersensitivity to streptococcal or protease drugs, hypersensitivity to the active ingredient/excipients of the investigational product, or history of severe allergic reaction/anaphylactic shock. 3. Clinically significant abnormalities in hematology or serum biochemistry parameters at screening. 4. Severe infection (requiring systemic anti-infective treatment) or other clinically significant disease that has not fully resolved and may interfere with the assessment of the investigational product within 4 weeks prior to enrollment. 5. Vaccination within 2 weeks prior to enrollment. 6. Any other conditions deemed inappropriate by the investigator for participation in the study.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Adverse Events (AEs);Laboratory examination;Vital sign parameters (blood pressure, heart rate, respiratory rate, body temperature);Anti-AAV9 binding antibodies; | — |
Secondary
| Measure | Time frame |
|---|---|
| Serum total IgG; | — |
Countries
China
Contacts
Public ContactZhichun Feng
The Seventh Medical Center of Chinese PLA General Hospital
Outcome results
None listed