Actively Recruiting
An Exploratory Clinical Trial of VGN-R08b in Patients With Type II Gaucher Disease
Led by Xinhua Hospital, Shanghai Jiao Tong University School of Medicine · Updated on 2024-02-22
6
Participants Needed
1
Research Sites
313 weeks
Total Duration
On this page
Sponsors
X
Xinhua Hospital, Shanghai Jiao Tong University School of Medicine
Lead Sponsor
S
Shanghai Vitalgen BioPharma Co., Ltd.
Collaborating Sponsor
AI-Summary
What this Trial Is About
This exploratory trial is to prove the tolerability and safety of VGN-R08b to treat infants with type II Gaucher disease.
CONDITIONS
Official Title
An Exploratory Clinical Trial of VGN-R08b in Patients With Type II Gaucher Disease
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Infants aged 24 months or younger
- Confirmed Gaucher disease diagnosis by GCase enzyme activity test with GBA1 biallelic mutations
- Presence of neurological signs or symptoms consistent with type II Gaucher disease
- Parent or legal guardian consent for study enrollment
- Parent or legal guardian agreement to provide disease information and assist with symptom assessments
You will not qualify if you...
- Diagnosis of other significant central nervous system diseases aside from type II Gaucher disease
- Ability to walk independently
- Severe visceral symptoms posing unacceptable risk or study interference as judged by the investigator
- Active infections including HIV, HBV, HCV, or syphilis
- Stable treatment with enzyme replacement, substrate reduction, or ambroxol for less than 2 months before enrollment
- Use of strong cytochrome CYP3A4 or P-glycoprotein inhibitors or inducers
- Prior gene or cell therapy
- Live vaccinations within the previous 4 weeks
- Use of systemic immunosuppressants or corticosteroids excluding topical dermatological treatments
- Presence of anti-AAV9 neutralizing antibodies above a specified level
- Brain MRI abnormalities preventing intracisternal injection
- Contraindications to sedation for surgery or imaging
- Other significant medical conditions posing unacceptable risks or interfering with study participation
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 1 location
1
Xinhua Hospital Affiliated to Shanghai Jiao Tong University School of Medicine
Shanghai, Shanghai Municipality, China, 200097
Actively Recruiting
Research Team
Z
Zhang Huiwen, Dr.
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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