An Exploratory Clinical Trial to Evaluate the Tolerability and Safety of VGN-R08b Via Intracerebroventricular Injection in Patients With Type II Gaucher Disease
Status: Recruiting
Location: See location...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Early Phase 1
SUMMARY
This exploratory trial is to prove the tolerability and safety of VGN-R08b to treat infants with type II Gaucher disease.
Eligibility
Participation Requirements
Sex: All
Maximum Age: 2
Healthy Volunteers: f
View:
• Infants with age of ≤24 months.
• Historical diagnosis of Gaucher disease confirmed by GCase enzyme activity test, and with GBA1 biallelic mutations.
• Neurological signs and/or symptoms consistent with diagnosis of GD2.
• Parent(s)/legal guardian(s) of subject must give their consent for subject to enroll in the study.
• Parent(s)/legal guardian(s) of the subject must agree to comply with the requirements of the study, including providing disease information and support disease assessment of symptoms.
Locations
Other Locations
China
Xinhua Hospital Affiliated to Shanghai Jiao Tong University School of Medicine
RECRUITING
Shanghai
Contact Information
Primary
Zhang Huiwen, Dr.
zhanghuiwen@xinhuamed.com.cn
18117165075
Time Frame
Start Date: 2023-03-01
Estimated Completion Date: 2029-02-28
Participants
Target number of participants: 6
Treatments
Experimental: type II Gaucher disease
This is a single-center, open, dose-climbing investigator-sponsored exploratory clinical study that included a dose-climbing phase and a dose-expanding phase. The sponsor plans to explore two dose levels in dose-climbing phase (one subject each cohort), then have additional 2\~4 subjects in dose-expanding phase
Related Therapeutic Areas
Sponsors
Collaborators: Shanghai Vitalgen BioPharma Co., Ltd.
Leads: Xinhua Hospital, Shanghai Jiao Tong University School of Medicine