Actively Recruiting
An Exploratory Clinical Trial to Evaluate the Tolerability and Safety of VGN-R08b Via Intracerebroventricular Injection 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
215 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
Type II Gaucher disease is a rare genetic disorder that affects infants, causing severe neurological and visceral problems often leading to death before age two. This trial aims to explore the safety and tolerability of a gene therapy called VGN-R08b, which is designed to address the underlying enzyme deficiency in affected patients. The study is an early-phase, open-label trial conducted at a single center, focusing on infants diagnosed with this condition. VGN-R08b is a gene therapy using an adeno-associated virus (AAV9) to deliver a functional copy of the GBA1 gene directly into the brain via intracerebroventricular injection. The study has two parts: a dose-escalation phase testing two dose levels with one patient each, followed by a dose-expansion phase including 2 to 4 additional patients. Treatment is given once, and the trial will investigate how well patients tolerate the gene therapy and monitor for side effects. Participants undergo regular assessments including monitoring for adverse events, survival rates up to 24 months of age, and changes in relevant enzyme activity and biomarkers in blood and cerebrospinal fluid over up to five years. The study includes immunogenicity testing and tracking of the gene therapy vector in the blood. Safety follow-up continues long term to assess lasting effects. Parents or guardians provide consent and help with study assessments throughout the participation period.
CONDITIONS
Brief 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 0 to 24 months
- Diagnosed with Gaucher disease confirmed by enzyme activity test and GBA1 gene mutations
- Presence of neurological signs or symptoms consistent with type II Gaucher disease
- Parent(s) or legal guardian(s) provide consent for participation
- Parent(s) or legal guardian(s) agree to comply with study requirements and provide disease information
You will not qualify if you...
- Diagnosis of other significant central nervous system diseases besides type II Gaucher disease
- Ability to walk independently
- Severe visceral symptoms posing risk or interfering with study participation
- Active infections such as HIV, hepatitis B or C, or syphilis
- Stable enzyme replacement or substrate reduction therapy less than 2 months before enrollment
- Use of strong CYP3A4 or P-glycoprotein inhibitors or inducers
- Previous gene or cell therapy
- Live vaccinations within 4 weeks prior to enrollment
- Use of systemic immunosuppressants or corticosteroids except specified topical treatments
- Anti-AAV9 neutralizing antibody titer above 1:5
- Brain MRI showing abnormalities preventing injection
- Contraindications to sedation for surgery or imaging
- Other medical conditions posing unacceptable risk or interfering with study conduct
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Up to 52 weeks
Participants receive VGN-R08b gene therapy via intracerebroventricular injection and are monitored for safety and tolerability.
Multiple visits during the first 52 weeks for treatment and safety assessments
Duration - Up to 5 years
Participants are followed for long-term safety and efficacy assessments including survival and biomarker evaluations.
Periodic visits for up to 5 years after treatment
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.
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
Frequently Asked Questions
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