Actively Recruiting
A Study to Evaluate the Tolerability, Safety and Efficacy of VGM-R02b
Led by Shanghai Vitalgen BioPharma Co., Ltd. · Updated on 2026-05-13
12
Participants Needed
1
Research Sites
169 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Phase I, open-label, single-arm, single-dose, trial of VGM-R02b (gene replacement therapy) in patients with Glutaric Acidemia Type I (GA-I) who meet enrollment criteria and are genetically confirmed by GCDH gene mutation. 1 to 3 patients aged≤ 6 years at the time of screening will be enrolled in each dose group in the dose escalation part. In the dose expansion part, the sample size will be statistically calculated and adjusted according to the efficacy and safety data in the dose escalation part.
CONDITIONS
Official Title
A Study to Evaluate the Tolerability, Safety and Efficacy of VGM-R02b
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Patients aged 6 years or younger
- History of Glutaric Acidemia Type I confirmed by biallelic GCDH gene mutation
- Presence of neurological symptoms such as macrocephaly, dystonia, poor motor or intellectual development, epilepsy, or abnormal EEG
- Currently receiving standard treatment with symptoms poorly controlled
- Plasma GA and 3-OHGA levels above normal at screening
You will not qualify if you...
- Prior gene therapy or stem cell transduction therapy or participation in another clinical trial within 3 months
- Recurrent seizures unsuitable for surgery as judged by investigator
- Severe liver, kidney, cardiovascular disease, coagulation dysfunction, autoimmune deficiency or uncontrolled autoimmune disease, need for long-term immunosuppressive treatment, poorly controlled diabetes (HbA1c 67%) or high blood pressure
- Active viral infections including HIV or positive hepatitis B, C, or syphilis serology
- History or presence of malignancy
- Systemic immunosuppressive therapy within 3 months prior to screening
- Vaccination within 4 weeks prior or planned within 1 year after administration
- Planned surgery during the study
- Current use of medications strongly affecting CYP3A4 or P-glycoprotein
- Abnormal brain structure unsuitable for lateral ventricle administration
- Abnormal lab results unsuitable for surgery as judged by investigator
- History of hypersensitivity to study drug, excipients, or immunosuppressants
- Contraindications to corticosteroids, sirolimus, general anesthesia, or sedation
- Inability to undergo lateral ventricle puncture, Ommaya capsule implantation, or lumbar puncture
- Inability to undergo CT or MRI
- Poor compliance
- Other conditions judged unsuitable by investigator
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 1 location
1
The Children's Hospital Zhejiang University Shcool of Medicine
Hangzhou, Zhejiang, China
Actively Recruiting
Research Team
X
Xianfeng Wang
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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