Status:
RECRUITING
A Study to Evaluate the Tolerability, Safety and Efficacy of VGM-R02b
Lead Sponsor:
Shanghai Vitalgen BioPharma Co., Ltd.
Conditions:
Glutaric Acidemia Type I
Glutaric Aciduria Type I
Eligibility:
All Genders
Up to 6 years
Phase:
PHASE1
Brief Summary
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 b...
Detailed Description
This study consists of screening period, treatment period and postoperative monitoring period and follow-up period. During the screening period (Days -28 to -1), patients whose parent(s)/legal guardia...
Eligibility Criteria
Inclusion
- Subjects must be ≤ 6 years;
- History of diagnosis of GA-I, and confirmed by gene mutation analysis with biallelic GCDH mutation;
- At the time of screening, there was one of the obvious neurological manifestations associated with the following diseases, including macrocephaly, dystonia, and motor/intellectual development Poor fertility, epilepsy, abnormal EEG;
- Those who are receiving standard treatment recommended by the guidelines and whose symptoms remain poorly controlled by the investigator;
- Plasma GA and 3-OHGA levels were higher than the normal range during screening;
Exclusion
- Participation in gene therapy or stem cell transduction therapy at any time prior to screening for this trial or participation in any other clinical trial within 3 months prior to screening;
- Recurrent seizures that are not suitable for surgery, based on Investigator judgment;
- Current severe liver or kidney or cardiovascular disease or coagulation dysfunction, autoimmune deficiency, or uncontrolled autoimmune disease or need immunosuppressive long-term treatment, poorly controlled diabetes (HBA1C ≥7% at screening) or high blood pressure;
- Active viral infection (includes HIV or serology positive for hepatitis B or C or syphilis);
- Presence or history of malignancy;
- Received systemic immunosuppressive therapy within 3 months prior to screening;
- Received vaccine within 4 weeks prior to administration or plan to receive vaccine within 1 year after administration;
- Plan to receive surgery during the study;
- Current using medications including, drugs, herbal or OTC medications that strongly inhibit or induce CYP3A4 or P-glycoprotein (P-gp), e.g., metoclopramide, grapefruit juice, ketoconazole, erythromycin;
- Abnormal brain structure, not suitable for lateral ventricle administration;
- Abnormal laboratory test results, which are judged by the investigator not suitable for surgery;
- History of systemic hypersensitivity reaction to investigational product, the excipients contained in the formulation, or prophylactic immunosuppressant;
- Contraindicated use of corticosteroids and sirolimus;
- Contraindicated with general anesthesia or sedation;
- As judged by the investigator, unable to perform lateral ventricle puncture or Ommaya capsule implantation or lumbar puncture;
- Unable to perform CT or MRI;
- Poor compliance;
- Any other situation where, judged by the investigator, the subject is not suitable for participating in this study.
Key Trial Info
Start Date :
April 29 2024
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
August 1 2026
Estimated Enrollment :
12 Patients enrolled
Trial Details
Trial ID
NCT06217861
Start Date
April 29 2024
End Date
August 1 2026
Last Update
May 17 2024
Active Locations (1)
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1
The Children's Hospital Zhejiang University Shcool of Medicine
Hangzhou, Zhejiang, China