A Study to Evaluate the Tolerability, Safety and Efficacy of VGM-R02b

Recruiting now · Phase 1

Conditions studied: Glutaric Acidemia Type I, Glutaric Aciduria Type I

In brief

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.

Key facts

Study ID

NCT06217861

Run by

Shanghai Vitalgen BioPharma Co., Ltd.

People needed

12

Starts

2024-04-29

Expected to finish

2027-08-01

Last updated by the study team

2026-05-13

Who can join

Age: any, up to 6. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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;

You may not qualify if…

• 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.

Where it is running

• The Children's Hospital Zhejiang University Shcool of Medicine — Hangzhou, Zhejiang, China (enrolling)

Full record on ClinicalTrials.gov

Trial information comes from ClinicalTrials.gov and is refreshed daily. TrialsForMe does not provide medical care and does not run the studies it lists.