A Study to Evaluate the Tolerability, Safety and Efficacy of VGM-R02b
Trial statusRecruiting
Trial phasePhase 1
Trial typeInterventional
Biological sexAll
AgeUp to 6
SponsorShanghai Vitalgen BioPharma Co., Ltd.
About this trial
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.
Eligibility criteria
Qualifiers
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;
Disqualifiers
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);
Trial design
Treatments tested in this trial
- VGM-R02b
Treatment groups
12 Participants
are divided into 1 treatment groupSponsors and collaborators
Source ClinicalTrials.gov