Phase
Condition
Phenylketonuria
Treatment
VGM-R02b
Clinical Study ID
Ages < 6 All Genders
Study Summary
Eligibility Criteria
Inclusion
Inclusion Criteria:
Subjects must be ≤ 6 years;
History of diagnosis of GA-I, and confirmed by gene mutation analysis with biallelicGCDH mutation;
At the time of screening, there was one of the obvious neurological manifestationsassociated with the following diseases, including macrocephaly, dystonia, andmotor/intellectual development Poor fertility, epilepsy, abnormal EEG;
Those who are receiving standard treatment recommended by the guidelines and whosesymptoms remain poorly controlled by the investigator;
Plasma GA and 3-OHGA levels were higher than the normal range during screening;
Exclusion
Exclusion Criteria:
Participation in gene therapy or stem cell transduction therapy at any time prior toscreening for this trial or participation in any other clinical trial within 3months prior to screening;
Recurrent seizures that are not suitable for surgery, based on Investigatorjudgment;
Current severe liver or kidney or cardiovascular disease or coagulation dysfunction,autoimmune deficiency, or uncontrolled autoimmune disease or need immunosuppressivelong-term treatment, poorly controlled diabetes (HBA1C ≥7% at screening) or highblood pressure;
Active viral infection (includes HIV or serology positive for hepatitis B or C orsyphilis);
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 vaccinewithin 1 year after administration;
Plan to receive surgery during the study;
Current using medications including, drugs, herbal or OTC medications that stronglyinhibit or induce CYP3A4 or P-glycoprotein (P-gp), e.g., metoclopramide, grapefruitjuice, ketoconazole, erythromycin;
Abnormal brain structure, not suitable for lateral ventricle administration;
Abnormal laboratory test results, which are judged by the investigator not suitablefor surgery;
History of systemic hypersensitivity reaction to investigational product, theexcipients 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 orOmmaya 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 suitablefor participating in this study.
Study Design
Study Description
Connect with a study center
Shanghai Vitalgen Biopharma Co.,Ltd.
Shanghai, Shanghai
ChinaSite Not Available
The Children's Hospital Zhejiang University Shcool of Medicine
Hangzhou, Zhejiang
ChinaActive - Recruiting
Not the study for you?
Let us help you find the best match. Sign up as a volunteer and receive email notifications when clinical trials are posted in the medical category of interest to you.