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
This trial does not accept healthy volunteersQualifiers
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
Design model
Single group
Treatments tested in this trial
VGM-R02b
Biological/VaccineAdministered as specified in the treatment arm.
Treatment groups
12 Participants
are divided into 1 treatment groupGroup A: VGM-R02bExperimental treatment 1 intervention
Trial outcomes
Primary outcomes
1
The incidence and severity of AEs and SAEs
An AE is any untoward medical occurrence (eg any unfavorable and unintended sign \[including abnormal laboratory findings\], symptom or disease) in a clinical investigation participant after providing written informed consent for participation in the study. Therefore, an AE may or may not be temporally or causally associated with the use of a medicinal (investigational) product.
Time frame
up to 52 weeks2
Evaluate changes from baseline in vital signs, and clinical laboratory results
Clinically significant abnormal laboratory values or test results must be identified through a review of values outside of normal ranges/clinically notable ranges, significant changes from baseline or the previous visit, or values which are considered to be non-typical in participant with the underlying disease.
Time frame
up to 52 weeksSecondary outcomes
1
Changes in Barry Albright Dystonia Scale from baseline.
The BAD Scale is a 5-point, criterion-based, ordinal scale designed to assess dystonia in eight body regions: eyes, mouth, neck, trunk, and the four extremities. Raters score dystonia as none (0), slight (1), mild (2), moderate (3), or severe (4).
Time frame
up to 52 weeks2
Changes in Peabody Developmental Motor Scale (PDMS-2) from baseline.
The Peabody Developmental Motor Scales, a norm referenced tool commonly used to assess infants' fine and gross motor development, also is widely used for children of preschool age.
Time frame
up to 52 weeks3
Changes in Bayley Scales of Infant and Toddler Development (BSID) or Wechsler Preschool and Primary Scale of Intelligence (WPPSI) from baseline.
BSID is an extensive formal developmental assessment tool for diagnosing developmental delays in early childhood, this will be used in 1\~42 month patients. The WPPSI uses subtests to determine a child's Verbal and Performance IQ scores as well as the processing speed for children ages over 42 months.
Time frame
up to 52 weeks4
Neuroimaging.
Changes in clinically significant abnormalities on brain MRI and MRS compared to Baseline.
Time frame
up to 52 weeksSponsors and contacts
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Source ClinicalTrials.gov