Glutaric Acidemia Type IPhase 1NCT06217861
A Study to Evaluate the Tolerability, Safety and Efficacy of VGM-R02b
A Phase 1 study testing VGM-R02b in people with Glutaric Acidemia Type I and Glutaric Aciduria Type I, run by Shanghai Vitalgen BioPharma Co., Ltd. at 1 site in China.
Trial 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 by GCDH gene mutation. Read more about this study
- Who is it for?
- Ages Up to 6 years
- Conditions the study is for
- Glutaric Acidemia Type I, Glutaric Aciduria Type I
- What is being tested?
- VGM-R02b
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 12 participants
- Where
- 1 siteChina
- Study dates
- 29 Apr 2024to Aug 2027
- Phase
- Phase 1
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Up to 6 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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;
Who cannot join
- 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;
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- 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.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site, 1 recruiting
- Recruiting
The Children's Hospital Zhejiang University Shcool of Medicine
Hangzhou, Zhejiang
Xianfeng Wang, 18816250806, xianfeng.wang@vitalgen.com
About this study
Official title: An Open-Label, Dose-Escalation and Dose-Expansion Phase I Clinical Study to Evaluate the Tolerability, Safety and Efficacy of VGM-R02b in Patients With Glutaric Acidemia Type I
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.
Read the 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 guardian(s) provide informed consent will complete screening procedures to determine eligibility for trial enrollment. Eligible subjects will be admitted to the clinical research center before surgical administration to complete the preoperative examination and determine the surgical plan. Ommaya fluid reservoir capsule implantation will be used in this study (if there were problems with Ommaya implantation, intra-cerebroventricular injection could also be used for drug administration). The day of administration set to be D1. Prophylactic immunosuppressive therapy including Methylprednisolone, Prednisolone and Rapamycin was initiated on D1. Then all the examinations during the 7-day postoperative observation period will be completed based on the evaluation time point specified in the Schedule of Assessments table. Subjects may be discharged 7 days after the infusion, based on Investigator judgment. During the outpatient follow-up period (up to 52 weeks after administration), subjects will return at regularly scheduled intervals for efficacy and safety assessments until the End of Trial. After the End of Trial visit, eligible patients will be asked to participate into the long-term follow up trial.
- Conditions
- Glutaric Acidemia Type I, Glutaric Aciduria Type I
- Being tested
- VGM-R02b
- Keywords
- AAV, gene therapy, ICV, GCDH
- Medical subject headings
- Glutaric Acidemia I
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
VGM-R02bBiological
Also called rAAV9-GCDH
Administered as specified in the treatment arm.
| Study group | Role | What they receive |
|---|---|---|
| VGM-R02b | Experimental | VGM-R02b is an adeno-associated viral vector 9 delivering human Glutaryl-CoA Dehydrogenase (GCDH) gene. Receives: Biological: VGM-R02b |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: up to 52 weeks
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.
Measured: up to 52 weeks
Secondary outcomes (6)
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).
Measured: up to 52 weeks
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.
Measured: up to 52 weeks
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.
Measured: up to 52 weeks
Neuroimaging.
Changes in clinically significant abnormalities on brain MRI and MRS compared to Baseline.
Measured: up to 52 weeks
Biomarkers.
Changes of C5DC GA, 3-OHGA in CSF and plasma.
Measured: up to 52 weeks
Immunogenicity.
The antibody titer and the number of subjects with positive anti-AAV9 and anti-hGCDH antibodiesin the blood and CSF.
The antibody titer and the number of subjects with positive anti-AAV9 and anti-hGCDH specific T cell immune response of peripheral blood mononuclear cells (PBMC).
Measured: up to 52 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Jan 2024
- Study start29 Apr 2024
- Primary completionAug 2027 (estimated)
- Study completionAug 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Glutaric Acidemia Type IStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 12 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Shanghai Vitalgen BioPharma Co., Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Qiang Shu, Principal investigator, The Children's Hospital of Zhejiang University School of MedicineRulai Yang, Principal investigator, The Children's Hospital of Zhejiang University School of MedicineGuanping Dong, Principal investigator, The Children's Hospital of Zhejiang University School of Medicine
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06217861
- Sponsor's protocol ID
- VGM-R02b-101
- Other IDs
- 2023LP01348
- Status last verified
- May 2026
- First submitted
- 12 Dec 2023
- Last updated
- 13 May 2026
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: IPD will be shared with other researchers when VGO-Cm01 is fully approved.
Study at a glance
From the registry record, last updated by the sponsor on 13 May 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-Label, Dose-Escalation and Dose-Expansion Phase I Clinical Study to Evaluate the Tolerability, Safety and Efficacy of VGM-R02b in Patients With Glutaric Acidemia Type I
- ClinicalTrials.gov ID
- NCT06217861
- Acronym / sponsor ID
- VGM-R02b-101
- Conditions
- Glutaric Acidemia Type I, Glutaric Aciduria Type I
- Keywords
- AAV, gene therapy, ICV, GCDH
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 12 participants (estimated)
- Lead sponsor
- Shanghai Vitalgen BioPharma Co., Ltd.Industry (pharma, biotech, devices)
- Study start
- 29 Apr 2024 (actual)
- Primary completion
- Aug 2027 (estimated)
- Study completion
- Aug 2027 (estimated)
- First posted
- 23 Jan 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
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 guardian(s) provide informed consent will complete screening procedures to determine eligibility for trial enrollment. Eligible subjects will be admitted to the clinical research center before surgical administration to complete the preoperative examination and determine the surgical plan. Ommaya fluid reservoir capsule implantation will be used in this study (if there were problems with Ommaya implantation, intra-cerebroventricular injection could also be used for drug administration). The day of administration set to be D1. Prophylactic immunosuppressive therapy including Methylprednisolone, Prednisolone and Rapamycin was initiated on D1. Then all the examinations during the 7-day postoperative observation period will be completed based on the evaluation time point specified in the Schedule of Assessments table. Subjects may be discharged 7 days after the infusion, based on Investigator judgment. During the outpatient follow-up period (up to 52 weeks after administration), subjects will return at regularly scheduled intervals for efficacy and safety assessments until the End of Trial. After the End of Trial visit, eligible patients will be asked to participate into the long-term follow up trial.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| VGM-R02b | Experimental | Biological: VGM-R02bVGM-R02b is an adeno-associated viral vector 9 delivering human Glutaryl-CoA Dehydrogenase (GCDH) gene. |
Who may qualify
- Ages
- Up to 6 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (5 listed)
- 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;
Key exclusion criteria (18 listed)
- 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;
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| VGM-R02bAlso: rAAV9-GCDHAdministered as specified in the treatment arm. | Biological | VGM-R02b |
Primary outcome
The incidence and severity of AEs and SAEs
Time frame: up to 52 weeks
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.
Evaluate changes from baseline in vital signs, and clinical laboratory results
Time frame: up to 52 weeks
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.
6 secondary outcomes
Changes in Barry Albright Dystonia Scale from baseline.
Time frame: up to 52 weeks
Changes in Peabody Developmental Motor Scale (PDMS-2) from baseline.
Time frame: up to 52 weeks
Changes in Bayley Scales of Infant and Toddler Development (BSID) or Wechsler Preschool and Primary Scale of Intelligence (WPPSI) from baseline.
Time frame: up to 52 weeks
Neuroimaging.
Time frame: up to 52 weeks
Biomarkers.
Time frame: up to 52 weeks
Immunogenicity.
Time frame: up to 52 weeks
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 13 May 2026.
- Individual participant dataSharing planned: Yes. IPD will be shared with other researchers when VGO-Cm01 is fully approved.
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RecruitingObservational study
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Urea Cycle Disorder, Organic Acidemia
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Qiang Shu principal investigator
The Children's Hospital of Zhejiang University School of Medicine
Rulai Yang principal investigator
The Children's Hospital of Zhejiang University School of Medicine
Guanping Dong principal investigator
The Children's Hospital of Zhejiang University School of Medicine
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing VGM-R02b in people with Glutaric Acidemia Type I and Glutaric Aciduria Type I, run by Shanghai Vitalgen BioPharma Co., Ltd. at 1 site in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 18 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 13 May 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Xianfeng Wang (18816250806, xianfeng.wang@vitalgen.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
The incidence and severity of AEs and SAEs (time frame: up to 52 weeks). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 13 May 2026.