Clinical trials: Glutaric Acidemia I
- Condition Glutaric Acidemia I
- Funder type Industry (pharma, biotech, devices)
- Age group Children (under 18)
Glutaric Acidemia Type I, Glutaric Aciduria Type I
A Study to Evaluate the Tolerability, Safety and Efficacy of VGM-R02b
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.
- Trial name
- NCT06217861
- Testing
- VGM-R02b
- Where
- China
- Volunteers
- 12 people
- Sponsor
- Shanghai Vitalgen BioPharma Co., Ltd.
Phenylketonurias, PKU
Evaluation of the Express Plus Range
A prospective, open label, acceptability study to evaluate PKU, MSUD, HCU, TYR and GA express plus in the dietary management of 40 patients with IEM.
- Conditions
- Phenylketonurias, PKU, Homocystinuria, Maple Syrup Urine Disease
- Trial name
- express plus (NCT05051657)
- Testing
- PKU express plus
- Where
- 6 sites in United Kingdom
- Volunteers
- 28 people
- Sponsor
- Vitaflo International, Ltd