drugset / Trial / NCT05815004
An Efficacy and Safety Study of AVR-RD-02 Compared to Enzyme Replacement Therapy for Treatment of Gaucher Disease Type 3
RandomizedParallel-groupQuadruple-blindTreatment
Summary
The purpose of this study is to evaluate the efficacy and safety of autologous hemotopoietic stem cell (HSC) gene therapy, AVR-RD-02, compared to enzyme replacement therapy, for the treatment of Gaucher disease Type 3 in male and female participants aged 2 to 25 years. The study will consist of 2 parts - Core (Part 1) followed by the ERT-crossover (Part 2)
Timeline
- Start
- 2023-10
- Primary completion
- 2027-12
- Completion
- 2027-12
Outcome
Outcome not reported
Stopped: “Further development of programs halted”
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | AVR-RD-02 | Unknown | — | — |
| Comparator | Imiglucerase | Protein / enzyme biologic | — | — |
| Comparator | Taliglucerase alfa | Protein / enzyme biologic | — | — |
| Comparator | velaglucerase | Unknown | — | — |