drugset / Trial / NCT05778877
A Study to Evaluate the Safety, Tolerability, and Pharmacodynamics of SEL-302 in Pediatric Subjects With MMA
Phase 1/2
Withdrawn
Selecta Biosciences, Inc.
National Human Genome Research Institute (NHGRI) · collab
Non-randomizedSequentialOpen-labelTreatment
Summary
This Phase 1/2 study will evaluate the safety and pharmacodynamics (PD) of SEL-302, which consists of the gene transfer vector MMA-101 following administration of an immunomodulatory SEL-110 agent in pediatric subjects with Methylmalonyl-CoA Mutase (MMUT) MMA.
Timeline
- Start
- 2022-12-19
- Primary completion
- 2025-08
- Completion
- 2029-08
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | MMA-101 | Gene therapy (AAV / viral vector) | 1e+13 vg/kg | Intravenous |
| Subject | Sirolimus | Small molecule | 0.15 mg/kg | — |
| Subject | Sirolimus | Small molecule | 0.3 mg/kg | — |