Study design & results
Available in the Gosset platform where reported.
Trial intelligence / Profile preview
Open-label UX003 Enzyme Replacement Therapy in Pediatric MPS 7 (<5 years)
2015-000104-26Phase 2
- Sponsor
- Ultragenyx Pharmaceutical Inc.
- Start
- Jul 2015
- Modality
- Replacement Enzymes → Therapeutic Enzymes → Recombinant Proteins and Enzymes
- Countries
- 1
01
Overview
Phase 2, open-label, single-arm trial of vestronidase alfa in mucopolysaccharidosis type VII, sponsored by Ultragenyx Pharmaceutical Inc. Primary endpoint: Mean percent change in urinary glycosaminoglycan (uGAG) excretion from Week 0 to Week 48.
02
Drugs
vestronidase alfa
03
Conditions
mucopolysaccharidosis type VII
Beyond the preview
Go deeper on 2015-000104-26.
Study design, results and competitive context are available in the Gosset platform where reported.
Competitive context
Available in the Gosset platform where reported.
Bring the full picture into focus.
See how Gosset can support your research on 2015-000104-26.