Study design & results
Available in the Gosset platform where reported.
Trial intelligence / Profile preview
Phase 2 open-label UX003 enzyme replacement therapy in Japanese patients with mucopolysaccharidosis type VII
jRCT2091220416Phase 2Completed
- Sponsor
- Osaka Metropolitan University Hospital
- Start
- Mar 2019
- 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 Osaka Metropolitan University Hospital. Primary endpoint: Reduction from baseline in urinary glycosaminoglycans at week 24. Started Mar 2019; status: completed.
02
Drugs
vestronidase alfa
03
Conditions
mucopolysaccharidosis type VII
Beyond the preview
Go deeper on jRCT2091220416.
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 jRCT2091220416.