An Open-Label Study of UX003-rhGUS Enzyme Replacement Treatment in MPS 7 Patients Less Than 5 Years of Age
Active, no longer recruiting
Phase of Trial: Phase II
Latest Information Update: 10 Mar 2017
At a glance
- Drugs Vestronidase alfa (Primary)
- Indications Mucopolysaccharidosis VII
- Focus Adverse reactions; Therapeutic Use
- Sponsors Ultragenyx Pharmaceutical
- 06 Mar 2017 Planned number of patients changed from 7 to 15.
- 06 Mar 2017 Status changed from recruiting to active, no longer recruiting.
- 05 Aug 2015 According to an Ultragenyx Pharmaceuticals media release, first patient has been dosed in this trial. Interim data from this study are expected by the end of 2016. This study is also intended to enroll patients with non-immune hydrops fetalis.