Mucopolysaccharidosis Type 7 Clinical Trial
Official title:
An Open-Label Phase 1/2 Study to Assess the Safety, Efficacy and Dose of UX003 rhGUS Enzyme Replacement Therapy in Patients With Mucopolysaccharidosis Type 7 (MPS 7)
UX003-CL201 is an open-label Phase 1/2 study to assess the safety, efficacy, and dose of
UX003 in MPS 7 patients via intravenous (IV) administration every other week (QOW) for 36
weeks with up to an additional 36 weeks from the optional continuation period. Up to 5
participants, who are between 5 and 30 years of age inclusive, will be enrolled and treated
with UX003.
The initial 12-week treatment period will be followed by a 24-week forced dose titration
period to assess the optimal dose. Participants who complete both the initial treatment and
forced dose titration periods will continue treatment in a 36- week continuation period.
n/a
| Status | Clinical Trial | Phase | |
|---|---|---|---|
| Enrolling by invitation |
NCT03655223 -
Early Check: Expanded Screening in Newborns
|
||
| No longer available |
NCT02097251 -
An Open-Label Treatment Protocol With UX003 rhGUS Enzyme Replacement Therapy for an Advanced Stage MPS 7 Patient
|
N/A |