Clinical Trial: An Open-Label Phase 1/2 Study to Assess the Safety, Efficacy and Dose of Study Drug UX003 Recombinant Human Beta-glucuronidase (rhGUS) Enzyme Replacement Therapy in Patients With MPS 7

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

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)

Brief Summary:

UX003-CL201 is an open-label Phase 1/2 study to assess the safety, efficacy, and dose of UX003 in MPS 7 subjects via IV administration every other week (QOW) for 36 weeks with up to an additional 36 weeks from the optional continuation period. Up to 5 subjects, 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. Subjects who complete both the initial treatment and forced dose titration periods will continue treatment in a 36 week continuation period


Detailed Summary:
Sponsor: Ultragenyx Pharmaceutical Inc

Current Primary Outcome:

  • Safety and tolerability of UX003 as measured by severity and number of Adverse Events (AEs) [ Time Frame: 36 weeks ]
  • Efficacy as determined by reduction of total urinary glycosaminoglycan (uGAG) excretion [ Time Frame: 36 weeks ]


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Walking capacity as measured by the 6 Minute Walk Test (6MWT). [ Time Frame: 36 weeks ]
  • Stair climbing capacity as measured by the 3 Minute Stair Climb Test [ Time Frame: 36 weeks ]
  • Pulmonary function as measured by Forced Vital Capacity(FVC), Forced Expiratory Volume (FEV1) and Maximal Voluntary Ventilation (MVV1) [ Time Frame: 36 weeks ]
  • Height and weight growth velocity compared to estimated pretreatment growth rate velocity from medical records (prepubertal pediatric patients only). [ Time Frame: 36 weeks ]
  • Shoulder range of motion as measured by goniometry. [ Time Frame: 36 wks ]
  • Acceptable dose as determined by total uGAG excretion using a forced dose titration regimen [ Time Frame: 36 weeks ]


Original Secondary Outcome: Same as current

Information By: Ultragenyx Pharmaceutical Inc

Dates:
Date Received: May 8, 2013
Date Started: October 2013
Date Completion:
Last Updated: May 8, 2017
Last Verified: May 2017