A Randomized, Placebo-Controlled, Blind-Start, Single-Crossover Phase 3 Study to Assess the Efficacy and Safety of UX003 rhGUS Enzyme Replacement Therapy in Patients With MPS 7

Status: Completed
Location: See all (4) locations...
Intervention Type: Drug, Other
Study Type: Interventional
Study Phase: Phase 3
SUMMARY

The Phase 3 study will use a novel randomized, intra-subject placebo-controlled, single crossover design, referred to as Blind Start, to evaluate the safety and efficacy of UX003. The Blind Start is a novel design whereby participants will be randomized to 1 of 4 groups, each representing a different treatment sequence, and will cross over to UX003 at different pre-defined time points in a blinded manner. All groups will receive a minimum of 24 weeks treatment with 4 mg/kg UX003 every other week (QOW).

Eligibility
Participation Requirements
Sex: All
Minimum Age: 5
Maximum Age: 35
Healthy Volunteers: No
View:

• Confirmed diagnosis of MPS 7 based on leukocyte or fibroblast glucuronidase enzyme assay or genetic testing.

• Elevated urinary glycosaminoglycan (uGAG) excretion at a minimum of 3-fold over the mean normal for age (at Screening).

• Apparent clinical signs of lysosomal storage disease as judged by the Investigator, including at least one of the following: enlarged liver and spleen, joint limitations, airway obstruction or pulmonary problems, limitation of mobility while still ambulatory.

• Aged 5 - 35 years, inclusive.

• Willing and able to provide written informed consent, or in the case of subjects under the age of 18 (or 16 years, depending on the region), provide written assent (if required) and written informed consent by a legally authorized representative after the nature of the study has been explained, and prior to any research-related procedures.

• Sexually active subjects must be willing to use acceptable highly effective methods of contraception while participating in the study and for 30 days following the last dose.

• Females of childbearing potential must have a negative pregnancy test at Screening and be willing to have additional pregnancy tests during the study. Females considered not of childbearing potential include those who have not experienced menarche, or have had tubal ligation at least one year prior to Screening, or who have had total hysterectomy.

• Naïve to treatment with UX003.

Locations
United States
California
Children's Hospital Oakland
Oakland
Children's Hospital of Orange County
Orange
Florida
Miami Children's Hospital
Miami
Minnesota
University of Minnesota
Minneapolis
Time Frame
Start Date: December 2014
Completion Date: May 2016
Participants
Target number of participants: 12
Treatments
Experimental: Group A: 4 mg/kg UX003
4 mg/kg UX003 QOW through Week 46
Experimental: Group B: 8 Weeks Placebo then 4 mg/kg UX003
Placebo QOW for the first 8 weeks followed by 4 mg/kg UX003 QOW through Week 46
Experimental: Group C: 16 Weeks Placebo then 4 mg/kg UX003
Placebo QOW for the first 16 weeks followed by 4 mg/kg UX003 QOW through Week 46
Experimental: Group D: 24 Weeks Placebo then 4 mg/kg UX003
Placebo QOW for the first 24 weeks followed by 4 mg/kg UX003 QOW through Week 46
Sponsors
Leads: Ultragenyx Pharmaceutical Inc

This content was sourced from clinicaltrials.gov

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