An Open-Label Study of UX003 Recombinant Human Beta-Glucuronidase (rhGUS) Enzyme Replacement Treatment in Mucopolysaccharidosis Type 7, Sly Syndrome (MPS 7) Patients Less Than 5 Years of Age
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|ClinicalTrials.gov Identifier: NCT02418455|
Recruitment Status : Completed
First Posted : April 16, 2015
Last Update Posted : April 2, 2019
|Condition or disease||Intervention/treatment||Phase|
|Sly Syndrome MPS VII Mucopolysaccharidosis Mucopolysaccharidosis VII||Drug: UX003||Phase 2|
Expanded Access : An investigational treatment associated with this study is available outside the clinical trial. More info ...
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||8 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||An Open-label Study of UX003 rhGUS Enzyme Replacement Therapy in MPS 7 Patients Less Than 5 Years Old|
|Actual Study Start Date :||July 21, 2015|
|Actual Primary Completion Date :||March 26, 2019|
|Actual Study Completion Date :||March 26, 2019|
Experimental: 4 mg/kg of UX003
Initial treatment period 48 weeks. Continuation period up to 240 weeks.
- Percent Change From Baseline in Urinary Glycosaminoglycans (uGAG) Excretion at Week 48 [ Time Frame: Baseline (Week 0), Week 48 ]Determined by the percent reduction of uGAG excretion after 48 weeks of treatment relative to the pre-treatment baseline.
- Safety and tolerability of UX003 (severity, frequency, and number of Adverse Events and Serious Adverse Events) [ Time Frame: Initial treatment period 48 weeks. Continuation period up to 240 weeks. ]
- Change From Baseline in Growth Velocity at Week 48 [ Time Frame: Baseline (Week 0), Week 48 ]Determined by changes in growth velocity after 48 weeks of treatment relative to pre-treatment baseline and published normative data for age and gender.
- Change From Baseline in Hepatosplenomegaly at Week 48 [ Time Frame: Baseline (Week 0), Week 48 ]Determined by the volume of the liver and spleen after 48 weeks of treatment relative to pre-treatment baseline and normal values for age and gender.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02418455
|United States, District of Columbia|
|Children's National Health System|
|Washington, District of Columbia, United States, 20010|
|United States, New York|
|New York University Langone Medical Center|
|New York, New York, United States, 10038|
|United States, Utah|
|University of Utah Hospital|
|Salt Lake City, Utah, United States, 84132|
|Centro Hospitalar do Porto|
|Porto, Portugal, 4099-345|
|Hospital Universitario Virgen Del Rocio|
|Sevilla, Spain, 41013|
|Study Director:||Medical Director||Ultragenyx Pharmaceutical Inc|