A Long-Term Open-Label Treatment and Extension Study of UX003 rhGUS Enzyme Replacement Therapy in Subjects With MPS 7
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT02432144|
Recruitment Status : Completed
First Posted : May 1, 2015
Last Update Posted : January 18, 2019
|Condition or disease||Intervention/treatment||Phase|
|Sly Syndrome MPS VII Mucopolysaccharidosis Mucopolysaccharidosis VII||Drug: UX003||Phase 3|
Expanded Access : An investigational treatment associated with this study is available outside the clinical trial. More info ...
Mucopolysaccharidosis type 7 (MPS 7, Sly syndrome) is an ultra-rare (< 100 cases currently identified worldwide), chronically debilitating and life threatening lysosomal storage disease. It is characterized by a deficiency of the lysosomal enzyme beta-glucuronidase (GUS), required for degradation of the glycosaminoglycans (GAGs): dermatan sulfate (DS), chondroitin-6-sulfate (CS) and heparan sulfate (HS). The GUS deficiency results in lysosomal accumulation of GAGs in multiple tissues and organs throughout the body and numerous clinical signs and symptoms as a result of tissue damage and organ dysfunction. There are currently no approved treatments for MPS 7.
UX003 (recombinant human beta glucuronidase, rhGUS) is intended as a long-term enzyme replacement therapy (ERT) for the treatment of MPS 7 via intravenous (IV) administration. Ultragenyx is conducting this treatment and extension study to assess the long-term safety and efficacy of UX003 treatment in subjects with MPS 7. Subjects with MPS 7 who are UX003 treatment-naïve or have been previously enrolled and treated with UX003 in other clinical studies or programs are eligible for enrollment.
The study will continue for up to 144 weeks or until one of the following occurs: the subject withdraws consent and discontinues from the study, the subject is discontinued from the study at the discretion of the Investigator or Ultragenyx, or the study is terminated.
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||12 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||A Long-Term Open-Label Treatment and Extension Study of UX003 rhGUS Enzyme Replacement Therapy in Subjects With MPS 7|
|Actual Study Start Date :||November 10, 2015|
|Actual Primary Completion Date :||January 14, 2019|
|Actual Study Completion Date :||January 14, 2019|
Experimental: 4 mg/kg of UX003
All subjects will receive 4 mg/kg UX003 every other week (QOW) unless data from prior studies define a different dose either for that subject or the use of UX003 in general.
UX003 is a sterile liquid buffered saline formulation of rhGUS that contains enzyme at a concentration of 2 mg/mL filled to allow the withdrawal of a 5.0 mL deliverable volume and supplied in a 10 mL glass vial. UX003 will be administered QOW by slow intravenous (IV) infusion over approximately 4 hours. Infusions will be administered on a rate schedule involving a slower infusion rate initially followed by an increase in rate to minimize the potential for infusion-associated reactions (IARs); the infusion rate may be slowed to manage or reduce IARs.
- Number of Participants with Adverse Events (AEs), Treatment-Related AEs, and Serious Adverse Events (SAEs) [ Time Frame: 144 weeks ]
- Percent Reduction From Baseline at Week 144 in uGAG Excretion [ Time Frame: 144 weeks ]First morning void urine will be evaluated for uGAG concentration and normalized to urinary creatinine concentration.
- Change From Baseline at Week 144 in uGAG Excretion [ Time Frame: 144 weeks ]First morning void urine will be evaluated for uGAG concentration and normalized to urinary creatinine concentration.
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): NCT02432144
|United States, California|
|Children's Hospital Oakland|
|Oakland, California, United States, 94609|
|Children's Hospital of Orange County|
|Orange, California, United States, 92868|
|United States, Minnesota|
|University of Minnesota|
|Minneapolis, Minnesota, United States, 55455|
|United States, Texas|
|Texas Children's Hospital|
|Houston, Texas, United States, 77030|
|Hospital Infantil Candido Fontoura Sao Paulo|
|Sao Paulo, Brazil|
|Centenario Hospital Miguel Hidalgo, Pediatrics|
|Aguascalientes, Mexico, 20230|
|Unidade de Doenças Metabólicas - Centro Hospitalar do Porto|
|Porto, Portugal, 4099-001|
|Study Director:||Medical Director||Ultragenyx Pharmaceuticals, Inc.|