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

Completed · Phase 2

Conditions studied: Sly Syndrome, MPS VII, Mucopolysaccharidosis, Mucopolysaccharidosis VII

In brief

The primary objective was to evaluate the effect of UX003 treatment in pediatric MPS VII participants less than 5 years of age on safety, tolerability, and efficacy as determined by the reduction of urinary glycosaminoglycans (uGAG) excretion.

Key facts

Study ID

NCT02418455

Run by

Ultragenyx Pharmaceutical Inc

People needed

8

Starts

2015-07-21

Expected to finish

2019-03-26

Last updated by the study team

2019-10-30

Who can join

Age: 0 and older, up to 5. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Confirmed diagnosis of MPS 7 based on leukocyte or fibroblast glucuronidase enzyme assay, or genetic testing.
• Under 5 years of age at the time of informed consent.
• Written informed consent of Legally Authorized Representative after the nature of the study has been explained, and prior to any research-related procedures.

You may not qualify if…

• Undergone a successful bone marrow or stem cell transplant or has evidence of any degree of detectable chimaerism with donor cells.
• Any known hypersensitivity to rhGUS or its excipients that, in the judgment of the Investigator, places the subject at increased risk for adverse effects.
• Use of any investigational product (drug or device or combination) other than UX003 within 30 days prior to Screening, or requirement for any investigational agent prior to completion of all scheduled study assessments at any time during the study.
• Has a condition of such severity and acuity, in the opinion of the Investigator, which may not allow safe study participation. For patients with hydrops fetalis, the ongoing interventions to manage fluid balance can be continued; if the addition of enzyme replacement therapy (ERT) is considered a fluid-overload risk, the individual should be excluded.
• Has a concurrent disease or condition that, in the view of the Investigator, places the subject at high risk of poor treatment compliance or of not completing the study, or would interfere with study participation or affect safety. Since hydropic patients have a high rate of mortality, the risk of death prior to 1 year of age should not be considered sufficient to exclude the patient from the study for compliance.

Where it is running

• Children's National Health System — Washington D.C., District of Columbia, United States
• New York University Langone Medical Center — New York, New York, United States
• University of Utah Hospital — Salt Lake City, Utah, United States
• Centro Hospitalar do Porto — Porto, Portugal
• Hospital Universitario Virgen Del Rocio — Seville, Spain

Full record on ClinicalTrials.gov

Trial information comes from ClinicalTrials.gov and is refreshed daily. TrialsForMe does not provide medical care and does not run the studies it lists.