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



Status:Completed
Conditions:Metabolic
Therapuetic Areas:Pharmacology / Toxicology
Healthy:No
Age Range:Any - 5
Updated:4/4/2019
Start Date:July 21, 2015
End Date:March 26, 2019

Use our guide to learn which trials are right for you!

An Open-label Study of UX003 rhGUS Enzyme Replacement Therapy in MPS 7 Patients Less Than 5 Years Old

UX003-CL203 is an open-label, multi-center, Phase 2 study to assess the safety and efficacy
of UX003 in pediatric MPS 7 subjects.


Inclusion Criteria:

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

2. Under 5 years of age at the time of informed consent.

3. Written informed consent of Legally Authorized Representative after the nature of the
study has been explained, and prior to any research-related procedures.

Exclusion Criteria:

1. Undergone a successful bone marrow or stem cell transplant or has evidence of any
degree of detectable chimaerism with donor cells.

2. Any known hypersensitivity to rhGUS or its excipients that, in the judgment of the
Investigator, places the subject at increased risk for adverse effects.

3. 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.

4. 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.

5. 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.
We found this trial at
4
sites
Washington, District of Columbia 20010
?
mi
from
Washington,
Click here to add this to my saved trials
550 1st Ave
New York, New York 10016
(212) 263-7300
Phone: 212-263-7744
New York University Langone Medical Center NYU NYU Langone Medical Center, a world-class, patient-centered, integrated,...
?
mi
from
New York, NY
Click here to add this to my saved trials
?
mi
from
Porto,
Click here to add this to my saved trials
Salt Lake City, Utah 84132
Phone: 801-587-3605
?
mi
from
Salt Lake City, UT
Click here to add this to my saved trials