RECRUITING

Mucopolysaccharidosis VII Disease Monitoring Program

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Conditions

Mucopolysaccharidosis VIIMPS VIIMPS 7Sly SyndromeUX003Mepseviivestronidase alfa-vjbkvestronidase alfarecombinant human beta-glucuronidase

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

The objectives of this study are to characterize MPS VII disease presentation and progression and assess long-term effectiveness and safety, including hypersensitivity reactions and immunogenicity of vestronidase alfa.

Official Title

Mucopolysaccharidosis VII Disease Monitoring Program (MPS VII DMP)

Quick Facts

Study Start:2018-01-29
Study Completion:2033-05
Study Type:Not specified
Phase:Not Applicable
Enrollment:Not specified
Status:RECRUITING

Study ID

NCT03604835

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:Not specified
Sexes Eligible for Study:ALL
Accepts Healthy Volunteers:No
Standard Ages:CHILD, ADULT, OLDER_ADULT
Inclusion CriteriaExclusion Criteria
  1. * Diagnosis of MPS VII based on laboratory diagnosis, including either enzymatic or mutation analysis.
  2. * Willing and able to provide written informed consent or, in the case of patients under the age of 18 (or below adult ages as defined by local laws and regulations) or patients \>18 years of age who have cognitive deficiencies, provide written assent (if required) and written informed consent by a legally authorized representative after the nature of the DMP has been explained, and prior to any research-related procedures.
  3. * Willing to comply with DMP visit schedule.
  1. * Concurrent participation in other pharmaceutical company-sponsored interventional clinical trial unless approved by Ultragenyx.

Contacts and Locations

Study Contact

Patients Contact: Trial Recruitment
CONTACT
1-888-756-8657
trialrecruitment@ultragenyx.com
HCPs Contact: Medical Information
CONTACT
1-888-756-8657
medinfo@ultragenyx.com

Principal Investigator

Medical Director
STUDY_DIRECTOR
Ultragenyx Pharmaceuticals Inc.

Study Locations (Sites)

Children's Hospital of Orange County
Orange, California, 92868
United States
Children's National Health System
Washington, District of Columbia, 20010
United States
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611
United States
University of Michigan
Ann Arbor, Michigan, 48109
United States
New York University Langone Medical Center
New York, New York, 10016
United States
University of Utah Medical Center
Salt Lake City, Utah, 84112
United States
Seattle Children's Hospital
Seattle, Washington, 98105
United States

Collaborators and Investigators

Sponsor: Ultragenyx Pharmaceutical Inc

  • Medical Director, STUDY_DIRECTOR, Ultragenyx Pharmaceuticals Inc.

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2018-01-29
Study Completion Date2033-05

Study Record Updates

Study Start Date2018-01-29
Study Completion Date2033-05

Terms related to this study

Keywords Provided by Researchers

  • UX003
  • Mepsevii
  • vestronidase alfa-vjbk
  • vestronidase alfa
  • recombinant human beta-glucuronidase

Additional Relevant MeSH Terms

  • Mucopolysaccharidosis VII
  • MPS VII
  • MPS 7
  • Sly Syndrome