Mucopolysaccharidosis VII Disease Monitoring Program

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.

Conditions

Mucopolysaccharidosis VII, MPS VII, MPS 7, Sly Syndrome

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Study Overview

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Study Details

Study overview

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.

Mucopolysaccharidosis VII Disease Monitoring Program (MPS VII DMP)

Mucopolysaccharidosis VII Disease Monitoring Program

Condition
Mucopolysaccharidosis VII
Intervention / Treatment

-

Contacts and Locations

Orange

Children's Hospital of Orange County, Orange, California, United States, 92868

Washington

Children's National Health System, Washington, District of Columbia, United States, 20010

Chicago

Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, Illinois, United States, 60611

Ann Arbor

University of Michigan, Ann Arbor, Michigan, United States, 48109

New York

New York University Langone Medical Center, New York, New York, United States, 10016

Salt Lake City

University of Utah Medical Center, Salt Lake City, Utah, United States, 84112

Seattle

Seattle Children's Hospital, Seattle, Washington, United States, 98105

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.

For general information about clinical research, read Learn About Studies.

Eligibility Criteria

  • * Diagnosis of MPS VII based on laboratory diagnosis, including either enzymatic or mutation analysis.
  • * 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.
  • * Willing to comply with DMP visit schedule.
  • * Concurrent participation in other pharmaceutical company-sponsored interventional clinical trial unless approved by Ultragenyx.

Ages Eligible for Study

to

Sexes Eligible for Study

ALL

Accepts Healthy Volunteers

No

Collaborators and Investigators

Ultragenyx Pharmaceutical Inc,

Medical Director, STUDY_DIRECTOR, Ultragenyx Pharmaceuticals Inc.

Study Record Dates

2033-05