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Individual Patient Compassionate Use of Mepsevii

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. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. Identifier: NCT03775174
Expanded Access Status : Available
First Posted : December 13, 2018
Last Update Posted : September 4, 2019
Information provided by (Responsible Party):
Ultragenyx Pharmaceutical Inc

Tracking Information
First Submitted Date December 11, 2018
First Posted Date December 13, 2018
Last Update Posted Date September 4, 2019
Descriptive Information
Brief Title Individual Patient Compassionate Use of Mepsevii
Official Title Not Provided
Brief Summary Individual patient expanded access requests may be considered for patients who have no other treatment options
Detailed Description Expanded access may provide access for treatment prior to approval by the local regulatory agency. For full details, please visit the link provided.
Study Type Expanded Access
Expanded Access Type Individual Patients
  • Mucopolysaccharidosis VII
  • Sly Syndrome
Intervention Drug: Mepsevii
Other Names:
  • UX003
  • recombinant human beta-glucuronidase
  • rhGUS
  • vestronidase alfa
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by Identifier (NCT Number) in Medline.
Recruitment Information
Expanded Access Status Available
Eligibility Criteria

Inclusion Criteria:


Exclusion Criteria:


Sexes Eligible for Study: All
Ages Child, Adult, Older Adult
Contact: Early Access 1-415-483-8800
Listed Location Countries Not Provided
Removed Location Countries  
Administrative Information
NCT Number NCT03775174
Other Study ID Numbers UX003-CU
Responsible Party Ultragenyx Pharmaceutical Inc
Study Sponsor Ultragenyx Pharmaceutical Inc
Collaborators Not Provided
Investigators Not Provided
PRS Account Ultragenyx Pharmaceutical Inc
Verification Date September 2019