Expanded Access to Mepsevii
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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. |
ClinicalTrials.gov Identifier: NCT03775174 |
Expanded Access Status :
Available
First Posted : December 13, 2018
Last Update Posted : March 3, 2021
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Condition or disease | Intervention/treatment |
---|---|
MPS VII Mucopolysaccharidosis VII Sly Syndrome | Drug: Mepsevii |
Study Type : | Expanded Access |
Expanded Access Type : | Individual Patients |
See clinical trials of the intervention/treatment in this expanded access record. |

- Drug: Mepsevii
Other Names:
- UX003
- recombinant human beta-glucuronidase
- rhGUS
- vestronidase alfa

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
Ages Eligible for Study: | Child, Adult, Older Adult |
Sexes Eligible for Study: | All |
Inclusion Criteria:
-
Exclusion Criteria:
-

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03775174
Contact: Early Access | 1-415-483-8800 | EarlyAccess@ultragenyx.com |
Responsible Party: | Ultragenyx Pharmaceutical Inc |
ClinicalTrials.gov Identifier: | NCT03775174 |
Other Study ID Numbers: |
UX003-EAP |
First Posted: | December 13, 2018 Key Record Dates |
Last Update Posted: | March 3, 2021 |
Last Verified: | March 2021 |
Expanded Access Compassionate Use |
Mucopolysaccharidoses Mucopolysaccharidosis VII Carbohydrate Metabolism, Inborn Errors Metabolism, Inborn Errors Genetic Diseases, Inborn |
Lysosomal Storage Diseases Mucinoses Connective Tissue Diseases Metabolic Diseases |