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Trial record 30 of 150 for:    mucopolysaccharidosis

An Open-Label Treatment Protocol With UX003 rhGUS Enzyme Replacement Therapy for an Advanced Stage MPS 7 Patient

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ClinicalTrials.gov Identifier: NCT02097251
Expanded Access Status : No longer available
First Posted : March 27, 2014
Last Update Posted : February 6, 2017
Sponsor:
Collaborator:
Ultragenyx Pharmaceutical Inc
Information provided by (Responsible Party):
Joyce Fox, Northwell Health

Brief Summary:
Emergency access granted to treat a single patient with advanced Mucopolysaccharidosis Type 7 with UX003 Recombinant Human Beta-glucuronidase (rhGUS) Enzyme Replacement Therapy via IV administration every other week (QOW) for up to 144 weeks.

Condition or disease Intervention/treatment
Mucopolysaccharidosis Type 7 Drug: UX003

Study Type : Expanded Access
Official Title: An Open-Label Treatment Protocol With UX003 Recombinant Human Beta-glucuronidase (rhGUS) Enzyme Replacement Therapy for an Advanced Stage Mucopolysaccharidosis Type 7 (MPS 7) Patient



Intervention Details:
  • Drug: UX003
    Open Label
    Other Names:
    • recombinant human beta glucuronidase
    • rhGUS

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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   Male
Criteria

Inclusion Criteria:

  • 1 specific patient
  • Confirmed diagnosis of MPS 7 based on leukocyte or fibroblast glucuronidase enzyme assay or genetic testing confirming diagnosis.
  • Written informed consent of parent(s) or legal guardian(s)

Information from the National Library of Medicine

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): NCT02097251


Locations
United States, New York
Cohen Children's Medical Center
New Hyde Park, New York, United States, 11040
Sponsors and Collaborators
Joyce Fox
Ultragenyx Pharmaceutical Inc

Responsible Party: Joyce Fox, Chief of Medical Genetics at Cohen Children's Medical Center, Northwell Health
ClinicalTrials.gov Identifier: NCT02097251     History of Changes
Other Study ID Numbers: 13-606A
First Posted: March 27, 2014    Key Record Dates
Last Update Posted: February 6, 2017
Last Verified: February 2017

Keywords provided by Joyce Fox, Northwell Health:
Mucopolysaccharidosis Type 7
Sly syndrome
UX003
Enzyme Replacement Therapy
rare disease
Lysosomal Storage Disease
metabolic disorder

Additional relevant MeSH terms:
Mucopolysaccharidoses
Carbohydrate Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Lysosomal Storage Diseases
Mucinoses
Connective Tissue Diseases
Metabolic Diseases