NCT02097251

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.

Trial Health

55
Monitor

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Geographic Reach
1 country

1 active site

Status
unknown

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

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Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

March 24, 2014

Completed
3 days until next milestone

First Posted

Study publicly available on registry

March 27, 2014

Completed
Last Updated

February 6, 2017

Status Verified

February 1, 2017

First QC Date

March 24, 2014

Last Update Submit

February 2, 2017

Conditions

Mucopolysaccharidosis Type 7

Keywords

Mucopolysaccharidosis Type 7Sly syndromeUX003Enzyme Replacement Therapyrare diseaseLysosomal Storage Diseasemetabolic disorder

Interventions

UX003DRUG

Open Label

Also known as: recombinant human beta glucuronidase, rhGUS

Eligibility Criteria

Sexmale
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • 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)

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Cohen Children's Medical Center

New Hyde Park, New York, 11040, United States

Location

MeSH Terms

Conditions

Mucopolysaccharidosis VIIRare DiseasesLysosomal Storage DiseasesMetabolic Diseases

Interventions

vestronidase alfa

Condition Hierarchy (Ancestors)

MucopolysaccharidosesCarbohydrate Metabolism, Inborn ErrorsMetabolism, Inborn ErrorsGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesMucinosesConnective Tissue DiseasesSkin and Connective Tissue DiseasesNutritional and Metabolic DiseasesDisease AttributesPathologic ProcessesPathological Conditions, Signs and Symptoms

Study Design

Study Type
expanded access
Sponsor Type
OTHER
Responsible Party
SPONSOR INVESTIGATOR
PI Title
Chief of Medical Genetics at Cohen Children's Medical Center

Study Record Dates

First Submitted

March 24, 2014

First Posted

March 27, 2014

Last Updated

February 6, 2017

Record last verified: 2017-02

Locations

US(1)