An Open-Label Treatment Protocol With UX003 rhGUS Enzyme... | NCT02097251

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

NCT02097251

Sponsor

Joyce Fox

Status

No longer available

Last Update Posted

Feb 6, 2017Estimated

Enrollment

Not provided

Phase

Not provided

Conditions

Mucopolysaccharidosis Type 7

Interventions

UX003

Countries

United States

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Results Section

No data available

No data is available for this block.

Annotation Section

No data available

No data is available for this block.

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Version Holder

Status Module

Record Verification Date

Feb 2017

Overall Recruitment Status or Expanded Access Status

No longer available

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

Not provided

Expanded Access Info

Not provided

Start Date

Not provided

Primary Completion Date

Not provided

Completion Date

Not provided

First Submitted Date

Mar 24, 2014

First Submission Date that Met QC Criteria

Mar 24, 2014

First Posted Date

Mar 27, 2014Estimated

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Feb 2, 2017

Last Update Posted Date

Feb 6, 2017Estimated

Name	Class
Ultragenyx Pharmaceutical Inc	INDUSTRY

Description Module

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.

Detailed Description

Not provided

Design Module

Study Type

Expanded Access

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Not provided

Target Follow-Up Duration

Not provided

Phases

Not provided

Interventional Study Design

No data available

No data is available for this block.

Biospecimen

No data available

No data is available for this block.

Enrollment

Not provided

Name	Type	Description	Arm Group Labels	Other Names
UX003	Drug	Open Label		recombinant human beta glucuronidase rhGUS

Eligibility Module

Eligibility 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)

Accepts Healthy Volunteers

Not provided

Sex

Male

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

Not provided

Maximum Age

Not provided

Standard Ages

ChildAdultOlder Adult

Study Population

Not provided

Sampling Method

Not provided

Facility	Status	City	State	ZIP	Country	Contacts
Cohen Children's Medical Center		New Hyde Park	New York	11040	United States

Condition Browse Module

MeSH Terms

ID	Term
D016538	Mucopolysaccharidosis VII
D035583	Rare Diseases
D016464	Lysosomal Storage Diseases
D008659	Metabolic Diseases

Ancestor Terms

ID	Term
D009083	Mucopolysaccharidoses
D002239	Carbohydrate Metabolism, Inborn Errors
D008661	Metabolism, Inborn Errors
D030342	Genetic Diseases, Inborn

Browse Leaves

Not provided

Browse Branches

Not provided