Expanded Access to Mepsevii | NCT03775174

Studies
Expanded Access to Mepsevii

NCT03775174

Sponsor

Ultragenyx Pharmaceutical Inc

Status

Available

Last Update Posted

Apr 7, 2026Actual

Enrollment

Not provided

Phase

Not provided

Conditions

MPS VII
Mucopolysaccharidosis VII
Sly Syndrome

Interventions

Mepsevii

Countries

Not provided

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

Apr 2026

Overall Recruitment Status or Expanded Access Status

Available

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

Not provided

Expanded Access Info

Start Date

Not provided

Primary Completion Date

Not provided

Completion Date

Not provided

First Submitted Date

Dec 11, 2018

First Submission Date that Met QC Criteria

Dec 11, 2018

First Posted Date

Dec 13, 2018Actual

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

Apr 6, 2026

Last Update Posted Date

Apr 7, 2026Actual

Description Module

Brief Summary

Individual patient expanded access requests may be considered for patients who have no other treatment options

Detailed Description

Expanded access may be available outside of the US in countries prior to approval by the local regulatory agencies. For full details, please visit the links provided below.

Design Module

Study Type

Expanded Access

Number of References to an Expanded Access Study

Expanded Access Types

Individual Patients

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
Mepsevii	Drug			UX003 recombinant human beta-glucuronidase rhGUS vestronidase alfa

Eligibility Module

Eligibility Criteria

Inclusion Criteria:

Exclusion Criteria:

Accepts Healthy Volunteers

Not provided

Sex

All

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

Name	Role	Phone	Extension	Email
Early Access	Contact	1-415-483-8800		EarlyAccess@ultragenyx.com

References Module

Citations

Not provided

Label	URL
Access to Investigational Therapies / Requesting Access	View source
Mepsevii Patient and Prescribing Information Page	View source

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