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Expanded Access to Mepsevii

Primary Purpose

MPS VII, Mucopolysaccharidosis VII, Sly Syndrome

Status
Available
Phase
Locations
Study Type
Expanded Access
Intervention
Mepsevii
Sponsored by
Ultragenyx Pharmaceutical Inc
About
Eligibility
Locations
Outcomes
Full info

About this trial

This is an expanded access trial for MPS VII focused on measuring Expanded Access, Compassionate Use

Eligibility Criteria

undefined - undefined (Child, Adult, Older Adult)All Sexes

Inclusion Criteria:

-

Exclusion Criteria:

-

Sites / Locations

    Outcomes

    Primary Outcome Measures

    Secondary Outcome Measures

    Full Information

    First Posted
    December 11, 2018
    Last Updated
    September 1, 2023
    Sponsor
    Ultragenyx Pharmaceutical Inc
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    1. Study Identification

    Unique Protocol Identification Number
    NCT03775174
    Brief Title
    Expanded Access to Mepsevii
    Study Type
    Expanded Access

    2. Study Status

    Record Verification Date
    September 2023
    Overall Recruitment Status
    Available
    Study Start Date
    undefined (undefined)
    Primary Completion Date
    undefined (undefined)
    Study Completion Date
    undefined (undefined)

    3. Sponsor/Collaborators

    Responsible Party, by Official Title
    Sponsor
    Name of the Sponsor
    Ultragenyx Pharmaceutical Inc

    4. Oversight

    5. Study Description

    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.

    6. Conditions and Keywords

    Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
    MPS VII, Mucopolysaccharidosis VII, Sly Syndrome
    Keywords
    Expanded Access, Compassionate Use

    7. Study Design

    8. Arms, Groups, and Interventions

    Intervention Type
    Drug
    Intervention Name(s)
    Mepsevii
    Other Intervention Name(s)
    UX003, recombinant human beta-glucuronidase, rhGUS, vestronidase alfa

    10. Eligibility

    Sex
    All
    Eligibility Criteria
    Inclusion Criteria: - Exclusion Criteria: -
    Central Contact Person:
    First Name & Middle Initial & Last Name or Official Title & Degree
    Early Access
    Phone
    1-415-483-8800
    Email
    EarlyAccess@ultragenyx.com

    12. IPD Sharing Statement

    Links:
    URL
    https://www.ultragenyx.com/our-purpose/supporting-access-for-patients/?#investigational-therapies
    Description
    Access to Investigational Therapies / Requesting Access
    URL
    https://www.mepsevii.com/en/
    Description
    Mepsevii Patient and Prescribing Information Page

    Learn more about this trial

    Expanded Access to Mepsevii

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