BMN 110 US Expanded Access Program

March 31, 2014 updated by: BioMarin Pharmaceutical

A Multicenter, Open-label BMN 110 US Expanded Access Program (BMN 110 US EAP) to Provide BMN 110 to Patients Diagnosed With MPS IVA

The Expanded Access Program (EAP) is an open-label, multicenter program to:

  1. Provide patients who have been diagnosed with Mucopolysaccharidosis IVA (MPS IVA) access to BMN 110 until commercial product is available
  2. Collect additional information on the safety and tolerability of BMN 110 administration in patients with MPS IVA

Patients enrolled in the EAP will receive 2.0 mg/kg intravenous infusions of BMN 110 every week during the program.

Study Overview

Status

Approved for marketing

Conditions

Intervention / Treatment

Study Type

Expanded Access

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Puerto Rico
      • Santurce, Puerto Rico
  • United States
    • Alabama
      • Birmingham, Alabama, United States, 35294
    • Arkansas
      • Little Rock, Arkansas, United States, 72202
    • California
      • Oakland, California, United States, 94609
      • Orange, California, United States
    • Colorado
      • Aurora, Colorado, United States
    • District of Columbia
      • Washington, District of Columbia, United States, 20010
    • Florida
      • Hollywood, Florida, United States, 33021
      • Miami, Florida, United States
    • Georgia
      • Atlanta, Georgia, United States, 30033
    • Illinois
      • Chicago, Illinois, United States, 60611
    • Kentucky
      • Louisville, Kentucky, United States
    • Louisiana
      • New Orleans, Louisiana, United States
    • Minnesota
      • Minneapolis, Minnesota, United States, 55404
    • New Jersey
      • Paterson, New Jersey, United States
    • New York
      • Manhasset, New York, United States, 11030
      • New York, New York, United States
    • Oregon
      • Portland, Oregon, United States, 97239
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States
      • Pittsburgh, Pennsylvania, United States, 15224
    • Tennessee
      • Nashville, Tennessee, United States
    • Texas
      • Houston, Texas, United States
    • Utah
      • Salt Lake City, Utah, United States, 84113
    • Washington
      • Seattle, Washington, United States
      • Tacoma, Washington, United States, 98405

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

N/A

Genders Eligible for Study

All

Description

Inclusion Criteria:

  • Diagnosed with MPS IVA as confirmed by either N-acetylgalactosamine-6-sulfatase (GALNS) enzymatic test (GALNS activity in affected range, beta-galactosidase and a second lysosomal sulfatase activity within normal range) or molecular diagnostic test (two mutations in GALNS identified that have previously been associated with an enzyme defect).
  • Willing and able to provide written, signed informed consent, or in the case of patients under the age of 18, provide written assent (as required by the IRB) and written informed consent by a legally authorized representative after the nature of the program has been explained, and prior to any program assessments or evaluations.
  • Sexually active patients must be willing to use an acceptable method of contraception while participating in the program.
  • Females of childbearing potential must have a negative pregnancy test at Baseline and be willing to have additional pregnancy tests during the program.
  • Willing and able to comply with all program procedures.

Exclusion Criteria:

  • Pregnant or breastfeeding at Baseline or planning to become pregnant (self or partner) at any time during the program. Patients who become pregnant during the program will be discontinued from the program.
  • Currently enrolled in an ongoing clinical study of BMN 110.
  • Discontinued from a BMN 110 clinical study secondary to a safety-related event.
  • Use of any investigational product (other than BMN 110 in a clinical study) or investigational medical device within 30 days prior to Baseline, or requirement for any investigational agent prior to completion of all scheduled program assessments.
  • Not a current US resident or expecting to have travel plans outside the US during the planned period of participation in the Expanded Access Program (EAP) that may interfere with dosing regimen, scheduled program visits and safety monitoring.
  • Any condition that, in the view of the Investigator or sponsor, places the patient at high risk of poor treatment compliance or of not completing the EAP.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

BioMarin Pharmaceutical

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

First Submitted

May 15, 2013

First Submitted That Met QC Criteria

May 16, 2013

First Posted (Estimate)

May 21, 2013

Study Record Updates

Last Update Posted (Estimate)

April 2, 2014

Last Update Submitted That Met QC Criteria

March 31, 2014

Last Verified

March 1, 2014

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 110-503

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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