Now Available for Public Comment: Notice of Proposed Rulemaking (NPRM) for FDAAA 801 and NIH Draft Reporting Policy for NIH-Funded Trials

BMN 110 US Expanded Access Program

This treatment has been approved for sale to the public.
Sponsor:
Information provided by (Responsible Party):
BioMarin Pharmaceutical
ClinicalTrials.gov Identifier:
NCT01858103
First received: May 15, 2013
Last updated: March 31, 2014
Last verified: March 2014
  Purpose

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.


Condition Intervention
Mucopolysaccharidosis IVA
Morquio A Syndrome
MPS IVA
Drug: BMN 110

Study Type: Expanded Access     What is Expanded Access?
Official Title: A Multicenter, Open-label BMN 110 US Expanded Access Program (BMN 110 US EAP) to Provide BMN 110 to Patients Diagnosed With MPS IVA

Resource links provided by NLM:


Further study details as provided by BioMarin Pharmaceutical:

  Eligibility

Genders Eligible for Study:   Both
Criteria

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.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01858103

  Show 25 Study Locations
Sponsors and Collaborators
BioMarin Pharmaceutical
  More Information

No publications provided

Responsible Party: BioMarin Pharmaceutical
ClinicalTrials.gov Identifier: NCT01858103     History of Changes
Other Study ID Numbers: 110-503
Study First Received: May 15, 2013
Last Updated: March 31, 2014
Health Authority: United States: Food and Drug Administration

Keywords provided by BioMarin Pharmaceutical:
Mucopolysaccharidosis IVA Type A
MPS IVA Type A
Mucopolysaccharidosis IVA
MPS IVA
Morquio A Syndrome
Lysosomal Storage Disorder
LSD
N-acetylgalactosamine-6-sulfatase
N-acetylgalactosamine-6-sulfate

Additional relevant MeSH terms:
Mucopolysaccharidoses
Mucopolysaccharidosis IV
Syndrome
Carbohydrate Metabolism, Inborn Errors
Connective Tissue Diseases
Disease
Genetic Diseases, Inborn
Lysosomal Storage Diseases
Metabolic Diseases
Metabolism, Inborn Errors
Mucinoses
Pathologic Processes

ClinicalTrials.gov processed this record on November 27, 2014