Purpose

The objectives of this program are: to characterize and describe the Mucopolysaccharidosis IV type A (MPS IVA) population as a whole, including the heterogeneity, progression, and natural history of MPS IVA; to evaluate the long-term effectiveness and safety of Vimizim®, including, but not limited to, the occurrence of serious hypersensitivity reactions, anaphylaxis, and changes in antibody status; to help the medical community with the development of recommendations for monitoring MPS IVA patients and reports on patient outcomes to optimize patient care; to collect data on other treatment paradigms, and evaluate the prevalences of their use and their effectiveness; to characterize the effects and safety of Vimizim treatment 5 years from enrollment in the Registry for patients under 5 years of age; to monitor pregnancy exposure, including maternal, neonatal, and infant outcomes; and to monitor patients who have completed the MOR-005 and MOR-007 clinical trials. These patients will be encouraged to enroll in the applicable Registry Substudy and will be monitored using the MOR-005 and MOR-007 assessment schedules, respectively.

Conditions

Eligibility

Eligible Ages
All ages
Eligible Genders
All
Accepts Healthy Volunteers
No

Inclusion Criteria

Patients eligible to participate in this Registry must meet all of the following criteria:

- Diagnosed with MPS IVA as confirmed by either N-acetylgalactosamine 6-sulfatase (GALNS) enzymatic test or by a diagnostic molecular test

- Willing and able to provide written, signed informed consent, or, in the case of patients age < 18 years, provide written assent (if required) and written informed consent, signed by a legally authorized representative after the nature of the Registry has been explained and prior to performance of any Registry-related procedures

- Willing to undergo assessments to establish baseline data or permit Investigator to enter assessment data recorded prior to Registry entry if available in the patient's medical records. Entry assessments may include: demographics, medical history, urinary keratan sulfate level, urinary protein level, immunogenicity testing, vital signs, physical examination, and height and weight

Patients eligible to participate in the Registry Substudy for MOR-005 must meet all of the following criteria:

- Must have completed the MOR-005 clinical trial

- Willing and able to provide written, signed informed consent, or, in the case of patients age < 18 years, provide written assent (if required) and written informed consent by a legally authorized representative after the nature of the Registry Substudy has been explained and prior to any Registry-related Substudy procedures

- Willing to permit Investigator to enter assessment data recorded prior to Registry

- Substudy entry if available in the patient's medical records

Patients eligible to participate in this Registry Substudy for MOR-007 must meet all of the following criteria:

- Must have completed the MOR-007 clinical trial

- Willing and able to provide written, signed informed consent, or in the case of patients age < 18 years, provide written assent (if required) and written informed consent by a legally authorized representative after the nature of the Registry Substudy has been explained, and prior to any Registry-related Substudy procedures

- Willing to permit Investigator to enter assessment data recorded prior to Registry Substudy entry if available in the patient's medical records

Exclusion Criteria

Patients who meet the following exclusion criterion will not be eligible to participate in the Registry or Registry Substudies:

• Patients currently participating in a BMN 110 (elosulfase alfa) clinical trial

Study Design

Phase
Study Type
Observational [Patient Registry]
Observational Model
Cohort
Time Perspective
Prospective

Recruiting Locations

Children's National Health System
Washington, District of Columbia 20010

Children's Hospital of Philadelphia
Philadelphia, Pennsylvania 19104

Duke University Medical Center
Durham, North Carolina 27710

NYU-Langone Medical Center School of Medicine
New York, New York 10016

Atlantic Health System
Morristown, New Jersey 07960

Children's Hospital of Pittsburgh of UPMC
Pittsburgh, Pennsylvania 15224

Children's Hospital of the King's Daughters
Norfolk, Virginia 23507

San Jorge Children's Hospital, Centro de Investigaciones Clinicas
San Juan, Puerto Rico 00912

Children's Hospital of Wisconsin
Milwaukee, Wisconsin 53226

Seattle Children's Hospital
Seattle, Washington 98145-5005

Children's Specialty Center of Nevada
Las Vegas, Nevada 89109

University of Michigan
Ann Arbor, Michigan 48109

Miami Children's Hospital
Miami, Florida 33155

A.I. Dupont Hospital for Children
Wilmington, Delaware 19803

Children's Hospital of Orange County
Orange, California 92868

University of Southern California
Los Angeles, California 90033

Emory University
Decatur, Georgia 30033

Kapiolani Medical Center for Women and Children
Honolulu, Hawaii 96826

Arkansas Children's Hospital
Little Rock, Arkansas 72202

Children's Hospital of Boston
Boston, Massachusetts 02115

Ann & Robert H. Lurie Children's Hospital
Chicago, Illinois 60611

Childrens Hospitals and Clinics of Minnesota
Minneapolis, Minnesota 55404

More Details

NCT ID
NCT02294877
Status
Recruiting
Sponsor
BioMarin Pharmaceutical

Study Contact

Trial Specialist
medinfo@bmrn.com

Detailed Description

MARS is a multicenter, multinational, observational disease registry for patients diagnosed with Mucopolysaccharidosis Type IVA (MPS IVA). The Registry will collect medical history, and clinical and safety assessments every six months, for up to 10 years. The Registry will enroll and collect data on patients over a period of at least 8 years from the time of the first marketing approval globally and data on individual patients will continue to be collected for at least 2 years from the time the last patient was enrolled or until the Registry is completed.

These assessments are designed to further characterize the spectrum of clinical signs and symptoms of the disease, and to further characterize the safety profile of Vimizim in a broader population. It is not required that patients receive Vimizim to be eligible to participate in this Registry.

In addition, this Registry will collect additional data on patients who have completed the MOR-005 and MOR-007 clinical trials. The MOR-005 and MOR-007 clinical trial patients will be enrolled into the appropriate Registry Substudy for a minimum of 5 years from the time of the patient's enrollment in the MOR-005 clinical study or MOR-007 clinical study. After the 5 year period, these patients should remain in MARS until the Registry is complete.

Notice

Study information shown on this site is derived from ClinicalTrials.gov (a public registry operated by the National Institutes of Health). The listing of studies provided is not certain to be all studies for which you might be eligible. Furthermore, study eligibility requirements can be difficult to understand and may change over time, so it is wise to speak with your medical care provider and individual research study teams when making decisions related to participation.