A Multicenter, Multinational, Observational Morquio A Registry Study (MARS)



Status:Recruiting
Conditions:Orthopedic, Metabolic, Metabolic
Therapuetic Areas:Pharmacology / Toxicology, Orthopedics / Podiatry
Healthy:No
Age Range:Any
Updated:8/19/2018
Start Date:September 2014
End Date:September 2024
Contact:MEDINFO
Email:MEDINFO@bmrn.com
Phone:615-523-0310

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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.

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.

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
We found this trial at
21
sites
Chicago, Illinois 60614
Principal Investigator: Barbara Burton, MD
Phone: 312-227-6764
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Los Angeles, California 90033
213) 740-2311
Principal Investigator: Shoji Yano, MD
Phone: 323-226-3816
University of Southern California The University of Southern California is one of the world’s leading...
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3100 SW 62nd Ave
Miami, Florida 33155
(305) 666-6511
Principal Investigator: Mislen Bauer, MD
Phone: 786-624-3534
Miami Children's Hospital Welcome to Miami Children
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Miami, FL
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601 Children's Lane
Norfolk, Virginia 23507
(757) 668-7000
Principal Investigator: Samantha A Vergano, MD
Phone: 757-401-1132
Children's Hospital of The King's Daughters Children
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1201 W La Veta Ave
Orange, California 92868
(714) 997-3000
Principal Investigator: Raymond Wang, MD
Phone: 714-509-3008
Children's Hospital of Orange County For more than 45 years, CHOC Children’s has been steadfastly...
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South 34th Street
Philadelphia, Pennsylvania 19104
 215-590-1000
Principal Investigator: Lynne Ierardi-Curto, MD
Phone: 267-426-1368
Children's Hospital of Philadelphia Since its start in 1855 as the nation's first hospital devoted...
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4401 Penn Avenue
Pittsburgh, Pennsylvania 15224
412-692-5325
Principal Investigator: Damara Ortiz, MD
Phone: 412-692-6065
Children's Hospital of Pittsburgh of UPMC UPMC is one of the leading nonprofit health systems...
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Boston, Massachusetts 02115
Principal Investigator: Amy Kritzer, MD
Phone: 857-218-4636
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Decatur, Georgia 30033
Principal Investigator: William Wilcox, MD
Phone: 404-778-8618
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2301 Erwin Rd
Durham, North Carolina 27710
919-684-8111
Principal Investigator: Priya Kishnani, MD
Phone: 919-681-1945
Duke Univ Med Ctr As a world-class academic and health care system, Duke Medicine strives...
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1319 Punahou St
Honolulu, Hawaii 96826
(808) 983-6000
Principal Investigator: Robert Wallerstein, MD
Phone: 808-983-6427
Kapiolani Medical Center for Women and Children Hawai‘i Pacific Health is an integrated health care...
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Las Vegas, Nevada 89109
Principal Investigator: Angela Berg, DNP
Phone: 702-862-1136
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1 Children's Way
Little Rock, Arkansas 72202
(501) 364-1100
Principal Investigator: Thomas Burrow, MD
Phone: 501-364-3120
Arkansas Children's Hospital Arkansas Children's Hospital (ACH) is the only pediatric medical center in Arkansas...
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Louisville, Kentucky 40202
Principal Investigator: Alexander Asamoah, MD
Phone: 502-629-2721
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9000 W Wisconsin Ave #270
Milwaukee, Wisconsin 53226
(414) 266-2000
Principal Investigator: Donald Basel, MD
Phone: 414-266-3072
Children's Hospital of Wisconsin Nothing matters more than our children. At Children's Hospital of Wisconsin,...
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Minneapolis, Minnesota 55404
Principal Investigator: Vikas Bhambani, MD
Phone: 612-813-6922
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New York, New York 10016
Principal Investigator: Heather Lau, MD
Phone: 212-263-0139
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4800 Sand Point Way NE
Seattle, Washington 98105
(206) 987-2000
Principal Investigator: Angela Sun, MD
Phone: 206-884-1264
Seattle Children's Hospital Seattle Children’s Hospital specializes in meeting the unique physical, emotional and developmental...
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Washington, District of Columbia 20010
Principal Investigator: Pranoot Tanpaiboon, MD
Phone: 202-476-6812
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Westmead, New South Wales 2145
Principal Investigator: Kaustuv Bharracharya, MD
Phone: +61 (2) 9845 3650
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Wilmington, Delaware 19803
Principal Investigator: Michael Bober, MD
Phone: 302-651-5916
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