Status:
COMPLETED
A Multicenter, Multinational, Observational Morquio A Registry Study (MARS)
Lead Sponsor:
BioMarin Pharmaceutical
Collaborating Sponsors:
ICON plc
Conditions:
Mucopolysaccharidosis IV Type A
Morquio A Syndrome
Eligibility:
All Genders
Brief Summary
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 M...
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 s...
Eligibility Criteria
Inclusion
- 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
- 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
Key Trial Info
Start Date :
September 1 2014
Trial Type :
OBSERVATIONAL
Allocation :
ACTUAL
End Date :
February 21 2024
Estimated Enrollment :
418 Patients enrolled
Trial Details
Trial ID
NCT02294877
Start Date
September 1 2014
End Date
February 21 2024
Last Update
March 5 2024
Active Locations (79)
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1
Phoenix Children's Hospital
Phoenix, Arizona, United States, 85016
2
Arkansas Children's Hospital
Little Rock, Arkansas, United States, 72202
3
Children's Hospital of Los Angeles
Los Angeles, California, United States, 90027
4
University of Southern California
Los Angeles, California, United States, 90033