Status:
COMPLETED
Psychological Concomitants of Morquio A Syndrome - Longitudinal Effects of Enzyme Replacement Therapy (The MAPLE Study)
Lead Sponsor:
Nadia Ali, PhD
Collaborating Sponsors:
BioMarin Pharmaceutical
Conditions:
Morquio A Syndrome
Mucopolysaccharidosis IV A
Eligibility:
All Genders
18+ years
Brief Summary
Mucopolysaccharidosis IV, also known as MPS IV or Morquio disease, is a rare autosomal recessive genetic lysosomal storage disorder. Research thus far regarding Morquio, has primarily focused on the p...
Detailed Description
Mucopolysaccharidosis IV, also known as MPS IV or Morquio disease, is a rare autosomal recessive genetic lysosomal storage disorder. Research thus far regarding lysosomal storage diseases (LSDs) in ge...
Eligibility Criteria
Inclusion
- Documented clinical diagnosis of MPS IVA based on clinical signs and symptoms of MPS IVA and documented reduced fibroblast or leukocyte GALNS enzyme activity or genetic testing confirming diagnosis of MPS IVA.
- Subject is at least 18 years old.
- Subject must provide informed consent prior to study participation.
- Subject was a participant in the MAP study (Phase I) and is now receiving (or plans to receive in the near future) enzyme replacement therapy in the EAP or commercial setting. If receiving ERT for the treatment of Morquio A syndrome, subject has been on treatment for less than 1 year.
- or-
- Subject was not enrolled in the MAP study, but plans to start receiving ERT for Morquio A syndrome in the near future and is willing to take all baseline questionnaires which were included in MAP, prior to beginning ERT for Morquio A syndrome .
Exclusion
- 1\. Previous treatment with ERT prior to participation in phase 1(MAP).
- 2\. Previous hematopoietic stem-cell transplant
- 3\. Patient has a clinically significant disease (with the exception of symptoms of Morquio A syndrome), including clinically significant cardiovascular, hepatic, immunologic, pulmonary, neurologic, or renal disease, or other medical condition, serious intercurrent illness, or extenuating circumstances that, in the opinion of the investigator, would confound the effects of Morquio A syndrome upon study variables.
- 4\. Any condition that, in the view of the Investigator, places the patient at high risk of poor compliance or of not completing the study.
Key Trial Info
Start Date :
March 1 2014
Trial Type :
OBSERVATIONAL
Allocation :
ACTUAL
End Date :
March 1 2018
Estimated Enrollment :
12 Patients enrolled
Trial Details
Trial ID
NCT02208661
Start Date
March 1 2014
End Date
March 1 2018
Last Update
April 11 2018
Active Locations (1)
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1
Emory University
Decatur, Georgia, United States, 30033