Clinical Trials List
2017-03-30 - 2025-02-20
Others
Not yet recruiting8
Terminated1
Study ended1
ICD-10E76.01
Hurler's syndrome
ICD-10E76.02
Hurler-Scheie syndrome
ICD-10E76.03
Scheie's syndrome
ICD-10E76.1
Mucopolysaccharidosis, type II
ICD-10E76.210
Morquio A mucopolysaccharidoses
ICD-10E76.211
Morquio B mucopolysaccharidoses
ICD-10E76.219
Morquio mucopolysaccharidoses, unspecified
ICD-10E76.22
Sanfilippo mucopolysaccharidoses
ICD-10E76.29
Other mucopolysaccharidoses
ICD-10E76.3
Mucopolysaccharidosis, unspecified
ICD-10E76.8
Other disorders of glucosaminoglycan metabolism
ICD-10E76.9
Glucosaminoglycan metabolism disorder, unspecified
ICD-9277.5
Mucopolysaccharidosis
NCT02294877
-
Trial Applicant
ICON Clinical Research Pte Ltd
-
Sponsor
BioMarin Pharmaceutical Inc.
-
Trial scale
Multi-Regional Multi-Center
-
Update
2026/09/14
Investigators and Locations
Co-Principal Investigator
- 林翔宇 Division of Pediatrics
The Actual Total Number of Participants Enrolled
0 Not yet recruiting
Co-Principal Investigator
- WUH-LIANG HWU Division of Pediatrics
The Actual Total Number of Participants Enrolled
0 Not yet recruiting
Co-Principal Investigator
- 陳冠容 Division of Pediatrics
The Actual Total Number of Participants Enrolled
0 Not yet recruiting
Co-Principal Investigator
The Actual Total Number of Participants Enrolled
0 Not yet recruiting
Co-Principal Investigator
- 蔡孟哲 Division of Pediatrics
The Actual Total Number of Participants Enrolled
0 Not yet recruiting
Co-Principal Investigator
- 柯瑜媛 Division of Pediatrics
The Actual Total Number of Participants Enrolled
0 Not yet recruiting
Co-Principal Investigator
The Actual Total Number of Participants Enrolled
0 Not yet recruiting
Co-Principal Investigator
The Actual Total Number of Participants Enrolled
0 Not yet recruiting
Co-Principal Investigator
Co-Principal Investigator
The Actual Total Number of Participants Enrolled
0 Terminated
The Actual Total Number of Participants Enrolled
0 Study ended
Condition/Disease
Objectives
Test Drug
Active Ingredient
Dosage Form
Dosage
Endpoints
Efficacy of Vimizim as measured by the 6MWT, 3MSCT, RFTs, FEV1, FIVC, FVC, and MVV
Inclution 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
The Estimated Number of Participants
-
Taiwan
16 participants
-
Global
520 participants
