問卷

TPIDB > Search Result > Clinical Trials List

Clinical Trials List

Protocol Number110-504
NCT Number(ClinicalTrials.gov Identfier)NCT02294877
Active

2017-03-30 - 2025-02-20

Phase III

Not yet recruiting8

Recruiting2

Terminated2

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

Morquio A registry Study

  • Trial Applicant

    ICON Clinical Research Pte Ltd

  • Sponsor

    BioMarin Pharmaceutical Inc.

  • Trial scale

    Multi-Regional Multi-Center

  • Update

    2026/02/01

Investigators and Locations

Principal Investigator 林炫沛 Division of Pediatrics

Co-Principal Investigator

The Actual Total Number of Participants Enrolled

0 Not yet recruiting

Principal Investigator 趙美琴 Division of Pediatrics

Co-Principal Investigator

The Actual Total Number of Participants Enrolled

0 Not yet recruiting

Principal Investigator CHIA -CHI HSU Division of Pediatrics

Co-Principal Investigator

The Actual Total Number of Participants Enrolled

0 Not yet recruiting

Principal Investigator Ru-Li Lin Division of Pediatrics

Co-Principal Investigator

The Actual Total Number of Participants Enrolled

0 Not yet recruiting

Principal Investigator Fuu-Jen Tsai Division of Pediatrics

Co-Principal Investigator

The Actual Total Number of Participants Enrolled

0 Not yet recruiting

Principal Investigator 邱寶琴 Division of Pediatrics

Co-Principal Investigator

The Actual Total Number of Participants Enrolled

0 Not yet recruiting

Principal Investigator Yin-Hsiu Chien Division of Pediatrics

Co-Principal Investigator

The Actual Total Number of Participants Enrolled

0 Not yet recruiting

Principal Investigator Hui-Pin Hsiao Division of Pediatrics

Co-Principal Investigator

The Actual Total Number of Participants Enrolled

0 Not yet recruiting

Principal Investigator Yen-Yin Chou Division of Pediatrics

Co-Principal Investigator

The Actual Total Number of Participants Enrolled

0 Recruiting

Principal Investigator Dau-Ming Niu Division of Pediatrics

Co-Principal Investigator

The Actual Total Number of Participants Enrolled

0 Stop recruiting

Principal Investigator 林炫沛 Division of Pediatrics

Co-Principal Investigator

The Actual Total Number of Participants Enrolled

0 Stop recruiting

Principal Investigator 李振豪 Division of Pediatrics

Co-Principal Investigator

The Actual Total Number of Participants Enrolled

0 Recruiting

Condition/Disease

Mucopolysaccharidosis type IVA

Objectives

An observation study of patients with Mucopolysaccharidosis type IVA, either with or without enzyme replacement therapy (Vimizim)

Test Drug

Vimizim

Active Ingredient

NA

Dosage Form

NA

Dosage

2 mg/kg/qwk

Endpoints

Descriptive observation and data collect of patients with Mucopolysaccharidosis type IVA, either with or without enzyme replacement therapy (Vimizim)
-natural course, physical examination and biochemical data
-long ter safty and averse events
-efficacy

Inclution Criteria

patients confirmed with Mucopolysaccharidosis type IVA and sign with the informed consent

Exclusion Criteria

those who disagree with the informed consent

The Estimated Number of Participants

  • Taiwan

    16 participants

  • Global

    520 participants