|
Main
|
|
Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
|
Register:
|
ClinicalTrials.gov |
|
Last refreshed on:
|
12 April 2021 |
|
Main ID: |
NCT00214773 |
|
Date of registration:
|
13/09/2005 |
|
Prospective Registration:
|
No |
|
Primary sponsor: |
|
|
Public title:
|
Mucopolysaccharidosis (MPS) VI Clinical Surveillance Program (CSP)
|
|
Scientific title:
|
MPS VI Clinical Surveillance Program (CSP) |
|
Date of first enrolment:
|
July 2005 |
|
Target sample size:
|
237 |
|
Recruitment status: |
Completed |
|
URL:
|
https://clinicaltrials.gov/show/NCT00214773 |
|
Study type:
|
Observational |
|
Study design:
|
|
|
Phase:
|
|
|
|
Countries of recruitment
|
|
Australia
|
Austria
|
Belgium
|
France
|
Germany
|
Ireland
|
Italy
|
Lithuania
|
|
Netherlands
|
Portugal
|
Sweden
|
United Kingdom
|
United States
| | | |
|
Contacts
|
|
Name:
|
Julie Johnson |
|
Address:
|
|
|
Telephone:
|
|
|
Email:
|
|
|
Affiliation:
|
BioMarin Pharmaceutical |
| | |
|
Key inclusion & exclusion criteria
|
Inclusion Criteria
All patients must meet the following criteria to qualify for enrollment in the CSP:
- Patient or patient's parent or legal guardian, if child is under 18 year old or is
unable to consent, has provided a signed Patient Information and Authorization Form.
- Patient has laboratory results confirming a diagnosis of MPS VI disease based on
detection of deficient ARSB activity (on fibroblasts, leucocytes or dried blood
spots)and/or abnormality on the ARSB gene.
- Patient is willing to undergo general assessments to establish baseline data or
permits physician to enter assessment data recorded prior to CSP entry if available in
the patient's medical records. General assessments include: urinary GAG level, urinary
protein level, serum sample for antibody levels, height, weight, and patient history.
Age minimum:
N/A
Age maximum:
N/A
Gender:
All
|
|
Health Condition(s) or Problem(s) studied
|
|
Mucopolysaccharidosis VI (MPS VI, Maroteaux-Lamy Syndrome)
|
|
Primary Outcome(s)
|
|
To further characterize the natural progression of MPS VI disease, irrespective of treatment modality and to evaluate efficacy and safety treatment with Galsulfase.
[Time Frame: at least 15 years]
|
|
Secondary ID(s)
|
|
MPSVI CSP
|
|
Source(s) of Monetary Support
|
|
Please refer to primary and secondary sponsors
|
|
Results
|
|
Results available:
|
|
|
Date Posted:
|
|
|
Date Completed:
|
|
|
URL:
|
|
|
|