Back to resultsObservational Study of Patients With Mucopolysaccharidosis (MPS) VI Who Previously Participated in ASB-00-02
Primary Purpose
MPS VI
Status
Completed
Phase
Locations
International
Study Type
Observational
Intervention
Sponsored by
About this trial
This is an observational trial for MPS VI
Eligibility Criteria
Inclusion Criteria:
- Willing and able to provide written, signed informed consent, or in the case of patients under the age of 16 years, provide written assent (if required) and written informed consent by a legally authorized representative after the nature of the study has been explained, and prior to any research-related procedures.
- Previously participated in Survey Study ASB-00-02, and met the criteria of a MPS VI diagnosis.
- Willing to perform all study assessments and procedures as physically possible.
Exclusion Criteria:
- Concurrent disease or condition that would interfere with study participation or safety.
- Any condition that, in the view of the Investigator would place the patient at high risk of not completing the study.
- Concurrent enrollment and randomization into a clinical study of MPS VI treatment. Patients enrolled in the BioMarin MPS VI Clinical Surveillance Program may be enrolled in the Re-survey Study ASB-00-03.
Sites / Locations
- Children's Hospital and Research Center
- Women's and Children's hospital
- Hospital de Clinicas de Porto Alegre
- Hôpital Femme Mère Enfant
- Prof Michael Beck
- Al. Prof Hernani Monteiro
- Manchester Academic Health Sciences Centre
Outcomes
Primary Outcome Measures
Linear Comparison of assessments completed for Survey Study ASB-00-02 and and Re-Survey StudyASB-00-03
This study is designed to be performed in a similar manner to the Survey Study ASB-00-02. Similar assessments will be performed in order to make linear comparisons in outcomes over a long-term period.
Secondary Outcome Measures
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT01387854
Brief Title
Observational Study of Patients With Mucopolysaccharidosis (MPS) VI Who Previously Participated in ASB-00-02
Official Title
A Re-Survey Study of Patients With MPS VI (Maroteaux-Lamy Syndrome) Who Previously Participated in ASB-00-02
Study Type
Observational
2. Study Status
Record Verification Date
March 2014
Overall Recruitment Status
Completed
Study Start Date
June 2011 (undefined)
Primary Completion Date
October 2012 (Actual)
Study Completion Date
February 2013 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
BioMarin Pharmaceutical
4. Oversight
Data Monitoring Committee
No
5. Study Description
Brief Summary
There is limited information on the long-term effects of treating patients with MPS VI with Naglazyme® and limited data on the natural history of treated and untreated MPS VI patients. The Re-survey Study ASB-00-03 will assist in understanding the effects of long-term Naglazyme treatment and the natural history of the MPS VI patient population.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
MPS VI
7. Study Design
Enrollment
59 (Actual)
Primary Outcome Measure Information:
Title
Linear Comparison of assessments completed for Survey Study ASB-00-02 and and Re-Survey StudyASB-00-03
Description
This study is designed to be performed in a similar manner to the Survey Study ASB-00-02. Similar assessments will be performed in order to make linear comparisons in outcomes over a long-term period.
Time Frame
Variable dependent on date of assessment performed fo rthe Survey STudy ASB-00-02
10. Eligibility
Sex
All
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Willing and able to provide written, signed informed consent, or in the case of patients under the age of 16 years, provide written assent (if required) and written informed consent by a legally authorized representative after the nature of the study has been explained, and prior to any research-related procedures.
Previously participated in Survey Study ASB-00-02, and met the criteria of a MPS VI diagnosis.
Willing to perform all study assessments and procedures as physically possible.
Exclusion Criteria:
Concurrent disease or condition that would interfere with study participation or safety.
Any condition that, in the view of the Investigator would place the patient at high risk of not completing the study.
Concurrent enrollment and randomization into a clinical study of MPS VI treatment. Patients enrolled in the BioMarin MPS VI Clinical Surveillance Program may be enrolled in the Re-survey Study ASB-00-03.
Study Population Description
Patients with MPS VI who previously participated in ASB-00-02
Sampling Method
Non-Probability Sample
Facility Information:
Facility Name
Children's Hospital and Research Center
City
Oakland
State/Province
California
ZIP/Postal Code
94609
Country
United States
Facility Name
Women's and Children's hospital
City
North Adelaide
Country
Australia
Facility Name
Hospital de Clinicas de Porto Alegre
City
Porto Alegre
ZIP/Postal Code
21615
Country
Brazil
Facility Name
Hôpital Femme Mère Enfant
City
Lyon
ZIP/Postal Code
69677
Country
France
Facility Name
Prof Michael Beck
City
Mainz
ZIP/Postal Code
55131
Country
Germany
Facility Name
Al. Prof Hernani Monteiro
City
Porto
ZIP/Postal Code
4202-451
Country
Portugal
Facility Name
Manchester Academic Health Sciences Centre
City
Manchester
ZIP/Postal Code
M13 9 WL
Country
United Kingdom
12. IPD Sharing Statement
Citations:
PubMed Identifier
24764221
Citation
Giugliani R, Lampe C, Guffon N, Ketteridge D, Leao-Teles E, Wraith JE, Jones SA, Piscia-Nichols C, Lin P, Quartel A, Harmatz P. Natural history and galsulfase treatment in mucopolysaccharidosis VI (MPS VI, Maroteaux-Lamy syndrome)--10-year follow-up of patients who previously participated in an MPS VI Survey Study. Am J Med Genet A. 2014 Aug;164A(8):1953-64. doi: 10.1002/ajmg.a.36584. Epub 2014 Apr 24.
Results Reference
derived
Learn more about this trial
Observational Study of Patients With Mucopolysaccharidosis (MPS) VI Who Previously Participated in ASB-00-02
We'll reach out to this number within 24 hrs