Observational Study of Patients With Mucopolysaccharidosis (MPS) VI Who Previously Participated in ASB-00-02
This study has been completed.
Information provided by (Responsible Party):
First received: June 29, 2011
Last updated: March 31, 2014
Last verified: March 2014
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.
||A Re-Survey Study of Patients With MPS VI (Maroteaux-Lamy Syndrome) Who Previously Participated in ASB-00-02
Primary Outcome Measures:
| Study Start Date:
| Study Completion Date:
| Primary Completion Date:
||October 2012 (Final data collection date for primary outcome measure)
|Genders Eligible for Study:
|Accepts Healthy Volunteers:
Patients with MPS VI who previously participated in ASB-00-02
- 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.
- 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.
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study.
To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below.
For general information, see Learn About Clinical Studies.
Please refer to this study by its ClinicalTrials.gov identifier: NCT01387854
|Children's Hospital and Research Center
|Oakland, California, United States, 94609 |
|Women's and Children's hospital
|North Adelaide, Australia |
|Hospital de Clinicas de Porto Alegre
|Porto Alegre, Brazil, 21615 |
|Hôpital Femme Mère Enfant
|Lyon, France, 69677 |
|Prof Michael Beck
|Mainz, Germany, 55131 |
|Al. Prof Hernani Monteiro
|Porto, Portugal, 4202-451 |
|Manchester Academic Health Sciences Centre
|Manchester, United Kingdom, M13 9 WL |
No publications provided
ClinicalTrials.gov processed this record on September 16, 2014
History of Changes
|Other Study ID Numbers:
|Study First Received:
||June 29, 2011
||March 31, 2014
||United States: Food and Drug Administration