Clinical trial NCT01387854: 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
Overall status: COMPLETED
Conditions: MPS VI
Lead sponsor: BioMarin Pharmaceutical
Sponsor class: INDUSTRY
First posted: 2011-07-06
Enrollment: 59
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Patients with MPS VI who previously participated in ASB-00-02

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.


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.

Primary outcomes:
measure: Linear Comparison of assessments completed for Survey Study ASB-00-02 and and Re-Survey StudyASB-00-03
timeFrame: Variable dependent on date of assessment performed fo rthe Survey STudy ASB-00-02
Secondary outcomes:
Other outcomes:
Locations:
city: Oakland
country: United States
facility: Children's Hospital and Research Center
state: California
city: North Adelaide
country: Australia
facility: Women's and Children's hospital
city: Porto Alegre
country: Brazil
facility: Hospital de Clinicas de Porto Alegre
city: Lyon
country: France
facility: Hôpital Femme Mère Enfant
city: Mainz
country: Germany
facility: Prof Michael Beck
city: Porto
country: Portugal
facility: Al. Prof Hernani Monteiro
city: Manchester
country: United Kingdom
facility: Manchester Academic Health Sciences Centre
Study officials:
References:
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.