Prevalence Assessment of Unrecognized Mucopolysaccharidosis I, II, IVA, and VI in Juvenile Idiopathic Arthritis Patients with low inflammatory markers

Completed

Conditions: Juvenile Arthritis
Juvenile Idiopathic Arthritis
10027424
10021605
10023213

Registration Number: NL-OMON45958

Lead Sponsor: Sanofi-aventis

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: Completed

Sex: Not specified

Target Recruitment: 120

Inclusion Criteria

-          Male or female juvenile idiopathic arthritis patients, between 6 months and 18 years of age, inclusive.
-          History of JIA documented at an experienced pediatric rheumatology clinic. All JIA subtypes can be included if patients have at least 1 low ESR (*20 mm/Hr) and/or CRP (*10 mg/L) value measured at a preceding visit (timelines of the precedent visit is defined as per the patient standard of care) or at the study visit, and assessed as being independent from concomitant anti-inflammatory/anti-infective treatments at the discretion of the investigator.
-          Signed informed consent/assent obtained from patient and patient*s legal representative (parents or guardians) according to local regulations.

Exclusion Criteria

-          Patients for whom MPS enzyme activity tests (ie, enzyme levels tested in fibroblasts, leukocytes, serum, or blood spots) have already been performed and for which the result was normal. (Patients who have been screened for MPS through urinary GAG and tested normal may be included in the study).
-          Patients with at least 1 high ESR (>20 mm/Hr) and/or CRP (>10 mg/L) value measured at a preceding visit or at the study visit, not related to an identified concomitant infection or intercurrent illness at the discretion of the investigator.
-          Patient has any medical condition or extenuating circumstance which, in the opinion of the Investigator, could interfere with the patient*s ability to complete the study procedure, or with the interpretation of study results.

Study & Design

Study Type: Observational invasive

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
<p>Prevalence rate (number of new diagnosed cases among the total enrolled<br /><br>patients) of MPS I, II, IVA, and VI in pediatric population treated in<br /><br>pediatric rheumatology clinics.</p><br>

Secondary Outcome Measures

Name	Time	Method
<p>- JADAS-27 score: Active Joint Count + Patient Global Evaluation +<br /><br>Physician Global Assessment + Erythrocyte Sedimentation Rate<br /><br>- Duration of morning stiffness</p><br>