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

Prevalence Assessment of Unrecognized Mucopolysaccharidosis I, II, IVA, and VI in Juvenile Idiopathic Arthritis Patients with low inflammatory markers - ASY13969

Status
Active, not recruiting
Phases
Unknown
Study type
Observational
Source
NL-OMON
Registry ID
NL-OMON45958
Enrollment
120
Registered
2015-12-09
Start date
2016-02-18
Completion date
Unknown
Last updated
2024-04-23

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Juvenile Arthritis Juvenile Idiopathic Arthritis

Interventions

None listed

Sponsors

Sanofi-aventis
Lead Sponsor

Eligibility

Age
2 Years to 99 Years

Inclusion criteria

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

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.

Design outcomes

Primary

MeasureTime frame
Prevalence rate (number of new diagnosed cases among the total enrolled patients) of MPS I, II, IVA, and VI in pediatric population treated in pediatric rheumatology clinics.

Secondary

MeasureTime frame
-          JADAS-27 score: Active Joint Count + Patient Global Evaluation + Physician Global Assessment + Erythrocyte Sedimentation Rate -          Duration of morning stiffness

Countries

Netherlands

Outcome results

None listed

Source: NL-OMON (via WHO ICTRP)