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Study to Detect Unrecognized Mucopolysaccharidosis in Children Visiting Rheumatology, Hand or Skeletal Dysplasia Clinics

NCT01675674 · Status: TERMINATED · Type: OBSERVATIONAL · Enrollment: 3000

Last updated 2013-05-24

No results posted yet for this study

Summary

This study is being done to learn how many children and young adults who come to pediatric rheumatology clinics may have mucopolysaccharidosis (MPS). The study tests for 4 of the types of MPS: I, II, IVA, and VI. This can help researchers decide whether to create a screening program for MPS at pediatric rheumatology clinics. This study is being done in rheumatology clinics because the first symptoms of MPS are often joint problems such as stiff joints, and rheumatologists may be the first doctors that a patient with MPS visits. The study will also evaluate the utility of dried blood spot testing for MPS.

Conditions

  • Mucopolysaccharidoses
  • Mucopolysaccharidosis I
  • Mucopolysaccharidosis II
  • Mucopolysaccharidosis IV
  • Mucopolysaccharidosis VI

Interventions

OTHER

Dried blood spot test for MPS

The dried blood spot test uses a few drops of blood on filter paper to screen for mucopolysaccharidoses (MPS I, MPS II, MPS IVA and MPS VI in this study).

Sponsors & Collaborators

  • MediResource Inc.

    collaborator OTHER

  • National MPS Society

    lead OTHER

Principal Investigators

  • Thomas JA Lehman, MD · Chief, Division of Pediatric Rheumatology, Hospital for Special Surgery; Professor of Clinical Pediatrics, Cornell University

Eligibility

Min Age
6 Months
Max Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2011-09-30
Primary Completion
2014-03-31
Completion
2014-03-31

Countries

  • United States

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT01675674 on ClinicalTrials.gov