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MR Spectroscopy and Disease Severity Score for Gaucher in Pediatric Population

NCT01397435 · Status: COMPLETED · Type: OBSERVATIONAL · Enrollment: 24

Last updated 2015-02-04

No results posted yet for this study

Summary

The bone status in Gaucher disease is very difficult to monitor precisely in children. This is a major problem because lack of optimal treatment, especially enzyme replacement, may cause irreversible severe bone damage that will impact an affected person's life. Currently, there are qualitative (subjective) methods, such as Magnetic resonance Imaging (MRI), to gauge the response to treatment. A quantitative (objective) measurement of Gaucher cell presence and activity in bone marrow could help with more precise and accurate monitoring of bone marrow disease in patients both treated and not (yet) being treated with enzyme replacement. The investigators will evaluate the efficacy of Magnetic Resonance Spectroscopy (MRS) as a quantitative assessment of bone marrow involvement in Children with Gaucher, and examine how this result correlates with semiquantitative MRI scales and overall disease severity.

Conditions

  • Gaucher

Sponsors & Collaborators

Principal Investigators

  • Diego Jaramillo, MD, MPH · Children's Hospital of Philadelphia

  • Paige Kaplan, MBBCh · Children's Hospital of Philadelphia

  • Maria A Bedoya, MD · Children's Hospital of Philadelphia

Eligibility

Min Age
5 Years
Max Age
20 Years
Sex
ALL
Healthy Volunteers
Yes

Timeline & Regulatory

Start
2011-07-31
Primary Completion
2013-12-31
Completion
2014-12-31

Countries

  • United States

Study Locations

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Entities

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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 NCT01397435 on ClinicalTrials.gov