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NCT01397435 · ClinicalTrials.gov registry record
MR Spectroscopy and Disease Severity Score for Gaucher in Pediatric Population
A clinical trial, sponsored by Children's Hospital of Philadelphia.
- Completed
- Registry status
- 24
- Enrollment target
NCT01397435: Completed study, sponsored by Children's Hospital of Philadelphia.
NCT01397435 is a clinical trial that has completed, run by Children's Hospital of Philadelphia. The registered enrollment target is 24 participants. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT01397435 has completed, sponsored by Children's Hospital of Philadelphia.
- COMPLETED
- Registry status
- 24 participants
- Enrollment target
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.
Primary Outcome
We will measure the fat fraction only once, at the time of recruitment. We will compare the fat fraction of an affected subject to that of an age matched control. We will determine if significant difference exist.
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 24 participants |
| Start Date | 2011-07 |
| Est. Completion | 2014-12 |
What the finished NCT01397435 record still lists
NCT01397435 is an observational study that tracks outcomes without assigning an intervention. The registry caps enrollment at 24 participants, a relatively small participant target.
The record links to 0 conditions, and to 0 interventions.
NCT01397435 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT01397435 about?
NCT01397435 is a clinical study titled "MR Spectroscopy and Disease Severity Score for Gaucher in Pediatric Population". 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 (sub...
What is the current status of trial NCT01397435?
This trial is currently completed. The enrollment target is 24 participants. The study started on 2011-07. Estimated completion is 2014-12.
Who is sponsoring clinical trial NCT01397435?
This trial is sponsored by Children's Hospital of Philadelphia, which has 431 total clinical trials registered on ClinicalTrials.gov.
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