Medical Information Only. Always consult your healthcare provider before enrolling in any clinical trial.
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 is a clinical trial that has completed, run by Children's Hospital of Philadelphia. The registered enrollment target is 24 participants.
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.
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 24 participants |
| Start Date | 2011-07 |
| Est. Completion | 2014-12 |
Interested in This Trial?
Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT01397435
The ClinicalTrials.gov registry entry for NCT01397435 describes a study currently listed as completed, categorized as an unspecified phase. The registered enrollment target is 24 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Children's Hospital of Philadelphia, which has 431 total studies on file at ClinicalTrials.gov.
The record links to 0 conditions, and to 0 interventions.
NCT01397435 reports 0 study locations.
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.
Learn More About Clinical Trials
Explore more on PlainTrial
Read our methodology - how this data is sourced, computed, and verified.
Related
Every figure on PlainTrial is rendered directly from the ClinicalTrials.gov registry, no number is typed in by an editor. This page mirrors this trial's own ClinicalTrials.gov registry record, live from the dataset. See our editorial standards & corrections policy, the methodology behind these numbers, or report a data error.