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NCT01707433 · ClinicalTrials.gov registry record
Diagnosis of Mucopolysaccharidosis Disorders in Patients Presenting With Bilateral Hip Disease
A clinical trial of Mucopolysaccharidosis VI and Mucopolysaccharidosis IV A, sponsored by Children's Hospitals and Clinics of Minnesota.
- Completed
- Registry status
- 22
- Enrollment target
- 2
- Study locations
NCT01707433 is a study of Mucopolysaccharidosis VI and Mucopolysaccharidosis IV A that has completed, run by Children's Hospitals and Clinics of Minnesota. The registered enrollment target is 22 participants, below the 65-participant average among 6 other Mucopolysaccharidosis VI trials with a reported enrollment target (66% lower). The trial reports 2 study locations across 1 state.
The verdict
NCT01707433, a study of Mucopolysaccharidosis VI and Mucopolysaccharidosis IV A, has completed, sponsored by Children's Hospitals and Clinics of Minnesota.
- COMPLETED
- Registry status
- 22 participants
- Enrollment target
- 2
- Study locations
Study Summary
BACKGROUND/OBJECTIVE: Quantitative urine screening for mucopolysaccharides (MPS) has been the primary method for detecting mucopolysaccharidoses in children. This method may not be sufficiently sensitive and may miss some patients with arylsulfatase B (ARSB) deficiency. Investigators propose to identify patients retrospectively and prospectively who carry a diagnosis of spondyloepiphyseal dysplasia, multiple epiphyseal dysplasia, bilateral proximal femoral epiphyseal dysplasia, or bilateral Legg-Calve-Perthes. For these patients, investigators will perform enzyme testing on a blood sample which will identify MPS VI or IVA. Patients who have an earlier diagnosis of MPS are likely to have better health outcomes with medical management. Therefore, it is important to determine effective diagnostic methods. Investigators believe that bilateral hip involvement should alert the clinician to the possibility of MPS VI and further examination. The purpose of this study is to test the hypothesis that the correct diagnoses of two MPS storage disorders are delayed in patients with bilateral proximal femoral epiphyseal dysplasia and normal quantitative urine MPS studies.
Conditions Studied
Interventions
- OTHER Enzyme testing
Study Locations (2)
Minnesota
- Children's Hospitals and Clinics of Minnesota - Minneapolis
- Gillette Children's Specialty Healthcare - Saint Paul
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 22 participants |
| Start Date | 2012-10 |
| Est. Completion | 2015-06 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT01707433
The ClinicalTrials.gov registry entry for NCT01707433 describes a study currently listed as completed, categorized as an unspecified phase. The registered enrollment target is 22 participants, a figure that helps gauge the scale of data the investigators plan to collect, below the 65-participant average among 6 other Mucopolysaccharidosis VI trials with a reported enrollment target (66% lower). The listed sponsor is Children's Hospitals and Clinics of Minnesota, which has 31 total studies on file at ClinicalTrials.gov.
The record links to 2 conditions, with Mucopolysaccharidosis VI appearing as the primary indexed condition, and to 1 intervention - of which Enzyme testing is the first listed.
NCT01707433 reports 2 study locations spanning 1 distinct geographic area - top geographies include Minnesota.
Frequently Asked Questions
What is clinical trial NCT01707433 about?
NCT01707433 is a clinical study titled "Diagnosis of Mucopolysaccharidosis Disorders in Patients Presenting With Bilateral Hip Disease". BACKGROUND/OBJECTIVE: Quantitative urine screening for mucopolysaccharides (MPS) has been the primary method for detecting mucopolysaccharidoses in children. This method may not be sufficiently sensitive and may miss some patients with arylsulfatase B (ARSB) deficiency. Investigators propose to iden...
What is the current status of trial NCT01707433?
This trial is currently completed. The enrollment target is 22 participants. The study started on 2012-10. Estimated completion is 2015-06.
What conditions does trial NCT01707433 study?
This clinical trial studies the following conditions: Mucopolysaccharidosis VI, Mucopolysaccharidosis IV A.
What interventions are being tested in trial NCT01707433?
The interventions under investigation include: Enzyme testing (OTHER).
Who is sponsoring clinical trial NCT01707433?
This trial is sponsored by Children's Hospitals and Clinics of Minnesota, which has 31 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT01707433 being conducted?
This trial has 2 study locations across Minnesota. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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