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Diagnosis of Mucopolysaccharidosis Disorders in Patients Presenting With Bilateral Hip Disease
NCT01707433 · View on ClinicalTrials.gov ↗
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. It is categorized as an unspecified phase, which is the standard way researchers label where a study sits along the investigational pathway from early safety work through later efficacy and post-marketing evaluation. The registered enrollment target is 22 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Children's Hospitals and Clinics of Minnesota, which has 41 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.
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. Interventions can include drugs, devices, procedures, behavioral programs, or observational arms, and each is tracked as a separate registry field so that downstream queries can filter accurately. When a trial lists multiple interventions, it usually reflects a multi-arm design or a comparison protocol rather than a single treatment being tested in isolation. The brief summary published in the registry is the clearest source of protocol intent and should be read before drawing conclusions from any sidebar tags.
Geographic footprint matters for practical reasons: NCT01707433 reports 2 study locations spanning 1 distinct geographic area — top geographies include Minnesota. A larger site network tends to correlate with broader recruitment capacity, but it does not imply anything about study quality, and site-level enrollment status can diverge from the overall registry status shown above. Every data point on this page comes from the public ClinicalTrials.gov dataset and is reproduced here for reference only; it is not a medical recommendation, an endorsement of the sponsor, or an invitation to enroll. Verify current status, eligibility criteria, and contact details directly at ClinicalTrials.gov, and discuss any participation decision with your own healthcare provider.
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. These conditions were identified from the trial registry and reflect the primary focus areas of the research.
What interventions are being tested in trial NCT01707433?
The interventions under investigation include: Enzyme testing (OTHER). Each intervention is being evaluated for safety and efficacy as part of this clinical study.
Who is sponsoring clinical trial NCT01707433?
This trial is sponsored by Children's Hospitals and Clinics of Minnesota, which has 41 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.
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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