Medical Information Only. Always consult your healthcare provider before enrolling in any clinical trial.
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: Completed study of Mucopolysaccharidosis VI and Mucopolysaccharidosis IV A, sponsored by Children's Hospitals and Clinics of Minnesota.
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. According to ClinicalTrials.gov, the official US trial registry.
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 |
What the finished NCT01707433 record still lists
NCT01707433 is an observational study that tracks outcomes without assigning an intervention. The registry caps enrollment at 22 participants, a relatively small participant target, below the 65-participant average among 6 other Mucopolysaccharidosis VI trials with a reported enrollment target (66% lower).
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 a single indexed study location in 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.
Learn More About Clinical Trials
Similar trials for Mucopolysaccharidosis VI
Matched on the same primary condition, ranked to surface studies in the same phase first, then by recruiting status, no relevance scoring or editorial curation.
-
Registry of Patients Diagnosed With Lysosomal Storage Diseases
RECRUITING
-
Study to Evaluate the Safety and Efficacy of Adalimumab in MPS I, II, and VI
ACTIVE NOT RECRUITING · Phase 1
-
Stem Cell Transplantation for Hurler
COMPLETED · Phase 2
-
Human Placental-Derived Stem Cell Transplantation
COMPLETED · Phase 1
-
Study of rhASB in Patients With Mucopolysaccharidosis VI
COMPLETED · Phase 3
-
A Phase 4 Two Dose Level Study of Naglazyme(TM) (Galsulfase) in Infants With MPS VI
COMPLETED · Phase 4
Nationwide trials with similar profiles
Cross-condition peers matched on enrollment target and registry start date, not the same-condition list above.
Similar enrollment target
- NCT02508038 · 22 participants · Phase 1
Alpha/Beta CD19+ Depleted Haploidentical Transplantation + Zometa for Pediatric Hematologic Malignancies and Solid Tumors
- NCT03099356 · 22 participants · Phase 2
Cyclophosphamide and Sirolimus for the Treatment of Metastatic, RAI-refractory, Differentiated Thyroid Cancer
- NCT03676647 · 22 participants
Diagnostic DNA Methylation Signature in Diagnosing Thyroid Cancer After Fine-Needle Aspiration in Patients With Thyroid Nodules
- NCT04569591 · 22 participants · NA
DDAVP for Pituitary Adenoma
Similar registry start date
- NCT05988463 · started 2027-01-01 · Phase 1
Dose-Escalation Study of Artesunate Patients With IPF
- NCT07219706 · started 2026-12-10 · NA
Dietary Fiber and Time Restricted Eating
- NCT02831335 · started 2026-12-01
Technical Development of Multi-Parametric Renocerebral MRI
- NCT05962346 · started 2026-12 · NA
Fetal Endoscopic Tracheal Occlusion for Congenital Diaphragmatic Hernia