An observational study in Mucopolysaccharidosis (MPS), sponsored by Shire. Terminated at 14 sites in 7 countries. Open to male participants aged Up to 7 Years. Per ClinicalTrials.gov, last updated 2021-03-17.
Sponsored by Shire (part of Takeda) · Observational
Among 37 studies in Mucopolysaccharidoses with a site in United States — 7 open to participants now, 9 under way, 1 of them phase 3. Mucopolysaccharidoses · United States.
Among the 37 studies in Mucopolysaccharidoses with a site in United States, this one is the 7th largest by enrollment of the 18 observational ones that give a count, is one of 5 that started in 2014, is one of 18 observational ones, and was the 17th registered.
59 studies on the registry are indexed under Mucopolysaccharidoses; 11 are open to participants now.
This study's enrollment of 159 is above the median of 50 across 30 observational studies indexed under Mucopolysaccharidoses.
Browse Mucopolysaccharidoses studies →7 of the 37 studies in Mucopolysaccharidoses with a site in United States are open now.
A study can be open while its sites in United States have not opened yet or have stopped. Only those recruiting at a site in United States →
Of the 6 Mucopolysaccharidoses studies recruiting now, 2 (33%) set no upper age limit and 3 (50%) stop at 75 or younger; 5 take children under 18; 2 take healthy volunteers.
This study: Up to 7 Years · male · no healthy volunteers.
The full table on the hub →37 studies; the count open now beside each. This study's row is marked.
A study in two phases is counted once above. Listed by phase, each with its combined phases: Phase 1 9 · Phase 2 10 · Phase 3 4 · Phase 4 1
First in 1996; this study’s year is marked. 5 started in 2014.
Median enrollment 11 across the 19 interventional studies, 78 across the 18 observational. Of the 26 that completed or were terminated, 11 (42%) have results posted.
17 of 37 have an industry lead sponsor. The 10 most frequent:
Shire is the lead sponsor of 351 studies on the registry; 2 are open to participants now.
Of its 48 completed or terminated interventional studies of FDA-regulated products, 48 (100%) have results posted.
Shire has 5 studies filed under Mucopolysaccharidoses — fewer than ten, so there is no comparison to draw.
1 of the studies in Mucopolysaccharidoses with a site in United States that are under way reach primary completion within a year, 0 of them phase 3. Phase 3 first, then by date:
| Study | Primary completion |
|---|---|
| NCT01962415Paul SzabolcsReduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT | 2026-11 |
Among the 37 studies in Mucopolysaccharidoses with a site in United States, from their registry updates:
Nothing recorded among them since 2026-09-25.
Mucopolysaccharidosis (MPS) type II (MPS II; Hunter syndrome) is a rare, X-linked disease caused by a deficiency of the lysosomal enzyme iduronate-2-sulfatase (I2S) and occurs almost exclusively in boys, with an incidence of approximately 1.3 per 100,000 live male births.1 Early identification of MPS II is challenging because some initial features, such as chronic runny nose, otitis media, and hernias, are commonly seen in the general population. As a result, even though the signs and symptoms of MPS II typically appear early in childhood, the diagnosis may lag behind by several years.
The primary objective of this international multi-center study is to evaluate the positive screening rate of MPS II subjects by screening a high-risk male pediatric population who have had or are scheduled for 1 or more specific ENT surgical procedures (adenoidectomy and/or tonsillectomy and/or tympanostomy) and who have a previously repaired or present evidence of an inguinal and/or umbilical hernia.
With evidence-based information from MPS registries regarding the types of surgical interventions that are most prevalent in MPS, this screening study is expected to provide the understanding of the role pediatric ENT surgeons can play in identifying young children with MPS.
Males of any ethnic background or race, \<7 years of age or under, who have had or are scheduled for adenoidectomy and/or tonsillectomy and/or tympanostomy (alone or in combination), will be enrolled. In addition, the subjects must have previous surgical repair or presence of inguinal and/or umbilical hernia.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Each subject must meet the following criteria to be enrolled in this study:
Exclusion Criteria:
Subjects who meet any of the following criteria will be excluded from the study:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
All subjects enrolled in the study who meet the eligibility criteria
The status of diagnosis of MPS II (either positive or negative) of each subject
To evaluate the positive screening rate of MPS II subjects by screening a high risk male pediatric population.
Time frame: Screening visit
The status of diagnosis of MPS I or VI (either positive or negative) of each subject
Time frame: Screening visit
Ran from May 2014 to primary completion in Aug 2015, 14 months.
How these figures are worked out, and which studies are left out of them.
This study is terminated, as verified in Mar 2021. You cannot join it, but the record below documents what was studied.
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