Mucopolysaccharidosis (MPS) I, II, and VI Screening in a High-Risk Population With Previous Surgical Repair or Presence of Inguinal and/or Umbilical Hernia in Combination With Pediatric ENT Surgery (The HATT Project)

Stopped early

Conditions studied: Mucopolysaccharidosis (MPS)

In brief

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.

Key facts

Study ID

NCT02095015

Run by

Shire

People needed

159

Starts

2014-05-21

Expected to finish

2015-08-14

Last updated by the study team

2021-03-17

Who can join

Age: any, up to 7. Sex: male. Healthy volunteers: not accepted.

You may qualify if…

• Each subject must meet the following criteria to be enrolled in this study:
• The subject is male.
• The subject is <7 years of age.
• The subject has had or is scheduled for ENT surgery for any of the following, alone or in combination: adenoidectomy, tonsillectomy, and tympanostomy.
• The subject has record of previous surgical repair or presence of inguinal and/or umbilical hernia.
• The subject's parent(s) or the subject's legally authorized representative(s) has provided written informed consent that has been approved by the Institutional Review Board/Independent Ethics Committee (IRB/IEC).

You may not qualify if…

• Subjects who meet any of the following criteria will be excluded from the study:
• The subject has a current confirmed diagnosis of any MPS disorder.
• The subject was born prematurely (defined as born before 37 weeks gestation).
• The subject has received a blood transfusion within the past 6 weeks.
• The subject has received a bone marrow transplant.

Where it is running

• Children's Hospital Los Angeles — Los Angeles, California, United States
• Nemours Children's Clinic — Jacksonville, Florida, United States
• Children's Hospital and Clinic — Minneapolis, Minnesota, United States
• Saint Louis Children's Hospital — St Louis, Missouri, United States
• Children's Hospital of Philadelphia — Philadelphia, Pennsylvania, United States
• Children's Hospital of Pittsburgh of UPMC — Pittsburgh, Pennsylvania, United States
• Intermountain Ear Nose and Throat Center — Salt Lake City, Utah, United States
• Montreal Children's Hospital — Montreal, Quebec, Canada
• Ospedale San Gerardo — Monza, Italy
• Ospedale Pediatrico Bambino Gesù — Passoscuro, Italy
• Christus Muguerza del Parque, S.A. de C.V. — Chihuahua City, Mexico
• Hospital de Cruces — Barakaldo, Spain
• Hacettepe Universitesi Tip Fakultesi Hastanesi — Ankara, Turkey (Türkiye)
• Royal Manchester Children's Hospital — Manchester, United Kingdom

Full record on ClinicalTrials.gov

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