Observational Study to Evaluate Neurodevelopmental Status in Pediatric Patients With Hunter Syndrome (MPS II)

Hunter Syndrome Clinical Trial

Official title:

A Prospective, Longitudinal, Observational Study to Evaluate Neurodevelopmental Status in Pediatric Patients With Hunter Syndrome (MPS II)

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01822184
• Other study ID #: HGT-HIT-090
• Status: Completed
• Start date: January 18, 2013
• Est. completion date: October 5, 2016

Study information

• Verified date: March 2021
• Source: Takeda
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial Summary

Hunter syndrome (Mucopolysaccharidosis II, [MPS II]) is a rare, genetically linked lysosomal storage disease (LSD) caused by deficiency of the enzyme, iduronate-2-sulfatase (I2S). Most MPS II patients will present with some degree of neurodevelopmental involvement, ranging from severe cognitive impairment and behavioral problems to mildly impaired cognition. This is an observational study; no investigational treatment will be administered. The primary objective of this study is to evaluate the neurodevelopmental status of pediatric patients with MPS II over time and to gain information to guide future treatment studies in this patient population.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 100
• Est. completion date: October 5, 2016
• Est. primary completion date: October 5, 2016
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 2 Years to 18 Years

Eligibility

Inclusion Criteria:

Patients must meet all of the following criteria to be considered eligible for enrollment:

1. a. The patient has a deficiency in iduronate-2-sulfatase enzyme activity AND b. The patient has a documented mutation in the iduronate-2-sulfatase gene. OR c. The patient has a normal enzyme activity level of one other sulfatase

2. The patient is male, and is at least 2 years of age and less than 18 years of age at the time of informed consent.

3. The patient must have sufficient auditory capacity at enrollment, with or without hearing aids, in the Investigator's judgment to complete the required protocol testing, and be compliant with wearing the aids on scheduled study visits.

4. The patient, patient's parent(s), or legally authorized guardian(s) has voluntarily signed an Institutional Review Board / Independent Ethics Committee-approved informed consent and/or assent form(s), as applicable.

Exclusion Criteria:

Patients who meet any of the following criteria will be excluded from the study.

1. The patient has clinically significant non-Hunter syndrome-related CNS involvement or medical or psychiatric comorbidity(ies) which, in the investigator's judgment, may interfere with the accurate administration and interpretation of protocol assessments, affect study data, or confound the integrity of study results.

2. The patient has a general conceptual ability score (GCA) or a developmental quotient on the cognitive scale below 55 at Screening.

3. The patient is participating in an interventional clinical trial or has participated in an interventional clinical trial within 30 days prior to enrollment; participation in non interventional observational studies is permitted.

Related Conditions & MeSH terms

• Hunter Syndrome
• Mucopolysaccharidoses
• Mucopolysaccharidosis (MPS)
• Mucopolysaccharidosis II
• Syndrome

Locations

Country	Name	City	State
Argentina	Hospital Universitario Austral	Pilar	Buenos Aires
Mexico	Instituto Nacional De Pediatria	Mexico City
Spain	Hospital Infantil Universitario	Madrid
United Kingdom	Central Manchester University Hospitals NHS Foundation Trust Willink Biochemical Genetics Unit, St. Mary's Hospital	Manchester	M13 9wl
United States	University of North Carolina Division of Genetics and Metabolism	Chapel Hill	North Carolina
United States	Ann & Robert H. Lurie Children's Hospital of Chicago	Chicago	Illinois
United States	Childrens Hospital & Research Center Oakland	Oakland	California

Sponsors (1)

Lead Sponsor	Collaborator
Shire

Countries where clinical trial is conducted

United States,  Argentina,  Mexico,  Spain,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Neurodevelopmental parameters of cognitive function over time in pediatric patients with MPS II		24 months
Primary	Neurodevelopmental parameters of adaptive function over time in pediatric patients with MPS II		24 months
Secondary	Reported adverse events	Type and severity measurements	24 months
Secondary	Medication usage		24 months
Secondary	Quality of life		24 months

External Links

•   To obtain more information on the study, click here/on this link.