Screening Study to Identify Pediatric Patients With Hunter Syndrome Who Demonstrate Evidence of Central Nervous System (CNS) Involvement and Who Are Currently Receiving Treatment With Elaprase®

Shire

Status: Completed

Conditions: Hunter Syndrome

Treatments: Other: Visual and auditory assessments; Behavioral: Neurobehavioral testing

Study type: Observational

Funder types: Industry

Identifiers

NCT00937794

HGT-HIT-050

Details and patient eligibility

About

This study is being conducted to identify pediatric patients with Hunter syndrome who have neurodevelopmental disease characteristics, who are currently receiving treatment with Elaprase, and who may be suitable to participate in a clinical study with an investigational agent.

Enrollment: 33 patients

Sex: Male

Ages: 32 months to 18 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

1. The patient is male and is ≥3 and <18 years of age
2. The patient is currently receiving weekly IV infusions of Elaprase.
3. The patient, patient's parent(s), or legally authorized guardian(s) has voluntarily signed an Institutional Review Board / Independent Ethics Committee-approved informed consent form after all relevant aspects of the study have been explained and discussed with the patient. The guardians' consent and subject's assent, as relevant, must be obtained.

Exclusion criteria

1. The patient has a CNS shunt.
2. The patient has received a hematopoietic stem cell transplant.
3. The patient is currently enrolled in a clinical trial.
4. The patient has a significant medical or psychiatric comorbidity(ies) that might affect study data or confound the integrity of study results.

Trial design

33 participants in 1 patient group

No treatment

Description:

This is a screening study designed to evaluate the behavioral, physical, and neurodevelopmental status in pediatric patients with Hunter syndrome who have early signs and symptoms of CNS involvement and who are currently receiving treatment with Elaprase.

Treatment:

Other: Visual and auditory assessments

Behavioral: Neurobehavioral testing