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®

NCT ID: NCT00937794

Last Updated: 2021-06-14

Basic Information

• Recruitment Status: COMPLETED
• Total Enrollment: 33 participants
• Study Classification: OBSERVATIONAL
• Study Start Date: 2009-07-02
• Study Completion Date: 2011-07-13

Brief Summary

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.

Conditions

Hunter Syndrome

Study Design

• Observational Model Type: COHORT
• Study Time Perspective: PROSPECTIVE

Study Groups

No treatment

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.

Neurobehavioral testing

Intervention Type: BEHAVIORAL

If the patient is found to be eligible after completion of a telephone interview, he will undergo further testing to assess his neurodevelopmental status using a standardized battery of neurobehavioral testing.

Visual and auditory assessments

Intervention Type: OTHER

If the patient is found to be eligible after completion of a telephone interview, he will undergo further testing to evaluate his visual and auditory function.

Interventions

Neurobehavioral testing

If the patient is found to be eligible after completion of a telephone interview, he will undergo further testing to assess his neurodevelopmental status using a standardized battery of neurobehavioral testing.

Intervention Type: BEHAVIORAL

Visual and auditory assessments

If the patient is found to be eligible after completion of a telephone interview, he will undergo further testing to evaluate his visual and auditory function.

Intervention Type: OTHER

Eligibility Criteria

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.

• Minimum Eligible Age: 32 Months
• Maximum Eligible Age: 18 Years
• Eligible Sex: MALE
• Accepts Healthy Volunteers: No

Sponsors

Shire

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Study Director

Role: STUDY_DIRECTOR

Takeda

Locations

University of North Carolina at Chapel Hill

Chapel Hill, North Carolina, United States

Birmingham Children's Hospital

Birmingham, , United Kingdom

Countries

United States; United Kingdom

Other Identifiers

HGT-HIT-050

Identifier Type: -

Identifier Source: org_study_id