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®

Completed

• Conditions: Hunter Syndrome
• Interventions: Behavioral: Neurobehavioral testing; Other: Visual and auditory assessments

• Registration Number: NCT00937794
• Lead Sponsor: Shire

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.

Detailed Description

Not available

Study Timeline

• Study Start: 2009-07-02
• Study First Submitted: 2009-07-09
• Study First Submitted QC: 2009-07-10
• Study First Posted: 2009-07-13
• Primary Completion: 2011-07-13
• Study Completion: 2011-07-13
• Results First Submitted: 2015-12-18
• Results First Submitted QC: 2016-04-11
• Results First Posted: 2016-05-16
• Status Verified: 2021-05
• Last Update Submitted: 2021-05-18
• Last Update Posted: 2021-06-14

Recruitment & Eligibility

• Status: COMPLETED
• Sex: Male
• Target Recruitment: 33

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.

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
No treatment	Neurobehavioral testing	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.
No treatment	Visual and auditory assessments	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.

Primary Outcome Measures

Name	Time	Method
Number of Participants With a Score of at Least 90 on The General Conceptual Ability (GCA) Sub-Scale of The Differential Ability Scale (DAS)	1 month	The GCA sub-scale of the DAS, Second Edition (DAS-II) was used to obtain a general measure of cognitive ability.The maximum score is 120, with a higher score indicating greater cognitive ability. A score of 100 is considered an average score.
Number of Participants Who Were Screened For The Follow-On Study With an Investigational Agent	1 month	Standardized tests were used to identify patients who were receiving treatment with Elaprase, had cognitive impairment, and were suitable to participate in the follow-on clinical study (HGT-HIT-045). Assessments included: 1-Cognition: The Differential Ability Scale, Second Edition (DAS-II) or the Bayley Scales of Infant Development, Third Edition (BSID-III); 2-Adaptive Behavior: The Scale of Independent Behavior-Revised (SIB-R); 3-Executive Function: The Behavior Rating Inventory of Executive Function-Preschool version (BRIEF-P) for children or the Behavior Rating Inventory of Executive Function (BRIEF) for children less than or ≥6 years of age, respectively; 4-Motor: The Peabody Developmental Motor Scales-2 (PDMS-2) or the Bruininks-Oseretsky Test of Motor Proficiency, Second Edition (BOT-2) for children less than or ≥6 years of age, respectively.

Trial Locations

Locations (2)

University of North Carolina at Chapel Hill; 🇺🇸 Chapel Hill, North Carolina, United States

Birmingham Children's Hospital; 🇬🇧 Birmingham, United Kingdom