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®

Hunter Syndrome Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00937794
• Other study ID #: HGT-HIT-050
• Status: Completed
• Start date: July 2, 2009
• Est. completion date: July 13, 2011

Study information

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

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 33
• Est. completion date: July 13, 2011
• Est. primary completion date: July 13, 2011
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 32 Months to 18 Years

Eligibility

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.

Related Conditions & MeSH terms

• Hunter Syndrome
• Mucopolysaccharidosis II
• Syndrome

Intervention

Behavioral:
• 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.

Other:
• 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.

Locations

Country	Name	City	State
United Kingdom	Birmingham Children's Hospital	Birmingham
United States	University of North Carolina at Chapel Hill	Chapel Hill	North Carolina

Sponsors (1)

Lead Sponsor	Collaborator
Shire

Countries where clinical trial is conducted

United States,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Number of Participants Who Were Screened For The Follow-On Study With an Investigational Agent	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.	1 month
Primary	Number of Participants With a Score of at Least 90 on The General Conceptual Ability (GCA) Sub-Scale of The Differential Ability Scale (DAS)	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.	1 month