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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®

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Shire
ClinicalTrials.gov Identifier:
NCT00937794
First received: July 9, 2009
Last updated: March 6, 2014
Last verified: March 2014
  Purpose

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.


Condition Intervention
Hunter Syndrome
Behavioral: Neurobehavioral testing
Other: Visual and auditory assessments

Study Type: Observational
Study Design: Observational Model: Cohort
Time Perspective: Prospective
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®

Resource links provided by NLM:


Further study details as provided by Shire:

Primary Outcome Measures:
  • To determine the neurodevelopmental status, including hearing ability, in a population of pediatric patients with Hunter syndrome who have demonstrated early cognitive impairment and who are receiving weekly infusions of Elaprase. [ Time Frame: 1 month ] [ Designated as safety issue: No ]

Enrollment: 33
Study Start Date: July 2009
Study Completion Date: August 2011
Primary Completion Date: July 2011 (Final data collection date for primary outcome measure)
Groups/Cohorts Assigned Interventions
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.
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.

  Eligibility

Ages Eligible for Study:   32 Months to 18 Years
Genders Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population

Initial patient eligibility will be based on patient age and gender. Patients must be receiving weekly IV Elaprase infusions to be eligible for enrollment.

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.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00937794

Locations
United States, North Carolina
University of North Carolina at Chapel Hill
Chapel Hill, North Carolina, United States, 27599
United Kingdom
Birmingham Children's Hospital
Birmingham, United Kingdom, B46NH
Sponsors and Collaborators
Shire
Investigators
Principal Investigator: Joseph Muenzer, MD, PhD University of North Carolina, Chapel Hill
  More Information

No publications provided

Responsible Party: Shire
ClinicalTrials.gov Identifier: NCT00937794     History of Changes
Other Study ID Numbers: HGT-HIT-050
Study First Received: July 9, 2009
Last Updated: March 6, 2014
Health Authority: United States: Food and Drug Administration

Additional relevant MeSH terms:
Mucopolysaccharidosis II
Syndrome
Carbohydrate Metabolism, Inborn Errors
Connective Tissue Diseases
Disease
Genetic Diseases, Inborn
Genetic Diseases, X-Linked
Heredodegenerative Disorders, Nervous System
Intellectual Disability
Lysosomal Storage Diseases
Mental Retardation, X-Linked
Metabolic Diseases
Metabolism, Inborn Errors
Mucinoses
Mucopolysaccharidoses
Nervous System Diseases
Neurobehavioral Manifestations
Neurologic Manifestations
Pathologic Processes

ClinicalTrials.gov processed this record on November 27, 2014