Clinical Trial: Long-Term Safety and Clinical Outcomes of Idursulfase IT and Elaprase Treatment in Pediatric Patients With Hunter Syndrome That Have Completed Study HGT-HIT-094
Study Status: Enrolling by invitation
Recruit Status: Enrolling by invitation
Study Type: Interventional
Official Title: An Open Label Extension of Study HGT-HIT-094 Evaluating Long Term Safety and Clinical Outcomes of Intrathecal Idursulfase Administered in Conjunction With Elaprase® in Patients With
Original Primary Outcome:
- Change in physical and neurological examination to evaluate long-term safety in patients [ Time Frame: Baseline to week 148 ]
- Change in height and weight [ Time Frame: Baseline to week 148 ]
- Change in head circumference [ Time Frame: Baseline to week 148 ]
- Change in auditory capacity for hearing assessment [ Time Frame: Baseline to week 148 ]
- Number of participants with adverse events as measured by type, severity, and relationship to treatment [ Time Frame: Baseline to week 148 ]
- Change in vital signs [ Time Frame: Baseline to week 148 ]
- Change in 12-lead ECG recordings [ Time Frame: Baseline to week 148 ]
- Change in clinical laboratory testings as measured by serum chemistry, hematology, and urinalysis to evaluate long-term safety in patients [ Time Frame: Baseline to week 148 ]
- Change in cerebrospinal fluid laboratory standard chemistry to evaluate long-term safety in patients [ Time Frame: Baseline to week 148 ]
- Change in cerebrospinal fluid laboratory parameters to evaluate long-term safety in patients [ Time Frame: Baseline to week 148 ]
- Change in anti-idursulfase antibodies in CSF and serum [ Time Frame: Baseline to week 148 ]
- Change in urine GAG and creatinine [ Time Frame: Baseline to week 148 ]
- Change in serum albumin [ Time Frame: Ba
Current Secondary Outcome:
- Change in standard scores in cluster areas of the DAS-II / BAS-II [ Time Frame: Baseline to month 60 ]
- Change in standard scores of the VABS-II domains [ Time Frame: Baseline to month 60 ]
- Change in T-scores for the subtests of the DAS-II [ Time Frame: Baseline to month 60 ]
- Change in V-scale scores of the VABS-II subdomains [ Time Frame: Baseline to month 60 ]
- Change in observed maladaptive levels of the VABS-II Maladaptive Behavior Index and its subscales [ Time Frame: Baseline to month 60 ]
- Change in brain structure volume as measured by magnetic resonance imagine (MRI) [ Time Frame: Baseline to month 60 ]
Original Secondary Outcome:
- Change in standard scores in cluster areas of the DAS-II / BAS-II [ Time Frame: Baseline to week 148 ]
- Change in standard scores of the VABS-II domains [ Time Frame: Baseline to week148 ]
- Change in T-scores for the subtests of the DAS-II [ Time Frame: Baseline to week 148 ]
- Change in V-scale scores of the VABS-II subdomains [ Time Frame: Baseline to week 148 ]
- Change in observed maladaptive levels of the VABS-II Maladaptive Behavior Index and its subscales [ Time Frame: Baseline to week 148 ]
- Change in brain structure volume as measured by magnetic resonance imagine (MRI) [ Time Frame: Baseline to week 148 ]
Information By: Shire
Dates:
Date Received: April 1, 2015
Date Started: April 2015
Date Completion: September 2022
Last Updated: March 28, 2017
Last Verified: March 2017