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

  • Change in physical and neurological examination to evaluate long-term safety in patients [ Time Frame: Baseline to month 60 ]
  • Change in height and weight [ Time Frame: Baseline to month 60 ]
  • Change in head circumference [ Time Frame: Baseline to month 60 ]
  • Change in auditory capacity for hearing assessment [ Time Frame: Baseline to month 60 ]
  • Number of participants with adverse events as measured by type, severity, and relationship to treatment [ Time Frame: Baseline to month 60 ]
  • Change in vital signs [ Time Frame: Baseline to month 60 ]
  • Change in 12-lead ECG recordings [ Time Frame: Baseline to month 60 ]
  • Change in clinical laboratory testings as measured by serum chemistry, hematology, and urinalysis to evaluate long-term safety in patients [ Time Frame: Baseline to month 60 ]
  • Change in cerebrospinal fluid laboratory standard chemistry to evaluate long-term safety in patients [ Time Frame: Baseline to month 60 ]
  • Change in cerebrospinal fluid laboratory parameters to evaluate long-term safety in patients [ Time Frame: Baseline to month 60 ]
  • Change in anti-idursulfase antibodies in CSF and serum [ Time Frame: Baseline to month 60 ]
  • Change in urine GAG and creatinine [ Time Frame: Baseline to month 60 ]
  • Change in serum albumin [ Time Frame: Ba

    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