Clinical Trial: Long-term Evaluation on Height and Weight in Patients With MPS II Who Started Treatment at < 6 Years of Age

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional

Official Title: A Long-Term, Open-Label, Multicenter, Phase IV Study to Assess Longitudinal Changes on Height and Weight in Patients With MPS II Who Are Receiving Elaprase and Started Tre

Brief Summary: This long-term study will provide Elaprase treatment to children enrolled in this study and will utilize data from both enrolled patients and Hunter Outcome Survey (HOS) patient registry data to conduct the primary growth analysis to assess changes in height and weight in patients with Mucopolysaccharidosis II (Hunter syndrome) MPS II.

Detailed Summary:
Sponsor: Shire

Current Primary Outcome:

  • Change in height [ Time Frame: Screening to End-of-Study (5-10 years) ]
  • Change in weight Z-scores [ Time Frame: Screening to End-of-Study (5-10 years) ]
  • Change in neurological examination to evaluate long-term safety in patient [ Time Frame: Screening to End-of-Study (5-10 years) ]
  • Number of participants with adverse events as measured by type, severity, and relationship to treatment [ Time Frame: Screening to End-of-Study (5-10 years) ]
  • Change in clinical laboratory testings as measured by urinalysis to evaluate long-term safety in patients [ Time Frame: Screening to End-of-Study (5-10 years) ]
  • Change in clinical laboratory testings as measured by serum chemistry to evaluate long-term safety in patients [ Time Frame: Screening to End-of-Study (5-10 years) ]
  • Change in clinical laboratory testings as measured by hematology to evaluate long-term safety in patients [ Time Frame: Screening to End-of-Study (5-10 years) ]


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Urinary glycosaminoglycans (uGAG) levels normalized to urine creatinine [ Time Frame: Screening to End-of-Study (5-10 years) ]
  • Normalized uGAG divided by upper limit of normal for age (uGAG/ULN) every 12 months [ Time Frame: Baseline to End-of-Study (5-10 years) ]
  • Joint mobility, as measured by Joint Range of Motion (JROM) scores, including global, upper-limb, and lower-limb joint scores [ Time Frame: Screening to End-of-Study (5-10 years) ]
  • Distance walked, as measured by the Six Minute Walk Test (6MWT) [ Time Frame: Screening to End-of-Study (5-10 years) ]
  • Quality of Life, as measured by the Hunter-Syndrome Functional Outcome in Clinical Understanding Scale [ Time Frame: Screening to End-of-Study (5-10 years) ]
  • Impact of illness on ability to function in daily life, as measured by the Childhood Health Assessment Questionnaire (CHAQ Parent Report) [ Time Frame: Screening to End-of-Study (5-10 years) ]
  • Adaptive behavior, as measured by the Vineland Adaptive Behavior Scales (VABS II) [ Time Frame: Screening to End-of-Study (5-10 years) ]
  • Change in anti-idursulfase antibodies in serum [ Time Frame: Screening to End-of-Study (5-10 years) ]
    number and percentage of patients testing anti-idursulfase antibody positive and negative at each time point


Original Secondary Outcome: Same as current

Information By: Shire

Dates:
Date Received: May 20, 2015
Date Started: June 2015
Date Completion: August 2022
Last Updated: March 10, 2017
Last Verified: March 2017