Clinical Trial: Natural History Study of Patients With MPS IIIA

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Observational

Official Title: An Observational, Prospective, Multi-center, Natural History Study of Patients With Mucopolysaccharidosis Type IIIA (MPS IIIA)

Brief Summary: Evaluate the clinical progression in patients with MPS IIIA who are untreated with any investigational product and to obtain standardized assessments: neurocognitive, behavioral, sleep-wake habits and effect of MPS IIIA on the quality of life of patients and their families.

Detailed Summary: This is a multicenter, multinational, longitudinal, observational study in children aged up to and including 9 years, who have been diagnosed with MPS IIIA. The study will detail the natural course of MPS IIIA via standardized clinical, biochemical, neurocognitive, developmental, behavioral and imaging measures. This information is anticipated to inform the design and interpretation of future interventional studies.
Sponsor: LYSOGENE

Current Primary Outcome: The change from baseline in cognitive function using the Bayley scales of infant and toddler development third edition [ Time Frame: Baseline, and every 6 months, for up to 24 months ]

Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Change from baseline in the adaptive behavior composite standard score as measured by the Vineland Adaptive Behavior scale [ Time Frame: Baseline and every 6 months up to 24 months ]
  • Sleep disturbances measured by Actigraphy [ Time Frame: Baseline and every 3 months up to 24 months ]
  • Patient Quality of Life Questionnaires [ Time Frame: Baseline and every 6 months up to 24 months ]
  • Change from baseline in total cortical grey matter volume [ Time Frame: Baseline, 12 months, 24 months ]


Original Secondary Outcome: Same as current

Information By: LYSOGENE

Dates:
Date Received: April 15, 2016
Date Started: April 2016
Date Completion: March 2019
Last Updated: October 12, 2016
Last Verified: October 2016