Clinical Trial: Psychological Concomitants of Morquio Syndrome (The MAP Study)

Study Status: Completed
Recruit Status: Completed
Study Type: Observational

Official Title: Psychological Concomitants of Morquio Syndrome

Brief Summary:

Mucopolysaccharidosis IV, also known as MPS IV or Morquio disease, is a rare autosomal recessive genetic lysosomal storage disorder. Research thus far regarding lysosomal storage diseases (LSDs) in general, including Morquio, has primarily focused on exploring the causes of and finding a treatment for the physical aspects of the various diseases. Less attention has been paid to the psychological or emotional toll of these diseases, whether they are direct symptoms of the diseases themselves or reactions to living with a chronic progressive disease.

It is well established in the health psychology literature, however, that the interaction between our physical health and our psychological health is bidirectional; that is, just as our physical health affects us emotionally (e.g. chronic pain can contribute to depression), so can our psychological health affect us physically (e.g. anxiety can contribute to feelings of chest pain). It is thus critically important to pay attention to the emotional and psychological symptoms associated with all lysosomal storage diseases, including Morquio, and expand our treatment standard of care to include mental health treatment, if necessary.

The first step in understanding and treating psychological conditions in Morquio disease is determining the natural occurrence of psychological symptoms in this population in comparison with non-medical populations. As little has been done in this regard, a pilot study documenting the occurrence rate of psychological issues and overall quality of life in patients with Morquio is the first item in order and will be the focus of this study.

Approximately 20 patients with Morquio disease will be invited to participate, recruited through Emory's Lysosomal Storage Disease Center, as well as through attendance

Detailed Summary:
Sponsor: Nadia Ali, PhD

Current Primary Outcome: ASEBA Self-Report [ Time Frame: At enrollment, as a single-timepoint only ]

Self-report questionnaire assessing psychological and adaptive functioning well-being


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Brief Pain Inventory [ Time Frame: At enrollment, as a single-timepoint only ]
    Self-report measure of subjective pain levels and interference of pain in daily functioning
  • SF-36 [ Time Frame: At enrollment, as a single-timepoint only ]
    Brief self-report measure of quality of life


Original Secondary Outcome: Same as current

Information By: Emory University

Dates:
Date Received: December 13, 2012
Date Started: July 2012
Date Completion:
Last Updated: December 2, 2013
Last Verified: December 2013