Working...
ClinicalTrials.gov
ClinicalTrials.gov Menu

Proxy-Reported Symptoms and Quality of Life Survey in Zellweger Spectrum Disorders

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT03440905
Recruitment Status : Completed
First Posted : February 21, 2018
Last Update Posted : January 9, 2019
Sponsor:
Collaborators:
National Institutes of Health (NIH)
Montclair State University
University of Nebraska
Information provided by (Responsible Party):
University of South Florida

Brief Summary:
The purpose of this study is to characterize the symptoms of Zellweger Spectrum Disorder (ZSD) and related peroxisome disorders, and to assess the quality of life of family caregivers (parents, stepparents, legal guardians) of patients diagnosed with ZSD or a related peroxisome disorder. All family caregivers of patients enrolled in the Rare Diseases Clinical Research Network (RDCRN) Contact Registry who are diagnosed with ZSD or a related peroxisome disorder will be invited via email to participate in this study.

Condition or disease
Zellweger Spectrum

  Show Detailed Description

Layout table for study information
Study Type : Observational
Actual Enrollment : 92 participants
Observational Model: Cohort
Time Perspective: Other
Official Title: Proxy-Reported Symptoms and Quality of Life Survey in Zellweger Spectrum Disorders
Actual Study Start Date : January 29, 2018
Actual Primary Completion Date : November 30, 2018
Actual Study Completion Date : November 30, 2018





Primary Outcome Measures :
  1. Characterization of Symptoms [ Time Frame: 6 months from the study start date ]
    To characterize the symptoms of Zellweger spectrum disorder (ZSD) and related peroxisome disorders through family caregiver-reported measures using a customized survey tool.


Secondary Outcome Measures :
  1. Quality of Life Assessment [ Time Frame: 6 months from the study start date ]
    To assess quality of life for family caregivers of children with ZSD and related peroxisome disorders through the domains of communication, medical care, emotional distress and well-being, role function, family interaction, parenting, and disability-related support, using the validated Pediatric Inventory for Parents (PIP).

  2. Quality of Life Assessment [ Time Frame: 6 months from the study start date ]
    To assess quality of life for family caregivers of children with ZSD and related peroxisome disorders through the domains of communication, medical care, emotional distress and well-being, role function, family interaction, parenting, and disability-related support, using the FQOL Survey.


Other Outcome Measures:
  1. Quality of Life Caregiver-reported Comparison [ Time Frame: 6 months from the study start date ]
    To compare family caregiver-reported quality of life between male and female family caregivers of children with ZSD and related peroxisome disorders through the domains of communication, medical care, emotional distress and well-being, role function, family interaction, parenting, and disability-related support, using the validated Pediatric Inventory for Parents (PIP).

  2. Quality of Life Caregiver-reported Comparison [ Time Frame: 6 months from the study start date ]
    To compare family caregiver-reported quality of life between male and female family caregivers of children with ZSD and related peroxisome disorders through the domains of communication, medical care, emotional distress and well-being, role function, family interaction, parenting, and disability-related support, using the FQOL Survey.



Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Layout table for eligibility information
Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   Yes
Sampling Method:   Non-Probability Sample
Study Population
Family caregivers (parents, stepparents, legal guardians) of patients (living or deceased) with ZSD or related peroxisome disorder diagnosis
Criteria

Inclusion Criteria:

  • Family caregiver (parents, stepparents, legal guardians) of child (living or deceased) diagnosed with ZSD, acyl CoA oxidase (ACOX) deficiency or D-bifunctional protein deficiency (DBPD)
  • Family caregiver is able to complete surveys

Exclusion Criteria:

  • Inability of family caregiver to provide informed consent and complete survey
  • Parents/primary caregivers of children who have not been diagnosed with ZSD, acyl CoA oxidase deficiency and D-bifunctional protein deficiency

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03440905


Locations
Layout table for location information
United States, Florida
University of South Florida
Tampa, Florida, United States, 33512
Sponsors and Collaborators
University of South Florida
National Institutes of Health (NIH)
Montclair State University
University of Nebraska

Publications:

Layout table for additonal information
Responsible Party: University of South Florida
ClinicalTrials.gov Identifier: NCT03440905     History of Changes
Other Study ID Numbers: STAIR 7010
First Posted: February 21, 2018    Key Record Dates
Last Update Posted: January 9, 2019
Last Verified: January 2019

Layout table for additional information
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:
Layout table for MeSH terms
Zellweger Syndrome
Liver Diseases
Digestive System Diseases
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Kidney Diseases
Urologic Diseases
Abnormalities, Multiple
Congenital Abnormalities
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Peroxisomal Disorders
Metabolic Diseases