CompletedNot Applicable

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

United States92 participantsStarted 2018-01-29

Plain-language 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.

Who can participate

Age range18 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

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

What they're measuring

Characterization of Symptoms

Timeframe: 6 months from the study start date

Trial details

NCT IDNCT03440905

SponsorUniversity of South Florida

Sponsor typeOTHER

Study typeOBSERVATIONAL

Primary completion2018-11-30

View on ClinicalTrials.gov More Zellweger Spectrum trials