Survey to Identify Burdens and Unmet Needs of Patients With Epidermolysis Bullosa

NCT03158662 | Completed

• 1 other identifier: EB Survery 2017
• Study Type: observational
• Target: 150
• Locations: 1 country
• Sites: 1

Brief Summary

This survey intends to collect information on key aspects of life with epidermolysis bullosa (EB), including diagnostic journey, treatment, management, daily living challenges, and overall psycho-social, socio-economic, academic and family impact. Objectives:

• To understand the unmet needs for people living with EB in the US
• To assess the differences/similarities in the management/treatment of EB patients (including wound care, symptom management and other issues)
• To assess the EB patients' and caregivers' perceptions of current management/treatment
• To assess the challenges and the burden of daily living with EB
• To understand EB diagnostic journey (the time to diagnosis and by what type of healthcare provider)
• To identify professional disciplines involved in the diagnosis and management of EB
• To understand the psycho-social, socio-economic, academic, and family impact of EB

Conditions

Epidermolysis Bullosa

Keywords

Epidermolysis Bullosa; Recessive Dystrophic; Simplex; Junctional Non-Herlitz

Outcome Measures

Primary Outcomes (1)

• Current quality of life burdens for EB patients

  Outcome will be assessed immediately after subjects answer the questionnaire as this is a cross-sectional survey

Interventions

Survey OTHER

Survey Study

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

EB Patients 18 years of age or older or caregivers of patients under 18 years of age

You may qualify if:

• Confirmed diagnosis of EB including subtype
• Resident of the US
• Informed Consent
• Patient must be 18 years or older
• If the survey is done by a caregiver, they must be a parent or legal guardian and must be 18 years or older to answer on behalf of a patient under 18 years or on behalf of a patient 18 years or older who is unable to answer for themselves.
• Must participate in and complete a one hour telephone interview

Sponsors & Collaborators

• Amicus Therapeutics: lead

Study Sites (1)

Amicus Therapeutics

Cranbury, New Jersey, 08512, United States

Location

MeSH Terms

Conditions

Epidermolysis Bullosa

Interventions

Surveys and Questionnaires

Condition Hierarchy (Ancestors)

Skin Abnormalities; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Skin Diseases, Genetic; Genetic Diseases, Inborn; Skin Diseases; Skin and Connective Tissue Diseases; Skin Diseases, Vesiculobullous

Intervention Hierarchy (Ancestors)

Data Collection; Epidemiologic Methods; Investigative Techniques; Health Care Evaluation Mechanisms; Quality of Health Care; Health Care Quality, Access, and Evaluation; Public Health; Environment and Public Health

Study Design

• Study Type: observational
• Observational Model: CASE ONLY
• Time Perspective: CROSS SECTIONAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 10, 2017
• First Posted: May 18, 2017
• Study Start: April 2, 2017
• Primary Completion: June 30, 2017
• Study Completion: June 30, 2017
• Last Updated: August 24, 2017

Record last verified: 2017-08

Locations

US (1)