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

August 23, 2017 updated by: Amicus Therapeutics

Survey to Identify Burdens and Unmet Needs of EB Patients in the US

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

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Detailed Description

Epidermolysis bullosa (EB) is a rare, often severe genetic disorder characterized by mechanical fragility and blistering or erosion of the skin, mucosa, or epithelial lining of other organs, in response to little or no apparent trauma.

EB is chronic, potentially disfiguring, and in some cases fatal. Patients with EB have painful wounds and blisters that can lead to infection and scarring. There are many genetic and symptomatic variations of EB, but all forms share the common symptom of fragile skin that blisters and tears, sometimes from the slightest friction or trauma. There is currently no approved treatment for EB. Current standard of care consists of pain management and the bandaging and cleaning of open wounds to prevent infection.

While there are a number of publications/guidance/consensus statements related to the diagnosis and management of EB from the point of view of the health-care provider/disease expert, there is a need for more research to define the key aspects of life with EB (i.e. diagnostic journey, treatment, management, daily living challenges, and overall disease burden) from the perspective of the patient.

Study Type

Observational

Enrollment (Actual)

150

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • New Jersey
      • Cranbury, New Jersey, United States, 08512
        • Amicus Therapeutics

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Sampling Method

Non-Probability Sample

Study Population

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

Description

Inclusion Criteria:

  • 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

Exclusion Criteria:

  • Patient failing to meet the inclusion criteria above

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Observational Models: Case-Only
  • Time Perspectives: Cross-Sectional

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Current quality of life burdens for EB patients
Time Frame: Outcome will be assessed immediately after subjects answer the questionnaire as this is a cross-sectional survey
Outcome will be assessed immediately after subjects answer the questionnaire as this is a cross-sectional survey

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Amicus Therapeutics

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

April 2, 2017

Primary Completion (Actual)

June 30, 2017

Study Completion (Actual)

June 30, 2017

Study Registration Dates

First Submitted

May 10, 2017

First Submitted That Met QC Criteria

May 16, 2017

First Posted (Actual)

May 18, 2017

Study Record Updates

Last Update Posted (Actual)

August 24, 2017

Last Update Submitted That Met QC Criteria

August 23, 2017

Last Verified

August 1, 2017

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • EB Survery 2017

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

Clinical Trials on Epidermolysis Bullosa

Clinical Trials on Survey

Search Similar Trials

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Mauritania

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations

3
Subscribe