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

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Brief Title

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

Official Title

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

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
    

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


Primary Outcome

Current quality of life burdens for EB patients


Condition

Epidermolysis Bullosa

Intervention

Survey


Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Other

Estimated Enrollment

150

Start Date

April 2, 2017

Completion Date

June 30, 2017

Primary Completion Date

June 30, 2017

Eligibility Criteria

        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
      

Gender

All

Ages

18 Years - N/A

Accepts Healthy Volunteers

No

Contacts

, , 

Location Countries

United States

Location Countries

United States

Administrative Informations


NCT ID

NCT03158662

Organization ID

EB Survery 2017


Responsible Party

Sponsor

Study Sponsor

Amicus Therapeutics


Study Sponsor

, , 


Verification Date

August 2017