Epidermolysis BullosaNCT03158662
Survey to Identify Burdens and Unmet Needs of Patients With Epidermolysis Bullosa
An observational study of Epidermolysis Bullosa, run by Amicus Therapeutics at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Epidermolysis Bullosa
- What is being tested?
- Survey
- How many people
- 150 participants
- Where
- 1 siteUnited States
- Study dates
- 2 Apr 2017to 30 Jun 2017
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: EB Patients 18 years of age or older or caregivers of patients under 18 years of age
Who may be able to join
- 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
Who cannot join
- Patient failing to meet the inclusion criteria above
Trial locations and contacts
1 site in 1 country.
About this study
Official title: 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
Read the 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.
- Conditions
- Epidermolysis Bullosa
- Being tested
- Survey
- Keywords
- Epidermolysis Bullosa, Recessive Dystrophic, Simplex, Junctional Non-Herlitz
- Medical subject headings
- Epidermolysis Bullosa
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SurveyOther
Survey Study
What is being measured
The primary outcome is the main question the study is designed to answer.
Current quality of life burdens for EB patients
Measured: Outcome will be assessed immediately after subjects answer the questionnaire as this is a cross-sectional survey
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 May 2017
- Study start2 Apr 2017
- Primary completion30 Jun 2017
- Study completion30 Jun 2017
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Epidermolysis BullosaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Case only
- Time perspective
- Cross-sectional
- Enrolment
- 150 participants (actual)
Sponsor and investigators
- Lead sponsor
- Amicus Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03158662
- Sponsor's protocol ID
- EB Survery 2017
- Status last verified
- Aug 2017
- First submitted
- 10 May 2017
- Last updated
- 24 Aug 2017
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 24 Aug 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Survey to Identify Burdens and Unmet Needs of EB Patients in the US
- ClinicalTrials.gov ID
- NCT03158662
- Acronym / sponsor ID
- EB Survery 2017
- Conditions
- Epidermolysis Bullosa
- Keywords
- Epidermolysis Bullosa, Recessive Dystrophic, Simplex, Junctional Non-Herlitz
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 150 participants (actual)
- Lead sponsor
- Amicus TherapeuticsIndustry (pharma, biotech, devices)
- Study start
- 2 Apr 2017 (actual)
- Primary completion
- 30 Jun 2017 (actual)
- Study completion
- 30 Jun 2017 (actual)
- First posted
- 18 May 2017 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Observational model
- Case only
- Time perspective
- Cross-sectional
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- EB Patients 18 years of age or older or caregivers of patients under 18 years of age
Key inclusion criteria (6 listed)
- 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.
Key exclusion criteria (1 listed)
- Patient failing to meet the inclusion criteria above
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| SurveySurvey Study | Other | – |
Primary outcome
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
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 Aug 2017.
Related trials for Epidermolysis Bullosa
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Efficacy and Safety Study to Evaluate SD-101 in Epidermolysis Bullosa
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A Study to Evaluate ENERGI-F703EB Cream in Epidermolysis Bullosa
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A Study to Assess the Incidence of Skin Cancers in Patients With Epidermolysis Bullosa Receiving Filsuvez
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Epidermolysis Bullosa, run by Amicus Therapeutics at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 24 Aug 2017. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 24 Aug 2017.