Survey to Identify Burdens & Unmet Needs of Patients With Epidermolysis

Status Completed

Clinical 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.

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

Clinical Trial 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

Related Conditions & MeSH terms

Epidermolysis Bullosa

Clinical Trial Details

NCT number	NCT03158662
Study type	Observational
Source	Amicus Therapeutics
Contact
Status	Completed
Phase	N/A
Start date	April 2, 2017
Completion date	June 30, 2017

View Details

See also
Status	Clinical Trial	Phase
Completed	`NCT00380640` - The Efficacy of Trimethoprim in Wound Healing of Patients With Epidermolysis Bullosa	Phase 2
Completed	`NCT00004761` - Establishment of the National Epidermolysis Bullosa Registry	N/A
Completed	`NCT01263379` - Gene Transfer for Recessive Dystrophic Epidermolysis Bullosa	Phase 1/Phase 2
Completed	`NCT01716169` - Treatment of Chronic and Non-Chronic Wounds in Patients With Recessive Dystrophic Epidermolysis Bullosa Using Helicoll Collagen Dressings Versus Standard of Care	N/A
Withdrawn	`NCT01454687` - Grafting of Epidermolysis Bullosa Wounds Using Cultured Revertant Autologous Keratinocytes	N/A
Completed	`NCT00014729` - Phase I Study of Isotretinoin in Patients With Recessive Dystrophic Epidermolysis Bullosa	Phase 1
Recruiting	`NCT05838092` - Allogeneic ABCB5-positive Dermal Mesenchymal Stromal Cells for Treatment of Epidermolysis Bullosa (Phase III)	Phase 3
Recruiting	`NCT03269474` - Computational Drug Repurposing for All EBS Cases
Completed	`NCT03068780` - Phase III Efficacy and Safety Study of Oleogel-S10 in Epidermolysis Bullosa	Phase 3
Terminated	`NCT02090283` - Open-Label Extension Study to Evaluate the Safety of SD-101 Cream in Participants With Epidermolysis Bullosa	Phase 2
Completed	`NCT02582775` - MT2015-20: Biochemical Correction of Severe EB by Allo HSCT and Serial Donor MSCs	Phase 2
Recruiting	`NCT04213703` - A Pilot Study to Explore the Role of Gut Flora in Epidermolysis Bullosa
Completed	`NCT02384460` - ESSENCE Study: Efficacy and Safety of SD-101 Cream in Participants With Epidermolysis Bullosa	Phase 3
Terminated	`NCT02670330` - Open Label Extension Study to Evaluate the Long-term Safety of Zorblisa (SD-101-6.0) in Patients With Epidermolysis Bullosa	Phase 3
Terminated	`NCT01619670` - A Observational Study to Evaluate Apligraf(R) in Nonhealing Wounds of Subjects With Epidermolysis Bullosa	Phase 4
Recruiting	`NCT01340235` - Treatment of Dowling Maera Type of Epidermolysis Bullosa Simplex by Oral Erythromycin	Phase 3
Completed	`NCT02014376` - Study of Effectiveness and Safety of SD-101 in Participants With Epidermolysis Bullosa	Phase 2
Completed	`NCT04217538` - Observational Study of a Cohort of Patients With Hereditary Epidermolysis Bullosa
Completed	`NCT03942250` - Uses of Irradiated Human Amniotic Membrane in the Treatment of Dystrophic Epidermolysis Bullosa Patients	N/A
Completed	`NCT01033552` - Biochemical Correction of Severe EB by Allo HSCT and "Off-the-shelf" MSCs	Phase 1/Phase 2

Clinical trials are conducted in a series of steps, called phases - each phase is designed to answer a separate research question.

Phase 1: Researchers test a new drug or treatment in a small group of people for the first time to evaluate its safety, determine a safe dosage range, and identify side effects.
Phase 2: The drug or treatment is given to a larger group of people to see if it is effective and to further evaluate its safety.
Phase 3: The drug or treatment is given to large groups of people to confirm its effectiveness, monitor side effects, compare it to commonly used treatments, and collect information that will allow the drug or treatment to be used safely.
Phase 4: Studies are done after the drug or treatment has been marketed to gather information on the drug's effect in various populations and any side effects associated with long-term use.

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

Clinical Trial Summary

Clinical Trial Description

Study Design

Related Conditions & MeSH terms