The Natural History of Wounds in Patients With Dystrophic Epidermolysis Bullosa (DEB)

September 10, 2021 updated by: Krystal Biotech, Inc.

A Prospective Study to Evaluate the Natural History of Wounds in Patients With Dystrophic Epidermolysis Bullosa (DEB)

This study is a non-interventional, observational study that will evaluate the natural history of wounds in patients with Dystrophic Epidermolysis Bullosa (DEB) for inclusion into the Krystal Biotech Phase III protocol of B-VEC (previously KB103). Wound recurrence and wound size will be evaluated for up to four months.

Study Overview

Status

Withdrawn

Conditions

Detailed Description

The objective of this study is to analyze the selected wounds' natural history prior to including the selected wounds in the evaluation of safety and efficacy in Krystal Biotech's Phase III protocol of B-VEC.

Subjects will be enrolled upon obtaining consent and meeting eligibility criteria.

Patient Screening is done on-site, followed by the imaging of at least 1 wound, but up to 15 wounds. All remaining imaging will be performed remotely with a smartphone or tablet application.

Patients are on-trial for approximately four months.

Study Type

Observational

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
    • California
      • Redwood City, California, United States, 94063
        • Stanford University

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

10 months and older (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Sampling Method

Non-Probability Sample

Study Population

Patients must be 6 months of age or older, and have a diagnosis of DEB based on genetic analysis or histologic criteria. In addition, patients must have at least 1 wound that is suitable for imaging in investigator's opinion at time of screening.

Description

Inclusion Criteria:

  • Written informed consent is provided. Patients 18 years of age and older, and parent(s)/legal guardian(s) of patients younger than 18 years of age, must provide written informed consent prior to participating in the study; additionally, informed assent will be obtained from patients younger than 18 years of age as specified by local requirements.
  • Patient must have a documented diagnosis of DEB based on genetic analysis showing a mutation in COL7A1 or, in the opinion of the investigator, a confirmed diagnosis of DEB based on histologic criteria (antigen mapping or electron microscopy).
  • Age: 6 months and older
  • Patient must have at least 1 wound that is suitable for imaging, in the opinion of the investigator, at the time of enrollment.
  • Patient is willing and able to undergo the protocol-specified procedures.

Exclusion Criteria:

  • In the opinion of the investigator, inclusion poses an unacceptable risk to the patient or interpretation of these study data.

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: Prospective

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Change in wound surface area
Time Frame: Up to four months
Percent change in wound surface area from baseline will be measured from images uploaded to an imaging application.
Up to four months

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Time to Closure
Time Frame: Up to four months
The time it takes for a wound to completely close.
Up to four months
Duration of wound closure
Time Frame: Up to four months
If a closure occurs, the time a wound remains epithelialized after the first, and any subsequent closures.
Up to four months

Collaborators and Investigators

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

Sponsor

Krystal Biotech, Inc.

Collaborators

Stanford University

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 (Anticipated)

January 1, 2020

Primary Completion (Actual)

February 24, 2020

Study Completion (Actual)

February 24, 2020

Study Registration Dates

First Submitted

December 23, 2019

First Submitted That Met QC Criteria

December 26, 2019

First Posted (Actual)

December 30, 2019

Study Record Updates

Last Update Posted (Actual)

September 17, 2021

Last Update Submitted That Met QC Criteria

September 10, 2021

Last Verified

September 1, 2021

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • KB-WM-01

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

No

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.

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