Dystrophic Epidermolysis BullosaNCT04214002
The Natural History of Wounds in Patients With Dystrophic Epidermolysis Bullosa (DEB)
An observational study of Dystrophic Epidermolysis Bullosa and DEB - Dystrophic Epidermolysis Bullosa, run by Krystal Biotech, Inc. at 1 site in United States.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 6 months and older
- Conditions the study is for
- Dystrophic Epidermolysis Bullosa, DEB - Dystrophic Epidermolysis Bullosa
- How many people
- 0 participants
- Where
- 1 siteUnited States
- Study dates
- Jan 2020to 24 Feb 2020
- 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 6 months and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
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.
Who may be able to join
- 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.
Who cannot join
- In the opinion of the investigator, inclusion poses an unacceptable risk to the patient or interpretation of these study data.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: 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.
Read the 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.
- Keywords
- DEB, Dystrophic Epidermolysis Bullosa, COL7A1
- Medical subject headings
- Epidermolysis Bullosa Dystrophica
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in wound surface area
Percent change in wound surface area from baseline will be measured from images uploaded to an imaging application.
Measured: Up to four months
Secondary outcomes (2)
Time to Closure
The time it takes for a wound to completely close.
Measured: Up to four months
Duration of wound closure
If a closure occurs, the time a wound remains epithelialized after the first, and any subsequent closures.
Measured: Up to four months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted30 Dec 2019
- Study startJan 2020 (estimated)
- Primary completion24 Feb 2020
- Study completion24 Feb 2020
Why it stopped: Due to the COVID-19 Pandemic, the study was withdrawn.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Dystrophic Epidermolysis BullosaStudies of the same condition, open and under way.
- More trials run by Krystal Biotech, Inc.The sponsor's other registered studies.
- 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
- Prospective
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Krystal Biotech, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Stanford University
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04214002
- Sponsor's protocol ID
- KB-WM-01
- Status last verified
- Sep 2021
- First submitted
- 23 Dec 2019
- Last updated
- 17 Sep 2021
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 17 Sep 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Prospective Study to Evaluate the Natural History of Wounds in Patients With Dystrophic Epidermolysis Bullosa (DEB)
- ClinicalTrials.gov ID
- NCT04214002
- Acronym / sponsor ID
- KB-WM-01
- Keywords
- DEB, Dystrophic Epidermolysis Bullosa, COL7A1
- Study type
- Observational
- Recruitment status
- WithdrawnReason stopped: Due to the COVID-19 Pandemic, the study was withdrawn.
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Krystal Biotech, Inc.Industry (pharma, biotech, devices)
- Collaborators
- Stanford University
- Study start
- Jan 2020 (estimated)
- Primary completion
- 24 Feb 2020 (actual)
- Study completion
- 24 Feb 2020 (actual)
- First posted
- 30 Dec 2019 (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 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.
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 design
- Observational model
- Case only
- Time perspective
- Prospective
Who may qualify
- Ages
- 6 months and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- 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.
Key inclusion criteria (5 listed)
- 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.
Key exclusion criteria (1 listed)
- In the opinion of the investigator, inclusion poses an unacceptable risk to the patient or interpretation of these study data.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
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.
2 secondary outcomes
Time to Closure
Time frame: Up to four months
Duration of wound closure
Time frame: Up to four months
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 Sep 2021.
- Individual participant dataSharing planned: No
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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 Dystrophic Epidermolysis Bullosa and DEB - Dystrophic Epidermolysis Bullosa, run by Krystal Biotech, Inc. 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 5 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: withdrawn, as of the sponsor's last update on 17 Sep 2021. 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 was withdrawn before enrolling participants, so there are no results.
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?
Change in wound surface area (time frame: Up to four months). 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 17 Sep 2021.