Epidermolysis Bullosa DystrophicaNCT06563414
A Natural History Study of Corneal Abrasions in Patients With Dystrophic Epidermolysis Bullosa (DEB)
An observational study of Epidermolysis Bullosa Dystrophica and Epidermolysis Bullosa Dystrophica, Recessive, 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 corneal abrasions in patients with Dystrophic Epidermolysis Bullosa (DEB). Read more about this study
- Who is it for?
- Ages 6 months and older
- Conditions the study is for
- Epidermolysis Bullosa Dystrophica, Epidermolysis Bullosa Dystrophica, Recessive, Epidermolysis Bullosa Dystrophica Dominans
- How many people
- 40 participants
- Where
- 1 siteUnited States
- Study dates
- 2 Aug 2024to Dec 2025
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
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 with Dystrophic Epidermolysis Bullosa, aged 6 months or older.
Who may be able to join
- The subject or their respective legal guardian must have read, understood, and signed an Institutional Review Board (IRB) approved Informed Consent/Assent Form and must be able to and willing to follow study procedures and instructions.
- Age 6 months or older at time of Informed Consent/Assent.
- Prior genetic diagnosis of DEB due to mutation(s) in the collagen type VII alpha 1 chain (COL7A1) gene.
Who cannot join
None listed.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
Krystal Biotech
Pittsburgh, Pennsylvania, 15203
Brittani Agostini, 412-586-5830, debeyestudy@krystalbio.com
About this study
This study is a non-interventional, observational study that will evaluate the natural history of corneal abrasions in patients with Dystrophic Epidermolysis Bullosa (DEB).
Read the detailed description
The objective of this study is to characterize the natural history of corneal abrasions in patients with Dystrophic Epidermolysis Bullosa (DEB). Patients will be followed via remote weekly symptom diaries and monthly questionnaires to assess corneal abrasion symptoms, frequency, and duration.
- Conditions
- Epidermolysis Bullosa Dystrophica, Epidermolysis Bullosa Dystrophica, Recessive, Epidermolysis Bullosa Dystrophica Dominans
- Keywords
- Dystrophic Epidermolysis Bullosa, DEB, Corneal Abrasions
- Medical subject headings
- Epidermolysis Bullosa Dystrophica, Corneal Injuries
What is being measured
The primary outcome is the main question the study is designed to answer.
Corneal abrasion frequency
Frequency of corneal abrasion symptoms
Measured: Baseline through up to 1 year
Secondary outcomes (1)
Epidermolysis Bullosa Eye Disease Index Symptom Score
Symptom score for Epidermolysis Bullosa related eye problems, 0 to 30 (higher score indicates worse symptoms)
Measured: Baseline through up to 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Aug 2024
- Study start2 Aug 2024
- Primary completionDec 2025 (estimated)
- Study completionDec 2025 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Epidermolysis Bullosa DystrophicaStudies 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
- 40 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Krystal Biotech, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06563414
- Sponsor's protocol ID
- NHS
- Status last verified
- Jul 2025
- First submitted
- 19 Aug 2024
- Last updated
- 2 Jul 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 2 Jul 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Natural History Study of Corneal Abrasions in Patients With Dystrophic Epidermolysis Bullosa (DEB)
- ClinicalTrials.gov ID
- NCT06563414
- Acronym / sponsor ID
- NHS
- Conditions
- Epidermolysis Bullosa Dystrophica, Epidermolysis Bullosa Dystrophica, Recessive, Epidermolysis Bullosa Dystrophica Dominans
- Keywords
- Dystrophic Epidermolysis Bullosa, DEB, Corneal Abrasions
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 40 participants (estimated)
- Lead sponsor
- Krystal Biotech, Inc.Industry (pharma, biotech, devices)
- Study start
- 2 Aug 2024 (actual)
- Primary completion
- Dec 2025 (estimated)
- Study completion
- Dec 2025 (estimated)
- First posted
- 20 Aug 2024 (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 corneal abrasions in patients with Dystrophic Epidermolysis Bullosa (DEB).
Detailed description
The objective of this study is to characterize the natural history of corneal abrasions in patients with Dystrophic Epidermolysis Bullosa (DEB). Patients will be followed via remote weekly symptom diaries and monthly questionnaires to assess corneal abrasion symptoms, frequency, and duration.
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 with Dystrophic Epidermolysis Bullosa, aged 6 months or older.
Key inclusion criteria (3 listed)
- The subject or their respective legal guardian must have read, understood, and signed an Institutional Review Board (IRB) approved Informed Consent/Assent Form and must be able to and willing to follow study procedures and instructions.
- Age 6 months or older at time of Informed Consent/Assent.
- Prior genetic diagnosis of DEB due to mutation(s) in the collagen type VII alpha 1 chain (COL7A1) gene.
Key exclusion criteria (0 listed)
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
Corneal abrasion frequency
Time frame: Baseline through up to 1 year
Frequency of corneal abrasion symptoms
1 secondary outcome
Epidermolysis Bullosa Eye Disease Index Symptom Score
Time frame: Baseline through up to 1 year
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 2 Jul 2025.
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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.
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 Dystrophica and Epidermolysis Bullosa Dystrophica, Recessive, 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 3 inclusion and 0 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: looking for participants now, as of the sponsor's last update on 2 Jul 2025. 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?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact David Chien, MD (412-586-5830, dchien@krystalbio.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Corneal abrasion frequency (time frame: Baseline through up to 1 year). 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 2 Jul 2025.