Dystrophic Epidermolysis BullosaPhase 3IOLITENCT07016750
A Study Comparing KB803 and Matched Placebo in Patients With Dystrophic Epidermolysis Bullosa
A Phase 3 study testing KB803 in people with Dystrophic Epidermolysis Bullosa and DEB - Dystrophic Epidermolysis Bullosa, run by Krystal Biotech, Inc. at 5 sites in United States.
Trial summary
KB803-EYE-01 is a Phase 3 double-blind, randomized, placebo-controlled, crossover study to evaluate the safety and efficacy of KB803 versus matched placebo in pediatric and adult subjects with recurrent corneal abrasions due to dystrophic epidermolysis bullosa (DEB). 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, Recessive Dystrophic Epidermolysis Bullosa
- What is being tested?
- KB803
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 16 participants
- Where
- 5 sitesUnited States
- Study dates
- 20 Jun 2025to Dec 2026
- Phase
- Phase 3
- 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
Who may be able to join
- The subject and/or their parent/legal guardian must provide informed consent/assent and must be able to and willing to follow study procedures and instructions.
- Age 6 months or older at time of informed consent/assent.
- Confirmed diagnosis of DEB with a mutation in the COL7A1 gene.
- Meets minimum corneal abrasion symptom frequency in the NHS study.
Who cannot join
- Initiation of any new treatment regimen or change in treatment for ocular disease during the run-in period except for preservative free topical antibiotics or artificial tears/lubricants associated with standard of care treatment of corneal abrasions.
- Treatment with an investigational agent or off-label ophthalmic use of an approved product during the run-in period or planned use during the study (Exceptions may be approved by the medical monitor on a case-by-case basis).
- Any condition that, in the opinion of the Investigator, would impact the completion of all study-related assessments, interfere with the administration of study drug, and/or poses an additional risk to the subject.
- Women who are pregnant or nursing.
- Subject who is unwilling to comply with contraception requirements per protocol.
- Subject is known to be noncompliant or is unlikely to comply with the requirements of the study protocol in the opinion of the Investigator.
Trial locations and contacts
5 sites in 1 country. Contact the study team or a site near you to ask about taking part.
5 sites in 1 country
United States5 sites, 5 recruiting
- Recruiting
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229
Bret Augsburger, CCRP, 513-636-2009, Bret.Augsburger@cchmc.org
- Recruiting
Skin Research Institute
Coral Gables, Florida, 33146
Alex Cazzaniga, 305-444-5770, info@srinsitute.org
- Recruiting
Mission Dermatology Center
Rancho Santa Margarita, California, 92688
Shireen Guide, MD, 949-858-3376, admin@missiondermatology.com
sguide@gmail.com
- Recruiting
Texas Dermatology and Laser Specialists
San Antonio, Texas, 78218
Jennifer Patterson, 210-852-2779, jpatterson@texasdls.com
- Recruiting
UMass Memorial Medical Center
Worcester, Massachusetts, 01605
Priscilla Romano, MD, 508-856-1706, priscilla.romano@umassmed.edu
Isaac (Li-Chi) Chen, MD, 774-455-4758, lichi.chen6@umassmed.edu
About this study
Official title: A Double-Blind Crossover of KB803 and Matched Placebo for the Treatment and Prevention of Corneal Abrasions in Dystrophic Epidermolysis Bullosa
KB803-EYE-01 is a Phase 3 double-blind, randomized, placebo-controlled, crossover study to evaluate the safety and efficacy of KB803 versus matched placebo in pediatric and adult subjects with recurrent corneal abrasions due to dystrophic epidermolysis bullosa (DEB).
Read the detailed description
Subjects participating in the Sponsor's natural history study (protocol NHS) for at least 12 weeks and that meet all other eligibility criteria will be randomized 1:1 to receive ophthalmic administration of B-VEC three times a week or placebo three times a week for the first 12 week intervention period. During the second 12-week intervention period subjects will be crossed-over to receive the alternative Investigational Product (IP) for an additional 12 weeks. IP will be administered in the home setting by a trained designee. The Investigator (or designee) will contact subjects (or their parent/legal guardian) to assess for any adverse events or changes in medications, treatments, or procedures. Subjects (or their parent/legal guardian) will complete weekly symptom diaries and monthly questionnaires to document corneal abrasion symptoms and frequency and gauge overall subject (or parent/legal guardian) impressions of disease severity and disease/symptom changes during the study.
- Conditions
- Dystrophic Epidermolysis Bullosa, DEB - Dystrophic Epidermolysis Bullosa, Recessive Dystrophic Epidermolysis Bullosa, Dominant Dystrophic Epidermolysis Bullosa
- Being tested
- KB803, Placebo
- Keywords
- Dystrophic Epidermolysis Bullosa, DEB, Corneal Abrasions
- Medical subject headings
- Epidermolysis Bullosa Dystrophica, Corneal Injuries
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
KB803Biological
Ophthalmic suspension of replication-defective, non-integrating herpes simplex virus (HSV-1) expressing the human collagen VII protein
PlaceboDrug
Vehicle
| Study group | Role | What they receive |
|---|---|---|
| KB803 | Experimental | KB803 Receives: Biological: KB803 |
| Placebo | Placebo comparator | Vehicle Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
To evaluate the safety and tolerability of KB803.
Number of subjects with treatment related adverse events as assessed by CTCAE v5.
Measured: 24 weeks
To evaluate the effect of ophthalmic KB803 on frequency of corneal abrasion symptoms.
Average number of days per month with corneal abrasion symptoms.
Measured: 24 weeks
Secondary outcomes (1)
To evaluate the effect of ophthalmic KB803 on eye pain.
Average weekly eye pain score.
Pain will be measured using a numerical scale with 0 being no hurt through 10 being the worst hurt.
Measured: 24 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Jun 2025
- Study start20 Jun 2025
- Primary completionDec 2026 (estimated)
- Study completionDec 2026 (estimated)
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
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 16 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
- NCT07016750
- Sponsor's protocol ID
- KB803-EYE-01
- Acronym
- IOLITE
- Status last verified
- Mar 2026
- First submitted
- 27 May 2025
- Last updated
- 12 Mar 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 12 Mar 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Double-Blind Crossover of KB803 and Matched Placebo for the Treatment and Prevention of Corneal Abrasions in Dystrophic Epidermolysis Bullosa
- ClinicalTrials.gov ID
- NCT07016750
- Acronym / sponsor ID
- IOLITE / KB803-EYE-01
- Conditions
- Dystrophic Epidermolysis Bullosa, DEB - Dystrophic Epidermolysis Bullosa, Recessive Dystrophic Epidermolysis Bullosa, Dominant Dystrophic Epidermolysis Bullosa
- Keywords
- Dystrophic Epidermolysis Bullosa, DEB, Corneal Abrasions
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 16 participants (estimated)
- Lead sponsor
- Krystal Biotech, Inc.Industry (pharma, biotech, devices)
- Study start
- 20 Jun 2025 (actual)
- Primary completion
- Dec 2026 (estimated)
- Study completion
- Dec 2026 (estimated)
- First posted
- 12 Jun 2025 (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.
KB803-EYE-01 is a Phase 3 double-blind, randomized, placebo-controlled, crossover study to evaluate the safety and efficacy of KB803 versus matched placebo in pediatric and adult subjects with recurrent corneal abrasions due to dystrophic epidermolysis bullosa (DEB).
Detailed description
Subjects participating in the Sponsor's natural history study (protocol NHS) for at least 12 weeks and that meet all other eligibility criteria will be randomized 1:1 to receive ophthalmic administration of B-VEC three times a week or placebo three times a week for the first 12 week intervention period. During the second 12-week intervention period subjects will be crossed-over to receive the alternative Investigational Product (IP) for an additional 12 weeks. IP will be administered in the home setting by a trained designee. The Investigator (or designee) will contact subjects (or their parent/legal guardian) to assess for any adverse events or changes in medications, treatments, or procedures. Subjects (or their parent/legal guardian) will complete weekly symptom diaries and monthly questionnaires to document corneal abrasion symptoms and frequency and gauge overall subject (or parent/legal guardian) impressions of disease severity and disease/symptom changes during the study.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| KB803 | Experimental | Biological: KB803KB803 |
| Placebo | Placebo comparator | Drug: PlaceboVehicle |
Who may qualify
- Ages
- 6 months and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (4 listed)
- The subject and/or their parent/legal guardian must provide informed consent/assent and must be able to and willing to follow study procedures and instructions.
- Age 6 months or older at time of informed consent/assent.
- Confirmed diagnosis of DEB with a mutation in the COL7A1 gene.
- Meets minimum corneal abrasion symptom frequency in the NHS study.
Key exclusion criteria (6 listed)
- Initiation of any new treatment regimen or change in treatment for ocular disease during the run-in period except for preservative free topical antibiotics or artificial tears/lubricants associated with standard of care treatment of corneal abrasions.
- Treatment with an investigational agent or off-label ophthalmic use of an approved product during the run-in period or planned use during the study (Exceptions may be approved by the medical monitor on a case-by-case basis).
- Any condition that, in the opinion of the Investigator, would impact the completion of all study-related assessments, interfere with the administration of study drug, and/or poses an additional risk to the subject.
- Women who are pregnant or nursing.
- Subject who is unwilling to comply with contraception requirements per protocol.
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 |
|---|---|---|
| KB803Ophthalmic suspension of replication-defective, non-integrating herpes simplex virus (HSV-1) expressing the human collagen VII protein | Biological | KB803 |
| PlaceboVehicle | Drug | Placebo |
Primary outcome
To evaluate the safety and tolerability of KB803.
Time frame: 24 weeks
Number of subjects with treatment related adverse events as assessed by CTCAE v5.
To evaluate the effect of ophthalmic KB803 on frequency of corneal abrasion symptoms.
Time frame: 24 weeks
Average number of days per month with corneal abrasion symptoms.
1 secondary outcome
To evaluate the effect of ophthalmic KB803 on eye pain.
Time frame: 24 weeks
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 12 Mar 2026.
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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.
5 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing KB803 in people with Dystrophic Epidermolysis Bullosa and DEB - Dystrophic Epidermolysis Bullosa, run by Krystal Biotech, Inc. at 5 sites 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 4 inclusion and 6 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 12 Mar 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 5 sites 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. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
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 Sweet, MD, PhD (412-586-5830, dsweet@krystalbio.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
To evaluate the safety and tolerability of KB803. (time frame: 24 weeks). The study lists 2 primary outcomes in all. 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 12 Mar 2026.