NCT07016750 · Krystal Biotech, Inc.
A Study Assessing KB803 Compared to Matching Placebo for the Treatment and Prevention of Corneal Abrasions in Dystrophic Epidermolysis Bullosa
(IOLITE)
What this study is about
KB803-EYE-01 is a Phase 3 where neither patients nor doctors know which treatment is given, intra-patient crossover study, to evaluate the safety and effectiveness of KB803 versus matched placebo in pediatric and adult subjects with recurrent corneal abrasions due to dystrophic epidermolysis bullosa (DEB).
View original scientific description
KB803-EYE-01 is a Phase 3 double-blind, intra-patient 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).
Interventions
BIOLOGICAL
KB803
Ophthalmic suspension of replication-defective, non-integrating herpes simplex virus (HSV-1) expressing the human collagen VII protein
DRUG
Placebo
Vehicle
Primary outcome measures
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.
Who can participate
This study lists these criteria on ClinicalTrials.gov. A study coordinator reviews eligibility during screening — this page does not determine whether you qualify.
Inclusion criteria
- 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. 2. Age 6 months or older at time of informed consent/assent. 3. Confirmed diagnosis of DEB with a mutation in the COL7A1 gene. 4. Meets minimum corneal abrasion symptom frequency in the NHS study.
Exclusion criteria
- 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. 2. 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). 3. Any condition that, in the opinion of the Investigator, would impact the completion of all study-related asses
Where
- Rancho Santa Margarita, California
- Coral Gables, Florida
- Worcester, Massachusetts
- Cincinnati, Ohio
- San Antonio, Texas
Related conditions & keywords
Frequently asked questions
What is a clinical trial?
A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.
Is it safe to participate?
Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.
Will I be compensated?
Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.
Will I receive a placebo instead of treatment?
When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.
Can I leave a trial if I change my mind?
Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.
How long does a clinical trial last?
Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.
Data: ClinicalTrials.gov · synced Mar 12, 2026 · Source of record for eligibility and locations