DEB - Dystrophic Epidermolysis Bullosa, Dominant Dystrophic Epidermolysis Bullosa, Dystrophic Epidermolysis Bullosa, Recessive Dystrophic Epidermolysis Bullosa
Conditions
Keywords
Dystrophic Epidermolysis Bullosa, DEB, Corneal Abrasions
Brief 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).
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.
Interventions
BIOLOGICALKB803
Ophthalmic suspension of replication-defective, non-integrating herpes simplex virus (HSV-1) expressing the human collagen VII protein
DRUGPlacebo
Vehicle
Sponsors
Krystal Biotech, Inc.
Study design
Allocation
RANDOMIZED
Intervention model
CROSSOVER
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
6 Months to No maximum
Healthy volunteers
No
Inclusion criteria
1. 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
1. 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 assessments, interfere with the administration of study drug, and/or poses an additional risk to the subject. 4. Women who are pregnant or nursing. 5. Subject who is unwilling to comply with contraception requirements per protocol. 6. Subject is known to be noncompliant or is unlikely to comply with the requirements of the study protocol in the opinion of the Investigator.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| To evaluate the safety and tolerability of KB803. | 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. | 24 weeks | Average number of days per month with corneal abrasion symptoms. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| To evaluate the effect of ophthalmic KB803 on eye pain. | 24 weeks | Average weekly eye pain score. Pain will be measured using a numerical scale with 0 being no hurt through 10 being the worst hurt. |
Countries
United States
Contacts
CONTACTDavid Sweet, MD, PhD
CONTACTBrittani Agostini, RN, CCRC
Outcome results
None listed