KB803 eye drops versus placebo for people with dystrophic epidermolysis bullosa

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This is a Phase 3, double-blind, randomized, placebo-controlled crossover trial enrolling pediatric and adult participants with DEB and recurrent corneal abrasions. Eligible participants already in the sponsor's natural history study for at least 12 weeks will be randomized 1:1 to receive ophthalmic KB803 or matched placebo three times weekly for 12 weeks, then crossed over to the alternate treatment for an additional 12 weeks. Dosing is administered at home by a trained designee, with investigators monitoring safety via contacts for adverse events and changes in treatments. Participants complete weekly symptom diaries and monthly questionnaires to record corneal abrasion frequency, symptoms, and overall impressions of disease change.

Why it matters

Who can join: the full rules

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.

Where it's running

• Mission Dermatology Center
  Rancho Santa Margarita, California, United States Recruiting
• Skin Research Institute
  Coral Gables, Florida, United States Recruiting
• UMass Memorial Medical Center
  Worcester, Massachusetts, United States Recruiting
• Cincinnati Children's Hospital Medical Center
  Cincinnati, Ohio, United States Recruiting
• Texas Dermatology and Laser Specialists
  San Antonio, Texas, United States Recruiting

Study contacts

• Study coordinator: David Sweet, MD, PhD
• Email: dsweet@krystalbio.com
• Phone: 412-586-5830

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

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• Energy-F703EB Cream for Epidermolysis Bullosa: a skin cream test in adults

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• Long-Term Safety Follow-Up After pz-cel for RDEB

  This study follows people with RDEB who were treated with pz-cel to see how safe it is over up to 15 years and whether their wounds stay healed.

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• Ev.FV to heal wounds in dystrophic epidermolysis bullosa

  This trial will test whether Ev.FV, a mesenchymal stem cell-derived extracellular vesicle therapy, helps heal wounds in people with dystrophic epidermolysis bullosa.

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• Gene-modified autologous skin graft (GMEB-SASS) for RDEB

  This trial will try a gene-modified skin graft grown from a patient's own cells to help wounds heal in people with recessive dystrophic epidermolysis bullosa (RDEB).

  Recruiting Phase 1/2 CHU de Quebec-Universite Laval · Toronto, Ontario · NCT07193134

How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.