Observing corneal abrasions in patients with Dystrophic Epidermolysis Bullosa
Official title: A Natural History Study of Corneal Abrasions in Patients With Dystrophic Epidermolysis Bullosa (DEB)
Run by Krystal Biotech, Inc. · NCT06563414 · Data updated 2026-11-12
This study looks at how corneal abrasions develop over time in people with Dystrophic Epidermolysis Bullosa by tracking their symptoms through weekly diaries and monthly questionnaires.
Quick facts
| Study type | Observational |
|---|---|
| Ages | 6 Months and up |
| Sex | All |
| Enrollment | 40 (estimated) |
| Sponsor | Krystal Biotech, Inc. Industry-sponsored |
| Locations | 1 site (Pittsburgh, Pennsylvania) |
| Status | Recruiting |
| Started | August 2, 2024 |
| Main results expected | December 2025 |
| Study ends | December 2025 |
| Registry record updated | July 2, 2025 |
| Trial ID | NCT06563414 on ClinicalTrials.gov |
Is this trial a fit for you?
✓Likely a good fit
Ideal candidates for this study are individuals aged 6 months or older with a genetic diagnosis of Dystrophic Epidermolysis Bullosa due to mutations in the COL7A1 gene.
✕Probably not a fit
Patients without a confirmed genetic diagnosis of DEB or those under 6 months of age may not benefit from this study.
A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.
What this trial studies
This observational study aims to evaluate the natural history of corneal abrasions in patients diagnosed with Dystrophic Epidermolysis Bullosa (DEB). Participants will be monitored through remote weekly symptom diaries and monthly questionnaires to gather data on the symptoms, frequency, and duration of corneal abrasions. The study does not involve any interventions but focuses on understanding the condition's progression over time.
Why it matters
Potential benefit: If successful, this study could provide valuable insights into the management and treatment of corneal abrasions in DEB patients.
How similar studies have gone: While this study focuses on a specific aspect of DEB, similar observational studies have provided valuable data in understanding the natural history of rare conditions.
Who can join: the full rules
Show full inclusion / exclusion criteria
Inclusion Criteria: 1. 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. 2. Age 6 months or older at time of Informed Consent/Assent. 3. Prior genetic diagnosis of DEB due to mutation(s) in the collagen type VII alpha 1 chain (COL7A1) gene.
Where it's running
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Krystal Biotech
Pittsburgh, Pennsylvania, United States Recruiting
Study contacts
- Study coordinator: David Chien, MD
- Email: dchien@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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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).
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Understanding characteristics of patients with recessive dystrophic epidermolysis bullosa
This study looks at people with recessive dystrophic epidermolysis bullosa to gather information that could help find new treatments for their skin condition.
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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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KB803 eye drops versus placebo for people with dystrophic epidermolysis bullosa
This trial tests whether KB803 eye drops, given three times a week, can reduce recurrent corneal abrasions in children and adults with dystrophic epidermolysis bullosa.
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Using IV immunoglobulin to lower collagen VII antibodies in dystrophic epidermolysis bullosa
This trial tests whether IV immunoglobulin given to people with recessive dystrophic epidermolysis bullosa who are on VYJUVEK helps wounds heal faster and lowers collagen VII and HSV‑1 antibody levels.
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Long-term follow-up for gene therapy in skin conditions
This study is checking the long-term safety of a gene therapy for people with a skin condition called dystrophic epidermolysis bullosa who have already received the treatment.
How to take part
- Review the eligibility criteria above with your treating physician.
- Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
- Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.