Observing corneal abrasions in patients with Dystrophic Epidermolysis Bullosa
A Natural History Study of Corneal Abrasions in Patients With Dystrophic Epidermolysis Bullosa (DEB)
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 |
|---|---|
| Enrollment | 40 (estimated) |
| Ages | 6 Months and up |
| Sex | All |
| Sponsor | Krystal Biotech, Inc. Industry-sponsored |
| Locations | 1 site (Pittsburgh, Pennsylvania) |
| Trial ID | NCT06563414 on ClinicalTrials.gov |
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.
Who should consider this trial
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.
Not a fit: Patients without a confirmed genetic diagnosis of DEB or those under 6 months of age may not benefit from this study.
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 performed: 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.
Eligibility criteria
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 this trial is running
Pittsburgh, Pennsylvania
- Krystal Biotech — Pittsburgh, Pennsylvania, United States (Recruiting)
Study contacts
- Study coordinator: David Chien, MD
- Email: dchien@krystalbio.com
- Phone: 412-586-5830
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.