Eye drops for treating eye issues in patients with Epidermolysis Bullosa
Pilot, Non-Randomized, Open-Label Study to Determine the Effect of Topical ELK (ELK-003) Eye Drops Applied 6 Times Daily on Ocular Signs and Symptoms in Patients with Junctional and Dystrophic Subtypes of Epidermolysis Bullosa. Estudio Piloto, No Randomizado Y Abierto Para Determinar El Efecto De Las Gotas Oftalmológicas Tópicas ELK Aplicadas 6 Veces Al Día En Los Signos Y Síntomas Oculares De Pacientes Con Los Subtipos De La Unión Y Recesivo Distrófico De Epidermólisis Bulosa.
This study is testing if a new eye drop treatment can help improve eye problems in people with Epidermolysis Bullosa.
Quick facts
| Phase | Phase 1 |
|---|---|
| Study type | Interventional |
| Enrollment | 20 (estimated) |
| Ages | 2 Years and up |
| Sex | All |
| Sponsor | Fundación DEBRA Chile, Niños Piel de Cristal Academic / other |
| Locations | 1 site (Santiago, Huechuraba) |
| Trial ID | NCT06713434 on ClinicalTrials.gov |
What this trial studies
This pilot study evaluates the natural history of ocular manifestations in patients with Dystrophic and Junctional Epidermolysis Bullosa through an Observational Phase, followed by a Treatment Phase assessing the effects of ELK-003 eye drops. Participants will undergo three clinic visits over six months, where various assessments including imaging and visual acuity testing will be conducted. Each subject serves as their own control by comparing data from the Observational Phase to the Treatment Phase. Weekly questionnaires will track symptoms and quality of life, while healthcare providers will monitor any corneal abrasions that occur during the study.
Who should consider this trial
Good fit: Ideal candidates include individuals diagnosed with Recessive Dystrophic or Junctional Epidermolysis Bullosa who experience frequent corneal abrasions.
Not a fit: Patients with severe vision loss, acute eye infections, or those unable to attend required visits may not benefit from this study.
Why it matters
Potential benefit: If successful, this treatment could significantly improve the quality of life for patients suffering from ocular manifestations of Epidermolysis Bullosa.
How similar studies have performed: While this approach is novel, similar studies targeting ocular conditions have shown promise, suggesting potential for success.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * Confirmed diagnosis of Recessive Dystrophic and Junctional Epidermolysis bullosa * Frequent history of corneal abrasions (one or more abrasions every 3 months) * Age: older than 2 years at the time of joining the clinical trial * Patients and/or parent/legal guardian has given written informed consent in writing Exclusion Criteria: * Acute eye infection at time of enrollment * Patients with severe vision loss (\<20/200) or severe corneal opacification. * Contact lens wear or nocturnal eye ointment * Hypersensitivity to fluorescein (ocular stain used in slit lamp study) * History of alcohol abuse or drug addiction * Patient who reports difficulty to attend the 4 in-person controls * Patients who are suffering from cancer * Patients with any medical condition or situation which in the opinion of the investigators could put the patient at risk, or could interfere with the patient's participation in the study * Pregnant or breastfeeding women (a pregnancy test will be performed using a urine sample for female patients who have had menarche).
Where this trial is running
Santiago, Huechuraba
- Fundación DEBRA Chile, Niños Piel de Cristal — Santiago, Huechuraba, Chile (Recruiting)
Study contacts
- Study coordinator: Ignacia Fuentes, PhD
- Email: ifuentes@debrachile.cl
- Phone: +56 9 9310 8821
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.