Pilot Study of ELK-003 Eye Drops for Treating Ocular Manifestations of Epidermolysis Bullosa

Launched by FUNDACIÓN DEBRA CHILE, NIÑOS PIEL DE CRISTAL · Nov 27, 2024

Keywords

ClinConnect Summary

This clinical trial is exploring the use of a new eye drop treatment called ELK-003 for people with certain types of Epidermolysis Bullosa (EB), a condition that can cause very fragile skin and blisters. The study has two parts: first, researchers will observe how eye problems related to EB develop over time, and then they will give participants the ELK-003 eye drops to see if they help improve these eye issues. Each participant will help researchers understand the treatment's effects by comparing their eye health during the observation phase to their health while using the eye drops.

To join the trial, participants need to be diagnosed with specific types of Epidermolysis Bullosa and have a history of frequent eye injuries, like corneal abrasions, that happen at least once every three months. The study is open to anyone older than 2 years, and both patients and their parents or guardians must provide written consent to participate. Those interested should know that there are some exclusions, such as having a serious eye infection at the start of the study or severe vision problems. Participants will need to attend four in-person visits, where researchers will keep track of their eye health over time.

Gender

ALL

Eligibility 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).