Pilot Study of ELK-003 Eye Drops for Treating Ocular Manifestations of Epidermolysis Bullosa (NCT06713434) | Clinical Trial Compass
Active — Not RecruitingPhase 1
Pilot Study of ELK-003 Eye Drops for Treating Ocular Manifestations of Epidermolysis Bullosa
Chile20 participantsStarted 2024-05-07
Plain-language summary
This study consists of two phases: an Observational Phase to evaluate the natural history of ocular manifestations in subjects with Dystrophic and Junctional Epidermolysis Bullosa, followed by a Treatment Phase to assess the effects of ELK-003 eye drops. Each subject will serve as their own control by comparing ocular manifestations documented during the Observational Phase to those recorded during the Treatment Phase.
Who can participate
Age range
2 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
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).
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Safety of ELK-003 Eye Drops
Timeframe: 180 days (Treatment Phase)
Trial details
NCT IDNCT06713434
SponsorFundación DEBRA Chile, Niños Piel de Cristal