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.
Status: Recruiting
Location: See location...
Intervention Type: Biological
Study Type: Interventional
Study Phase: Phase 1
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.
Eligibility
Participation Requirements
Sex: All
Minimum Age: 2
Healthy Volunteers: f
View:
• 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
Locations
Other Locations
Chile
Fundación DEBRA Chile, Niños Piel de Cristal
RECRUITING
Santiago
Contact Information
Primary
Ignacia Fuentes, PhD
ifuentes@debrachile.cl
+56 9 9310 8821
Time Frame
Start Date: 2024-05-07
Estimated Completion Date: 2025-12
Participants
Target number of participants: 20
Treatments
Experimental: Experimental (Only Arm)
In this study, there are two main phases rather than separate arms. During the Observational Phase, no intervention is administered and subjects' natural history of ocular manifestations is documented. In the Treatment Phase, all subjects receive ELK-003 eye drops to assess their effect on ocular sign and symptons. Each subject serves as their own control by comparing outcomes between the Observational and Treatment Phases.
Related Therapeutic Areas
Sponsors
Leads: Fundación DEBRA Chile, Niños Piel de Cristal
Collaborators: Eliksa Therapeutics, Inc., Centro de la Vision