NCT06713434

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

• Status: Active Not Recruiting
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 20 (estimated)
• Sponsor: Fundación DEBRA Chile, Niños Piel de Cristal · Academic / Other
• Sex: All
• Age: 2 Years
• Healthy volunteers: Not accepted

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.

Detailed description

During this study, subjects will undergo evaluations at three scheduled clinic visits: at enrollment (baseline, time 0), at 4 months, and at the conclusion of the Treatment Phase (6 months post-treatment initiation). Assessments will include OCT imaging, slit lamp examination with photographs, keratograph analysis, visual acuity testing, osmolarity testing, InflammaDry, SPK scoring, Schirmer Test, and opacity scoring. Weekly questionnaires will be completed by subjects to assess symptoms, track corneal abrasions, and evaluate quality of life throughout the study. If subjects experience symptoms indicative of corneal abrasions, a healthcare provider will conduct a home visit to document the event, using fluorescein staining to confirm and record the corneal abrasion. Corneal abrasion symptoms, frequency, duration, and healing outcomes will be monitored.

Conditions

• Junctional Epidermolysis Bullosa
• Epidermolysis Bullosa (EB)
• Dystrophic Epidermolysis Bullosa
• Recessive Dystrophic Epidermolysis Bullosa

Interventions

Type	Name	Description
BIOLOGICAL	ELK-003 (Standarized Amniotic Fluid Secretome Eye Drops)	ELK-003 eye drops are a standardized amniotic fluid secretome formulation. It contains essential extracellular matrix proteins, such as collagen type VII and laminin-332, which are critical for maintaining corneal integrity in EB patients. In addition, ELK-003 also includes growth factors that stimulate corneal healing and tissue regeneration. This combination of matrix proteins and growth factors aims to enhance corneal surface stability.

Timeline

• Start date: 2024-05-07
• Primary completion: 2026-06-01
• Completion: 2026-06-01
• First posted: 2024-12-03
• Last updated: 2025-12-31

Locations

1 site across 1 country: Chile

Regulatory

• FDA-regulated drug study