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

Phase 1

Recruiting

• Conditions: Junctional Epidermolysis Bullosa; Epidermolysis Bullosa (EB); Dystrophic Epidermolysis Bullosa; Recessive Dystrophic Epidermolysis Bullosa
• Interventions: Biological: ELK-003 (Standarized Amniotic Fluid Secretome Eye Drops)

• Registration Number: NCT06713434
• Lead Sponsor: Fundación DEBRA Chile, Niños Piel de Cristal

Brief 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.

Study Timeline

• Study Start: 2024-05-07
• Status Verified: 2024-11
• Study First Submitted: 2024-11-12
• Study First Submitted QC: 2024-11-27
• Last Update Submitted: 2024-11-27
• Study First Posted: 2024-12-03
• Last Update Posted: 2024-12-03
• Primary Completion: 2025-12
• Study Completion: 2025-12

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 20

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).

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Experimental (Only Arm)	ELK-003 (Standarized Amniotic Fluid Secretome Eye Drops)	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.

Primary Outcome Measures

Name	Time	Method
Safety of ELK-003 Eye Drops	180 days (Treatment Phase)	The primary outcome is to evaluate the safety and tolerability of ELK-003 eye drops in subjects with EB. Safety assessments will focus on the incidence and severity of drug-related adverse events, including any signs of ocular irritation, monitored throughout the study.

Secondary Outcome Measures

Name	Time	Method
Frequency of Corneal Abrasions (Number of Events)	180 days (Treatment Phase)	The secondary outcome is to assess the efficacy of ELK-003 in improving ocular signs and symptoms associated with EB, measuring frequency of corneal abrasions (number of events) over the treatment period.
Days with Corneal Breakdown	180 days (Treatment Phase)	The secondary outcome is to assess the efficacy of ELK-003 in improving ocular signs and symptoms associated with EB, measuring days with corneal breakdown over the treatment period.

Trial Locations

Locations (1)

Fundación DEBRA Chile, Niños Piel de Cristal; 🇨🇱 Santiago, Huechuraba, Chile