Pilot Study of ELK-003 Eye Drops for Treating Ocular Manifestations of Epidermolysis Bullosa (GOTAS-ELK-EB)

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.

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.

Study Overview

• Status: Active, not recruiting
• Conditions: Dystrophic Epidermolysis Bullosa; Recessive Dystrophic Epidermolysis Bullosa; Junctional Epidermolysis Bullosa; Epidermolysis Bullosa (EB)
• Intervention / Treatment: Biological: ELK-003 (Standarized Amniotic Fluid Secretome Eye Drops)

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 Type: Interventional
• Enrollment (Estimated): 20
• Phase: Phase 1

Contacts and Locations

Study Locations

• Chile
  • Huechuraba
    • Santiago, Huechuraba, Chile, 8580670
      • Fundación DEBRA Chile, Niños Piel de Cristal

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

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 Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm

Intervention / Treatment

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.

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.; Other Names: ELK-003; Gotas ELK

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Safety of ELK-003 Eye Drops	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.	180 days (Treatment Phase)

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Frequency of Corneal Abrasions (Number of Events)	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.	180 days (Treatment Phase)
Days with Corneal Breakdown	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.	180 days (Treatment Phase)

Collaborators and Investigators

• Sponsor: Fundación DEBRA Chile, Niños Piel de Cristal
• Collaborators: Eliksa Therapeutics, Inc.; Centro de la Vision

Study record dates

Study Major Dates

• Study Start (Actual): May 7, 2024
• Primary Completion (Estimated): June 1, 2026
• Study Completion (Estimated): June 1, 2026

Study Registration Dates

• First Submitted: November 12, 2024
• First Submitted That Met QC Criteria: November 27, 2024
• First Posted (Actual): December 3, 2024

Study Record Updates

• Last Update Posted (Actual): December 31, 2025
• Last Update Submitted That Met QC Criteria: December 25, 2025
• Last Verified: December 1, 2025

More Information

Terms related to this study

• Keywords: Epidermolysis Bullosa; corneal abrasion; ocular manifestations of EB; ELK-003; Debra Chile; Eliksa Therapeutics
• Additional Relevant MeSH Terms: Nervous System Diseases; Wounds and Injuries; Genetic Diseases, Inborn; Connective Tissue Diseases; Eye Diseases; Skin Diseases; Corneal Diseases; Congenital Abnormalities; Craniocerebral Trauma; Trauma, Nervous System; Skin Diseases, Genetic; Skin Abnormalities; Skin Diseases, Vesiculobullous; Collagen Diseases; Eye Injuries; Facial Injuries; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Skin and Connective Tissue Diseases; Epidermolysis Bullosa; Epidermolysis Bullosa Dystrophica; Epidermolysis Bullosa, Junctional; Corneal Injuries; Pharmaceutical Solutions; Ophthalmic Solutions
• Other Study ID Numbers: 2022-03; 29792/23 (Other Identifier: ISP)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO
• IPD Plan Description: We do not plan to share Individual Participant Data with other researchers. However, we plan on publishing the study findings in a peer-reviewed journal.

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: Yes