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

NCT06713434 | Active Not Recruiting Phase 1 FDA Drug

• 2 other identifiers: 2022-0329792/23
• Study Type: interventional
• Target: 20
• Locations: 1 country
• Sites: 1

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.

Conditions

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

Keywords

ocular manifestations of EB; corneal abrasion; ELK-003; Debra Chile; Eliksa Therapeutics; Epidermolysis Bullosa

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (2)

• Frequency of Corneal Abrasions (Number of Events)

  180 days (Treatment Phase)

• Days with Corneal Breakdown

  180 days (Treatment Phase)

Study Arms (1)

Experimental (Only Arm)

EXPERIMENTAL

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)

Interventions

ELK-003 (Standarized Amniotic Fluid Secretome Eye Drops) BIOLOGICAL

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.

Also known as: ELK-003, Gotas ELK

Experimental (Only Arm)

Eligibility Criteria

• Age: 2 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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).

Sponsors & Collaborators

• Fundación DEBRA Chile, Niños Piel de Cristal: lead
• Eliksa Therapeutics, Inc.: collaborator
• Centro de la Vision: collaborator

Study Sites (1)

Fundación DEBRA Chile, Niños Piel de Cristal

Santiago, Huechuraba, 8580670, Chile

Location

MeSH Terms

Conditions

Epidermolysis Bullosa, Junctional; Epidermolysis Bullosa; Epidermolysis Bullosa Dystrophica; Corneal Injuries

Condition Hierarchy (Ancestors)

Skin Abnormalities; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Skin Diseases, Genetic; Genetic Diseases, Inborn; Skin Diseases; Skin and Connective Tissue Diseases; Skin Diseases, Vesiculobullous; Collagen Diseases; Connective Tissue Diseases; Eye Injuries; Facial Injuries; Craniocerebral Trauma; Trauma, Nervous System; Nervous System Diseases; Corneal Diseases; Eye Diseases; Wounds and Injuries

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 12, 2024
• First Posted: December 3, 2024
• Study Start: May 7, 2024
• Primary Completion (Estimated): June 1, 2026
• Study Completion (Estimated): June 1, 2026
• Last Updated: December 31, 2025

Record last verified: 2025-12

Data Sharing

• IPD Sharing: Will not share

Locations

CL (1)