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

Fundación DEBRA Chile, Niños Piel de Cristal

Status and phase: Active, not recruiting; Phase 1

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

Treatments: Biological: ELK-003 (Standarized Amniotic Fluid Secretome Eye Drops)

Study type: Interventional

Funder types: Other

Identifiers

NCT06713434

2022-03

29792/23 (Other Identifier)

Details and patient eligibility

About

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.

Full 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.

Enrollment: 20 estimated patients

Sex: All

Ages: 2+ years old

Volunteers: No Healthy Volunteers

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).

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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20 participants in 1 patient group

Experimental (Only Arm)

Experimental group

Description:

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.

Treatment:

Biological: ELK-003 (Standarized Amniotic Fluid Secretome Eye Drops)