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

Last updated: November 27, 2024
Sponsor: Fundación DEBRA Chile, Niños Piel de Cristal
Overall Status: Active - Recruiting

Phase

1

Condition

Epidermolysis Bullosa

Pemphigus Vulgaris (Pv)

Treatment

ELK-003 (Standarized Amniotic Fluid Secretome Eye Drops)

Clinical Study ID

NCT06713434
2022-03
29792/23
  • Ages > 2
  • All Genders

Study 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.

Eligibility Criteria

Inclusion

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

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 theinvestigators could put the patient at risk, or could interfere with the patient'sparticipation in the study

  • Pregnant or breastfeeding women (a pregnancy test will be performed using a urinesample for female patients who have had menarche).

Study Design

Total Participants: 20
Treatment Group(s): 1
Primary Treatment: ELK-003 (Standarized Amniotic Fluid Secretome Eye Drops)
Phase: 1
Study Start date:
May 07, 2024
Estimated Completion Date:
December 31, 2025

Study 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.

Connect with a study center

  • Fundación DEBRA Chile, Niños Piel de Cristal

    Santiago, Huechuraba 8580670
    Chile

    Active - Recruiting

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