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| ID | Type | Description | Link |
|---|---|---|---|
| 29792/23 | Other Identifier | ISP |
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| Name | Class |
|---|---|
| Eliksa Therapeutics, Inc. | UNKNOWN |
| Centro de la Vision | UNKNOWN |
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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.
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| 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. |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| 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. |
| 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) |
| 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 |
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Inclusion Criteria:
Exclusion Criteria:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Fundación DEBRA Chile, Niños Piel de Cristal | Santiago | Huechuraba | 8580670 | Chile |
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.
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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) |
| ID | Term |
|---|---|
| D016109 | Epidermolysis Bullosa, Junctional |
| D004820 | Epidermolysis Bullosa |
| D016108 | Epidermolysis Bullosa Dystrophica |
| D065306 | Corneal Injuries |
| ID | Term |
|---|---|
| D012868 | Skin Abnormalities |
| D000013 | Congenital Abnormalities |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D012873 | Skin Diseases, Genetic |
| D030342 | Genetic Diseases, Inborn |
| D012871 | Skin Diseases |
| D017437 | Skin and Connective Tissue Diseases |
| D012872 | Skin Diseases, Vesiculobullous |
| D003095 | Collagen Diseases |
| D003240 | Connective Tissue Diseases |
| D005131 | Eye Injuries |
| D005151 | Facial Injuries |
| D006259 | Craniocerebral Trauma |
| D020196 | Trauma, Nervous System |
| D009422 | Nervous System Diseases |
| D003316 | Corneal Diseases |
| D005128 | Eye Diseases |
| D014947 | Wounds and Injuries |
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