Junctional Epidermolysis BullosaPhase 1GOTAS-ELK-EBNCT06713434
Pilot Study of ELK-003 Eye Drops for Treating Ocular Manifestations of Epidermolysis Bullosa
A Phase 1 study testing ELK-003 (Standarized Amniotic Fluid Secretome Eye Drops) in people with Junctional Epidermolysis Bullosa and Epidermolysis Bullosa (EB), run by Fundación DEBRA Chile, Niños Piel de Cristal at 1 site in Chile.
Trial 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. Read more about this study
- Who is it for?
- Ages 2 years and older
- Conditions the study is for
- Junctional Epidermolysis Bullosa, Epidermolysis Bullosa (EB), Dystrophic Epidermolysis Bullosa
- What is being tested?
- ELK-003 (Standarized Amniotic Fluid Secretome Eye Drops)
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 20 participants
- Where
- 1 siteChile
- Study dates
- 7 May 2024to Jun 2026
- Phase
- Phase 1
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 2 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
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- 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 locations and contacts
1 site in 1 country.
Chile1 site
Fundación DEBRA Chile, Niños Piel de Cristal
Santiago, Huechuraba, 8580670
About this study
Official title: 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.
Read the 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.
- Conditions
- Junctional Epidermolysis Bullosa, Epidermolysis Bullosa (EB), Dystrophic Epidermolysis Bullosa, Recessive Dystrophic Epidermolysis Bullosa
- Being tested
- ELK-003 (Standarized Amniotic Fluid Secretome Eye Drops)
- Keywords
- ocular manifestations of EB, corneal abrasion, ELK-003, Debra Chile, Eliksa Therapeutics, Epidermolysis Bullosa
- Medical subject headings
- Epidermolysis Bullosa, Junctional, Epidermolysis Bullosa, Epidermolysis Bullosa Dystrophica, Corneal Injuries
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ELK-003 (Standarized Amniotic Fluid Secretome Eye Drops)Biological
Also called ELK-003, Gotas ELK
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.
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: Biological: ELK-003 (Standarized Amniotic Fluid Secretome Eye Drops) |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 180 days (Treatment Phase)
Secondary outcomes (2)
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.
Measured: 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.
Measured: 180 days (Treatment Phase)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Dec 2024
- Study start7 May 2024
- Primary completionJun 2026 (estimated)
- Study completionJun 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Junctional Epidermolysis BullosaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 20 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Fundación DEBRA Chile, Niños Piel de Cristal
- Sponsor type
- University, hospital or other
- Collaborators
- Eliksa Therapeutics, Inc., Centro de la Vision
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06713434
- Sponsor's protocol ID
- 2022-03
- Other IDs
- 29792/23
- Acronym
- GOTAS-ELK-EB
- Status last verified
- Dec 2025
- First submitted
- 12 Nov 2024
- Last updated
- 31 Dec 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No: 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.
Study at a glance
From the registry record, last updated by the sponsor on 31 Dec 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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.
- ClinicalTrials.gov ID
- NCT06713434
- Acronym / sponsor ID
- GOTAS-ELK-EB / 2022-03
- Conditions
- Junctional Epidermolysis Bullosa, Epidermolysis Bullosa (EB), Dystrophic Epidermolysis Bullosa, Recessive Dystrophic Epidermolysis Bullosa
- Keywords
- ocular manifestations of EB, corneal abrasion, ELK-003, Debra Chile, Eliksa Therapeutics, Epidermolysis Bullosa
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 20 participants (estimated)
- Lead sponsor
- Fundación DEBRA Chile, Niños Piel de CristalUniversity, hospital or other
- Collaborators
- Eliksa Therapeutics, Inc., Centro de la Vision
- Study start
- 7 May 2024 (actual)
- Primary completion
- Jun 2026 (estimated)
- Study completion
- Jun 2026 (estimated)
- First posted
- 3 Dec 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
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 design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Experimental (Only Arm) | Experimental | Biological: ELK-003 (Standarized Amniotic Fluid Secretome Eye Drops)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. |
Who may qualify
- Ages
- 2 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (4 listed)
- 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
Key exclusion criteria (9 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| ELK-003 (Standarized Amniotic Fluid Secretome Eye Drops)Also: ELK-003, Gotas ELKELK-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. | Biological | Experimental (Only Arm) |
Primary outcome
Safety of ELK-003 Eye Drops
Time frame: 180 days (Treatment Phase)
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.
2 secondary outcomes
Frequency of Corneal Abrasions (Number of Events)
Time frame: 180 days (Treatment Phase)
Days with Corneal Breakdown
Time frame: 180 days (Treatment Phase)
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 31 Dec 2025.
- Individual participant dataSharing planned: No. 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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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing ELK-003 (Standarized Amniotic Fluid Secretome Eye Drops) in people with Junctional Epidermolysis Bullosa and Epidermolysis Bullosa (EB), run by Fundación DEBRA Chile, Niños Piel de Cristal at 1 site in Chile.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 31 Dec 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Chile. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Safety of ELK-003 Eye Drops (time frame: 180 days (Treatment Phase)). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 31 Dec 2025.