Down Syndrome (DS)Phase 3NCT07135167
Compassionate Use Study of Epi-ON Corneal Collagen Crosslinking Performed Using UVA Exposure on Eyes With Ectatic Corneal Diseases for Subjects With Down Syndrome
A Phase 3 study testing Epi-ON corneal cross-linking (CXL) in people with Down Syndrome (DS) and Keratoconus, run by Woolfson Eye Institute at 12 sites in United States.
Trial summary
The goal of this clinical trial is to study the effects of an experimental (not Food and Drug Administration (FDA)-approved) treatment called corneal crosslinking (CXL) for conditions in which the cornea becomes progressively thin, steep, and misshapen, causing vision to be blurred. Read more about this study
- Who is it for?
- Ages 8 years and older
- Conditions the study is for
- Down Syndrome (DS), Keratoconus, Pellucid Marginal Degeneration
- What is being tested?
- Epi-ON corneal cross-linking (CXL)
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 225 participants
- Where
- 12 sitesUnited States
- Study dates
- 1 Aug 2025to 31 Dec 2028
- Phase
- Phase 3
- 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 8 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Be at least 8 years of age or older, male or female, of any race.
- Have a diagnosis of Down syndrome or other cognitive, developmental, or medical conditions that precludes the subject from reliably following instructions or performing some of the study examination procedures.
- Have a diagnosis of keratoconus, pellucid marginal degeneration, progressive ectasia after previous CXL treatment, other ectatic condition, or forme fruste keratoconus based on topography, tomography, and slit lamp examination.
- Provide written informed consent and a signed HIPAA form. Pediatric subjects less than 14 years of age must sign an assent, and a parent or legal guardian must sign an informed consent. If the subject has impaired consent capacity, determined by the consenter, the consent form must be signed by the subject's legally authorized representative on behalf of the subject.
- Satisfactory completion of the standardized clinical decision tool evaluation of patient suitability for CXL. (See Section 7.2.3)
- Be willing and able to follow all instructions to the best of his/her abilities and comply with the schedule for follow-up visits.
Who cannot join
- Normal corneal topography.
- A history of previous corneal transplant in the study eye.
- Minimum corneal thickness < 300 (measured by Pentacam and Ultrasound) at the screening exam.
- Previous ocular condition (other than refractive error) in the eye(s) to be treated that may predispose the eye to future complications or prevent the possibility of improved vision, for example:
- History of, or active, corneal disease (e.g., herpes simplex, herpes zoster keratitis, etc.)
- Clinically significant corneal scarring in the central cornea that, in the investigator's opinion, will not allow the subject to achieve functional vision, even with contact lens correction, after the crosslinking procedure.
Show all 12Show fewer
- A known contraindication, sensitivity, or allergy to the test article or its components or to study medications.
- Nystagmus or any other condition that would prevent a steady gaze during the crosslinking treatment or other diagnostic tests.
- If female, pregnant, lactating or planning a pregnancy, or having a positive urine pregnancy test prior to the randomization of, or treatment of either eye during the course of the study. [NOTE: If female and capable of becoming pregnant, must agree to use a medically acceptable form of birth control for at least one week prior to the treatment visit and to continue one month following treatment. ]
- 7. Inability to remove soft or scleral contact lenses at least 3 days before initial and follow-up examinations.
- 8. Inability to remove rigid gas-permeable contact lenses at least 2 weeks before initial and follow-up examinations.
- 9. Inability to return for required postoperative examinations. 10. Presence or history or any other condition or finding that, in the investigator's opinion, makes the subject unsuitable as a candidate for crosslinking or study participation or may confound the outcome of the study.
Trial locations and contacts
12 sites in 1 country. Contact the study team or a site near you to ask about taking part.
12 sites in 1 country
United States12 sites, 12 recruiting
- Recruiting
Woolfson Eye Institute
Asheville, North Carolina, 28803
LuAnn Bryant, 770-804-1684 ext. 155, lbryant@woolfsoneye.com
- Recruiting
Woolfson Eye Institute
Atlanta, Georgia, 30328
LuAnn Bryant, 770-804-1684 ext. 155, lbryant@woolfsoneye.com
Jonathan Woolfson, MD
- Recruiting
Woolfson Eye Institute
Atlanta, Georgia, 30349
LuAnn Bryant, 770-804-1684 ext. 155, lbryant@woolfsoneye.com
- Recruiting
Woolfson Eye Institute
Canton, Georgia, 30114
LuAnn Bryant, 770-804-1684 ext. 155, lbryant@woolfsoneye.com
- Recruiting
Woolfson Eye Institute
Chattanooga, Tennessee, 37421
LuAnn Bryant, 770-804-1684 ext. 155, lbryant@woolfsoneye.com
- Recruiting
Woolfson Eye Institute
Cumming, Georgia, 30041
LuAnn Bryant, 770-804-1684 ext. 155, lbryant@woolfsoneye.com
- Recruiting
Woolfson Eye Institute
Douglasville, Georgia, 30134
LuAnn Bryant, 770-804-1684 ext. 155, lbryant@woolfsoneye.com
- Recruiting
Woolfson Eye Institute
Johnson City, Tennessee, 37615
LuAnn Bryant, 770-804-1684 ext. 155
- Recruiting
Woolfson Eye Institute
Knoxville, Tennessee, 37922
LuAnn Bryant, 770-804-1684 ext. 155, lbryant@woolfsoneye.com
- Recruiting
Woolfson Eye Institute
Lawrenceville, Georgia, 30043
LuAnn Bryant, 770-804-1684 ext. 155, lbryant@woolfsoneye.com
- Recruiting
Woolfson Eye Institute
Marietta, Georgia, 30066
LuAnn Bryant, 770-804-1684 ext. 155, lbryant@woolfsoneye.com
- Recruiting
Woolfson Eye Institute
Snellville, Georgia, 30078
LuAnn Bryant, 770-804-1684 ext. 155
About this study
The goal of this clinical trial is to study the effects of an experimental (not Food and Drug Administration (FDA)-approved) treatment called corneal crosslinking (CXL) for conditions in which the cornea becomes progressively thin, steep, and misshapen, causing vision to be blurred.
CXL is performed by putting riboflavin (vitamin B2) drops onto the eye and then exposing it to ultraviolet (UVA) light at about the same intensity as you get outdoors on a bright, sunny day. It is designed to stop the progression of disease by strengthening the cornea. Study participants will be 8 years of age or older and have a diagnosis of Down syndrome.
Read more
The main question it aims to answer is:
- Does CXL help prevent or slow the progression of someone's corneal condition and vision loss?
Participants will:
- Attend up to a total of 7 in office visits over the course of 6 months, where several eye and vision tests will be given.
Receive CXL (applying riboflavin (Vitamin B2 eye drops) to the eye, then exposing the eye to ultraviolet (UV-A) light for 20 minutes).
- Conditions
- Down Syndrome (DS), Keratoconus, Pellucid Marginal Degeneration, Forme Fruste Keratoconus (FFK)
- Being tested
- Epi-ON corneal cross-linking (CXL)
- Keywords
- corneal crosslinking (CXL), cornea, riboflavin, vitamin B2, ultraviolet
- Medical subject headings
- Down Syndrome, Keratoconus, Corneal Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Epi-ON corneal cross-linking (CXL)Combination product
Corneal cross-linking involves the following steps:
- applying riboflavin (Vitamin B2 eye drops) to the eye, then
- exposing the eye to ultraviolet (UV-A) light for 20 minutes.
| Study group | Role | What they receive |
|---|---|---|
| Enrolled and treated subject arm | Experimental | Epi-ON corneal cross-linking (CXL) Receives: Combination Product: Epi-ON corneal cross-linking (CXL) |
What is being measured
The primary outcome is the main question the study is designed to answer.
The primary outcome measures for efficacy is corneal curvature, as measured by Kmax with the Pentacam, in the treated eyes.
Primarily using measurements to assess improvement of vision.
Measured: From enrollment to the end of treatment at 6 months.
Improvement of vision as assessed by corrected distance visual acuity (CDVA).
Change in CDVA compared to the baseline examination will be evaluated at 6 months postoperatively. Data will be summarized using descriptive statistics and experimental groups will be compared using nonparametric tests.
Measured: From enrollment to the end of treatment at 6 months.
Secondary outcomes (3)
Improvement in vision as measured by high order aberrations (HOA).
At the Sponsor-Investigator's discretion, the change in HOA from baseline may be evaluated at 6 months postoperatively for the eyes that underwent preoperative and postoperative wavefront testing. Data will be summarized using descriptive statistics and experimental groups will be compared using nonparametric tests.
Measured: From enrollment until end of follow-up period at 6 months.
Improvement in vision as assessed by change in coma.
At the Sponsor-Investigator's discretion, the change in coma from baseline may be evaluated at 6 months postoperatively for the eyes that underwent preoperative and postoperative wavefront testing. Data will be summarized using descriptive statistics and experimental groups will be compared using nonparametric tests.
Measured: Enrollment through the follow-up phase at 6 months.
Improved vision as assessed by Uncorrected Visual Acuity (UCVA).
Change in UCVA compared to the baseline examination will be evaluated at 6 months postoperatively. Data will be summarized using descriptive statistics and experimental groups will be compared using nonparametric tests.
Measured: Enrollment through the end of the follow-up period at 6 months.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Aug 2025
- Study start1 Aug 2025
- Primary completionNov 2028 (estimated)
- Study completion31 Dec 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Down Syndrome (DS)Studies 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 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 225 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Woolfson Eye Institute
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07135167
- Sponsor's protocol ID
- WEI-CXL-002
- Status last verified
- Jan 2026
- First submitted
- 5 Aug 2025
- Last updated
- 2 Feb 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- Yes
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 2 Feb 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Compassionate Use Study of Epi-ON Corneal Collagen Crosslinking Performed Using UVA Exposure on Eyes With Ectatic Corneal Diseases for Subjects With Down Syndrome
- ClinicalTrials.gov ID
- NCT07135167
- Acronym / sponsor ID
- WEI-CXL-002
- Conditions
- Down Syndrome (DS), Keratoconus, Pellucid Marginal Degeneration, Forme Fruste Keratoconus (FFK)
- Keywords
- corneal crosslinking (CXL), cornea, riboflavin, vitamin B2, ultraviolet
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 225 participants (estimated)
- Lead sponsor
- Woolfson Eye InstituteUniversity, hospital or other
- Study start
- 1 Aug 2025 (actual)
- Primary completion
- Nov 2028 (estimated)
- Study completion
- 31 Dec 2028 (estimated)
- First posted
- 21 Aug 2025 (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.
The goal of this clinical trial is to study the effects of an experimental (not Food and Drug Administration (FDA)-approved) treatment called corneal crosslinking (CXL) for conditions in which the cornea becomes progressively thin, steep, and misshapen, causing vision to be blurred.
CXL is performed by putting riboflavin (vitamin B2) drops onto the eye and then exposing it to ultraviolet (UVA) light at about the same intensity as you get outdoors on a bright, sunny day. It is designed to stop the progression of disease by strengthening the cornea. Study participants will be 8 years of age or older and have a diagnosis of Down syndrome.
The main question it aims to answer is:
- Does CXL help prevent or slow the progression of someone's corneal condition and vision loss?
Participants will:
- Attend up to a total of 7 in office visits over the course of 6 months, where several eye and vision tests will be given.
Receive CXL (applying riboflavin (Vitamin B2 eye drops) to the eye, then exposing the eye to ultraviolet (UV-A) light for 20 minutes).
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Enrolled and treated subject arm | Experimental | Combination Product: Epi-ON corneal cross-linking (CXL)Epi-ON corneal cross-linking (CXL) |
Who may qualify
- Ages
- 8 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (6 listed)
- Be at least 8 years of age or older, male or female, of any race.
- Have a diagnosis of Down syndrome or other cognitive, developmental, or medical conditions that precludes the subject from reliably following instructions or performing some of the study examination procedures.
- Have a diagnosis of keratoconus, pellucid marginal degeneration, progressive ectasia after previous CXL treatment, other ectatic condition, or forme fruste keratoconus based on topography, tomography, and slit lamp examination.
- Provide written informed consent and a signed HIPAA form. Pediatric subjects less than 14 years of age must sign an assent, and a parent or legal guardian must sign an informed consent. If the subject has impaired consent capacity, determined by the consenter, the consent form must be signed by the subject's legally authorized representative on behalf of the subject.
- Satisfactory completion of the standardized clinical decision tool evaluation of patient suitability for CXL. (See Section 7.2.3)
Key exclusion criteria (12 listed)
- Normal corneal topography.
- A history of previous corneal transplant in the study eye.
- Minimum corneal thickness < 300 (measured by Pentacam and Ultrasound) at the screening exam.
- Previous ocular condition (other than refractive error) in the eye(s) to be treated that may predispose the eye to future complications or prevent the possibility of improved vision, for example:
- A known contraindication, sensitivity, or allergy to the test article or its components or to study medications.
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 |
|---|---|---|
| Epi-ON corneal cross-linking (CXL)Corneal cross-linking involves the following steps: * applying riboflavin (Vitamin B2 eye drops) to the eye, then * exposing the eye to ultraviolet (UV-A) light for 20 minutes. | Combination product | Enrolled and treated subject arm |
Primary outcome
The primary outcome measures for efficacy is corneal curvature, as measured by Kmax with the Pentacam, in the treated eyes.
Time frame: From enrollment to the end of treatment at 6 months.
Primarily using measurements to assess improvement of vision.
Improvement of vision as assessed by corrected distance visual acuity (CDVA).
Time frame: From enrollment to the end of treatment at 6 months.
Change in CDVA compared to the baseline examination will be evaluated at 6 months postoperatively. Data will be summarized using descriptive statistics and experimental groups will be compared using nonparametric tests.
3 secondary outcomes
Improvement in vision as measured by high order aberrations (HOA).
Time frame: From enrollment until end of follow-up period at 6 months.
Improvement in vision as assessed by change in coma.
Time frame: Enrollment through the follow-up phase at 6 months.
Improved vision as assessed by Uncorrected Visual Acuity (UCVA).
Time frame: Enrollment through the end of the follow-up period at 6 months.
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 2 Feb 2026.
- Individual participant dataSharing planned: Undecided
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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.
12 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Epi-ON corneal cross-linking (CXL) in people with Down Syndrome (DS) and Keratoconus, run by Woolfson Eye Institute at 12 sites in United States.
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 6 inclusion and 12 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: looking for participants now, as of the sponsor's last update on 2 Feb 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 12 sites in United States. 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 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
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?
Contact LuAnn Bryant (770-804-1684 ext. 155, lbryant@woolfsoneye.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
The primary outcome measures for efficacy is corneal curvature, as measured by Kmax with the Pentacam, in the treated eyes. (time frame: From enrollment to the end of treatment at 6 months.). The study lists 2 primary outcomes in all. 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 2 Feb 2026.