Refractory AmblyopiaPhase 2NCT07554131
Evaluation of Echothiophate in Pediatric Patients With Refractory Amblyopia
A Phase 2 study testing Echothiophate Iodide 0.125% Ophthalmic Solution in people with Refractory Amblyopia, run by University of Oklahoma at 1 site in United States.
Trial summary
The goal of this clinical trial is to see if a medicine called echothiophate iodide (Phospholine Iodide) can help treat children with refractory amblyopia. Amblyopia (also called lazy eye) is when one eye doesn't see as well as it should because the brain isn't using that eye correctly. Read more about this study
- Who is it for?
- Ages 3 to 17 years
- Conditions the study is for
- Refractory Amblyopia
- What is being tested?
- Echothiophate Iodide 0.125% Ophthalmic Solution
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 20 participants
- Where
- 1 siteUnited States
- Study dates
- Jun 2026to Dec 2026
- Phase
- Phase 2
- 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 3 to 17 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Children/adolescents between 3 and <18 years old with amblyopia who are currently undergoing atropine treatment who are deemed to no longer be improving.
- Diagnosis of amblyopia associated with strabismus, anisometropia, or mixed types (as defined by previous PEDIG studies).
- Wearing appropriate spectacle correction based on cycloplegic refraction using PEDIG protocol recommendations.
- Currently undergoing atropine treatment (either 2x a week or every other day) with visual acuity in the amblyopic eye that has not improved with atropine treatment from participant's previous visit.
- Cycloplegic refraction that has been completed within 6 months of enrollment.
- Ability to measure visual acuity using standardized testing protocols.
- Visual acuity in the amblyopic eye 20/40 to 20/100. Visual acuity in the non-amblyopic eye 20/30 or better with intraocular difference greater than or equal to 2 lines.
Who cannot join
- Existing ocular conditions likely to interfere with treatment response (e.g., congenital cataract history, corneal opacities/scars, ptosis, retinal diseases).
- Neurologic or systemic conditions that impact vision.
- Severe developmental delays that might impact treatment adherence and outcome evaluation.
- Current use of prisms or bifocals.
- Active uveal inflammation or history of angle-closure glaucoma.
- Concurrent use of other anticholinesterase medications.
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- Anticipated surgical procedures during the study period.
- Prior surgical interventions for ocular conditions that could interfere with study outcomes.
- Myopia with spherical equivalent >6D.
- Any contraindications to the components of the medicine to be used in the study
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site
Dean McGee Eye Institute
Oklahoma City, Oklahoma, 73104
Crystal McAfee, 405-271-6307, Crystal-McAfee@dmei.org
Tammy Yanovitch, MD, MHSc
About this study
The goal of this clinical trial is to see if a medicine called echothiophate iodide (Phospholine Iodide) can help treat children with refractory amblyopia. Amblyopia (also called lazy eye) is when one eye doesn't see as well as it should because the brain isn't using that eye correctly. Refractory amblyopia means a lazy eye that's very difficult to treat and it doesn't improve even after using common treatments, such as putting atropine eye drops in the stronger eye to encourage use of the weaker eye.
The main questions this study aims to answer are:
Read more
- Can adding echothiophate iodide eye drops help children who stopped improving with atropine alone start making progress again?
- Does using atropine and echothiophate iodide eye drops together work better for older children and adolescents who typically do not respond well to atropine alone?
- Besides improving eyesight, can echothiophate iodide eye drops also help with things like depth perception, seeing in crowded spaces, or noticing motion
Participants will be asked to:
- Participate in the study for a total of 14 weeks
- Attend four clinic visits
- Use atropine drops in the stronger eye nightly for a total 12 weeks
- Use of echothiophate iodide eye drops in the weaker eye in addition to atropine use in the stronger eye, if vision does not improve after 4 weeks of use of atropine alone in the stronger eye
- Upload weekly photos of the eyes
- Have a slit lamp exam performed at each visit
- Have Best Corrected Visual Acuity (BCVA) eye measurements taken at each visit.
- Have Optical Coherence Tomography Angiography (OCTA) images taken at at each visit.
- Conditions
- Refractory Amblyopia
- Being tested
- Echothiophate Iodide 0.125% Ophthalmic Solution
- Keywords
- Refractory Amblyopia, Amblyopia
- Medical subject headings
- Amblyopia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Echothiophate Iodide 0.125% Ophthalmic SolutionDrug
Participants will receive echothiophate iodide eye drops in addition to standard atropine therapy to assess whether the combination improves visual function in refractory amblyopia.
| Study group | Role | What they receive |
|---|---|---|
| Atropine + Echothiophate Combination Therapy | Other | Single-group interventional Arm - Atropine + Echothiophate Combination Therapy Receives: Drug: Echothiophate Iodide 0.125% Ophthalmic Solution |
What is being measured
The primary outcome is the main question the study is designed to answer.
Best Corrected Visual Acuity (BCVA)
Best Corrected Visual Acuity (BCVA) after 8 weeks of echothiophate iodide treatment and after a 2-week washout period (study-certified visual acuity tester masked to the patient's treatment group)
Measured: Baseline, 4 weeks, 8 weeks, 12 weeks, 14 weeks.
Secondary outcomes (14)
Interocular visual acuity difference
Difference in BCVA between the amblyopic and fellow eye, measured in logMAR.
Measured: Baseline, 4 weeks, 8 weeks, 12 weeks, and 14 weeks.
Change in stereoacuity
Change in log arcseconds of stereoacuity assessed with the Randot Preschool Stereoacuity Test.
Measured: 4 weeks, 8 weeks, 12 weeks, 14 weeks
Refractive error changes
Changes in spherical equivalent refraction from baseline in the amblyopic eye, measured in diopters.
Measured: 4 weeks, 8 weeks, 12 weeks (after completion of echothiophate treatment), 14 weeks (after two-week wash-out period).
Ocular alignment
This measure evaluates the presence and magnitude of ocular deviation measured in prism diopters, assessed at distance and near fixation
Measured: Baseline, 4 weeks, 8 weeks, 12 weeks, and 14 weeks.
Contrast sensitivity
The ability of the amblyopic eye to distinguish an object from its background under varying levels of contrast, assessed using a standardized contrast sensitivity chart (e.g., Pelli-Robson) and reported in log contrast sensitivity units.
Measured: Baseline, 4 weeks, 8 weeks, 12 weeks, 14 weeks
Quality of life measures using the Pediatric Eye Disease Investigator Group (PEDIG) Pediatric Eye Questionnaire (PedEyeQ)
The PedEyeQ is an instrument specific to pediatric eye conditions designed to evaluate health-related quality of life in children with eye conditions. The assessment values are Rasch calibrated and scaled to score from 0 (worst) to 100 (best)
Measured: Baseline, 4 weeks, 8 weeks, 12 weeks, 14 weeks
Treatment adherence
This measure assess the proportion of prescribed treatments completed as directed over the study period, including frequency and consistency of administration
Measured: From baseline to the end of treatment at 14 weeks.
OCT-A foveal avascular zone (FAZ) area changes
Examine difference/change in OCT-A foveal avascular zone (FAZ) area between amblyopic and non-amblyopic eye with treatment. Units are in square millimeters (mm^2)
Measured: 4 weeks, 8 weeks, 12 weeks, 14 weeks
OCT-A foveal avascular zone (FAZ) perimeter changes
Examine changes in OCT-A foveal avascular zone (perimeter) changes between the amblyopic and non-amblyopic eye with treatment measured in millimeters (mm).
Measured: 4 weeks, 8 weeks, 12 weeks, 14 weeks
OCT-A foveal avascular zone (FAZ) circularity index changes
Examine changes in foveal avascular zone (FAZ) circularity index between the amblyopic and non-amblyopic eye with treatment. Circularity is measured by utilizing the FAZ area and perimeter by multiplying the area by 4 pi and dividing by the squared perimeter. This measurement is unitless.
Measured: 4 weeks, 8 weeks, 12 weeks, 14 weeks
OCT-A vessel density changes
Examine the differences in OCT-A between amblyopic and non-amblyopic eye with treatment. Measured as the proportion of the area occupied by vasculature.
Measured: 4 weeks, 8 weeks, 12 weeks, 14 weeks
OCT-A vessel diameter index (VDI) changes
Examine the changes in the VDI between amblyopic and non-amblyopic eye with treatment. Measured as the average vessel caliber in micrometers (µm).
Measured: 4 weeks, 8 weeks, 12 weeks, 14 weeks
OCT-A perfusion density (PD) changes
Changes in PD between the amblyopic and non-amblyopic eye with treatment. Measured as the total area of perfused vasculature per unit area (%)
Measured: 4 weeks, 8 weeks, 12 weeks, 14 weeks
OCT-A retinal thickness changes
Examine changes in retinal thickness between amblyopic and non-amblyopic eye with treatment. Measured in microns.
Measured: 4 weeks, 8 weeks, 12 weeks, 14 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Apr 2026
- Study startJun 2026 (estimated)
- Primary completionDec 2026 (estimated)
- Study completionDec 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Refractory AmblyopiaStudies 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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 20 participants (estimated)
Sponsor and investigators
- Lead sponsor
- University of Oklahoma
- Sponsor type
- University, hospital or other
- Collaborators
- Fera Pharmaceuticals, LLC
- Responsible party
- Sponsor
- Investigators
- Tammy Yanovitch, MD, Principal investigator, University of Oklahoma
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07554131
- Sponsor's protocol ID
- 19462
- Status last verified
- Apr 2026
- First submitted
- 23 Feb 2026
- Last updated
- 28 Apr 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 28 Apr 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Evaluation of Echothiophate in Pediatric Patients With Refractory Amblyopia
- ClinicalTrials.gov ID
- NCT07554131
- Acronym / sponsor ID
- 19462
- Conditions
- Refractory Amblyopia
- Keywords
- Refractory Amblyopia, Amblyopia
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 20 participants (estimated)
- Lead sponsor
- University of OklahomaUniversity, hospital or other
- Collaborators
- Fera Pharmaceuticals, LLC
- Study start
- Jun 2026 (estimated)
- Primary completion
- Dec 2026 (estimated)
- Study completion
- Dec 2026 (estimated)
- First posted
- 28 Apr 2026 (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 see if a medicine called echothiophate iodide (Phospholine Iodide) can help treat children with refractory amblyopia. Amblyopia (also called lazy eye) is when one eye doesn't see as well as it should because the brain isn't using that eye correctly. Refractory amblyopia means a lazy eye that's very difficult to treat and it doesn't improve even after using common treatments, such as putting atropine eye drops in the stronger eye to encourage use of the weaker eye.
The main questions this study aims to answer are:
- Can adding echothiophate iodide eye drops help children who stopped improving with atropine alone start making progress again?
- Does using atropine and echothiophate iodide eye drops together work better for older children and adolescents who typically do not respond well to atropine alone?
- Besides improving eyesight, can echothiophate iodide eye drops also help with things like depth perception, seeing in crowded spaces, or noticing motion
Participants will be asked to:
- Participate in the study for a total of 14 weeks
- Attend four clinic visits
- Use atropine drops in the stronger eye nightly for a total 12 weeks
- Use of echothiophate iodide eye drops in the weaker eye in addition to atropine use in the stronger eye, if vision does not improve after 4 weeks of use of atropine alone in the stronger eye
- Upload weekly photos of the eyes
- Have a slit lamp exam performed at each visit
- Have Best Corrected Visual Acuity (BCVA) eye measurements taken at each visit.
- Have Optical Coherence Tomography Angiography (OCTA) images taken at at each visit.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Atropine + Echothiophate Combination Therapy | Other | Drug: Echothiophate Iodide 0.125% Ophthalmic SolutionSingle-group interventional Arm - Atropine + Echothiophate Combination Therapy |
Who may qualify
- Ages
- 3 to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (7 listed)
- Children/adolescents between 3 and <18 years old with amblyopia who are currently undergoing atropine treatment who are deemed to no longer be improving.
- Diagnosis of amblyopia associated with strabismus, anisometropia, or mixed types (as defined by previous PEDIG studies).
- Wearing appropriate spectacle correction based on cycloplegic refraction using PEDIG protocol recommendations.
- Currently undergoing atropine treatment (either 2x a week or every other day) with visual acuity in the amblyopic eye that has not improved with atropine treatment from participant's previous visit.
- Cycloplegic refraction that has been completed within 6 months of enrollment.
Key exclusion criteria (10 listed)
- Existing ocular conditions likely to interfere with treatment response (e.g., congenital cataract history, corneal opacities/scars, ptosis, retinal diseases).
- Neurologic or systemic conditions that impact vision.
- Severe developmental delays that might impact treatment adherence and outcome evaluation.
- Current use of prisms or bifocals.
- Active uveal inflammation or history of angle-closure glaucoma.
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 |
|---|---|---|
| Echothiophate Iodide 0.125% Ophthalmic SolutionParticipants will receive echothiophate iodide eye drops in addition to standard atropine therapy to assess whether the combination improves visual function in refractory amblyopia. | Drug | Atropine + Echothiophate Combination Therapy |
Primary outcome
Best Corrected Visual Acuity (BCVA)
Time frame: Baseline, 4 weeks, 8 weeks, 12 weeks, 14 weeks.
Best Corrected Visual Acuity (BCVA) after 8 weeks of echothiophate iodide treatment and after a 2-week washout period (study-certified visual acuity tester masked to the patient's treatment group)
14 secondary outcomes
Interocular visual acuity difference
Time frame: Baseline, 4 weeks, 8 weeks, 12 weeks, and 14 weeks.
Change in stereoacuity
Time frame: 4 weeks, 8 weeks, 12 weeks, 14 weeks
Refractive error changes
Time frame: 4 weeks, 8 weeks, 12 weeks (after completion of echothiophate treatment), 14 weeks (after two-week wash-out period).
Ocular alignment
Time frame: Baseline, 4 weeks, 8 weeks, 12 weeks, and 14 weeks.
Contrast sensitivity
Time frame: Baseline, 4 weeks, 8 weeks, 12 weeks, 14 weeks
Quality of life measures using the Pediatric Eye Disease Investigator Group (PEDIG) Pediatric Eye Questionnaire (PedEyeQ)
Time frame: Baseline, 4 weeks, 8 weeks, 12 weeks, 14 weeks
Treatment adherence
Time frame: From baseline to the end of treatment at 14 weeks.
OCT-A foveal avascular zone (FAZ) area changes
Time frame: 4 weeks, 8 weeks, 12 weeks, 14 weeks
OCT-A foveal avascular zone (FAZ) perimeter changes
Time frame: 4 weeks, 8 weeks, 12 weeks, 14 weeks
OCT-A foveal avascular zone (FAZ) circularity index changes
Time frame: 4 weeks, 8 weeks, 12 weeks, 14 weeks
OCT-A vessel density changes
Time frame: 4 weeks, 8 weeks, 12 weeks, 14 weeks
OCT-A vessel diameter index (VDI) changes
Time frame: 4 weeks, 8 weeks, 12 weeks, 14 weeks
OCT-A perfusion density (PD) changes
Time frame: 4 weeks, 8 weeks, 12 weeks, 14 weeks
OCT-A retinal thickness changes
Time frame: 4 weeks, 8 weeks, 12 weeks, 14 weeks
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 28 Apr 2026.
- Individual participant dataSharing planned: Undecided
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.
Tammy Yanovitch, MD principal investigator
University of Oklahoma
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Echothiophate Iodide 0.125% Ophthalmic Solution in people with Refractory Amblyopia, run by University of Oklahoma at 1 site 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 7 inclusion and 10 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: opening soon, as of the sponsor's last update on 28 Apr 2026. 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 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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 Crystal McAfee (405-271-6307, Crystal-McAfee@dmei.org), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Best Corrected Visual Acuity (BCVA) (time frame: Baseline, 4 weeks, 8 weeks, 12 weeks, 14 weeks.). 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 28 Apr 2026.