Evaluation of Echothiophate in Pediatric Patients With Refractory Amblyopia

NCT07554131 | Not Yet Recruiting Phase 2 FDA Drug

• 1 other identifier: 19462
• Study Type: interventional
• Target: 20
• Locations: 1 country
• Sites: 1

Brief 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. 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.

Conditions

Refractory Amblyopia

Keywords

Refractory Amblyopia; Amblyopia

Outcome Measures

Primary Outcomes (1)

• 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)

  Baseline, 4 weeks, 8 weeks, 12 weeks, 14 weeks.

Secondary Outcomes (14)

• Interocular visual acuity difference

  Baseline, 4 weeks, 8 weeks, 12 weeks, and 14 weeks.

• Change in stereoacuity

  4 weeks, 8 weeks, 12 weeks, 14 weeks

• Refractive error changes

  4 weeks, 8 weeks, 12 weeks (after completion of echothiophate treatment), 14 weeks (after two-week wash-out period).

• Ocular alignment

  Baseline, 4 weeks, 8 weeks, 12 weeks, and 14 weeks.

• Contrast sensitivity

  Baseline, 4 weeks, 8 weeks, 12 weeks, 14 weeks

Study Arms (1)

Atropine + Echothiophate Combination Therapy

OTHER

Single-group interventional Arm - Atropine + Echothiophate Combination Therapy

Drug: Echothiophate Iodide 0.125% Ophthalmic Solution

Interventions

Echothiophate Iodide 0.125% Ophthalmic Solution DRUG

Participants will receive echothiophate iodide eye drops in addition to standard atropine therapy to assess whether the combination improves visual function in refractory amblyopia.

Atropine + Echothiophate Combination Therapy

Eligibility Criteria

• Age: 3 Years - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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.

You may not qualify if:

• 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.
• 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

Sponsors & Collaborators

• University of Oklahoma: lead
• Fera Pharmaceuticals, LLC: collaborator

Study Sites (1)

Dean McGee Eye Institute

Oklahoma City, Oklahoma, 73104, United States

Location

MeSH Terms

Conditions

Amblyopia

Interventions

Echothiophate Iodide; Ophthalmic Solutions

Condition Hierarchy (Ancestors)

Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Vision Disorders; Sensation Disorders; Neurologic Manifestations; Eye Diseases; Signs and Symptoms; Pathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

Organothiophosphates; Organophosphates; Organophosphorus Compounds; Organic Chemicals; Organothiophosphorus Compounds; Sulfur Compounds; Pharmaceutical Solutions; Solutions; Pharmaceutical Preparations; Therapeutic Uses; Pharmacologic Actions; Chemical Actions and Uses; Specialty Uses of Chemicals

Study Officials

• Tammy Yanovitch, MD

  University of Oklahoma

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Crystal McAfee

CONTACT

405-271-6307; Crystal-McAfee@dmei.org

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 23, 2026
• First Posted: April 28, 2026
• Study Start (Estimated): June 1, 2026
• Primary Completion (Estimated): December 1, 2026
• Study Completion (Estimated): December 1, 2026
• Last Updated: April 28, 2026

Record last verified: 2026-04

Locations

US (1)