Botulinum Toxin A vs Strabismus Surgery for Esotropia

NCT07470164 | Not Yet Recruiting Phase 3 DMC FDA Drug

• 2 other identifiers: ETS4UG1EY011751
• Study Type: interventional
• Target: 244
• Locations: 0 countries
• Sites: N/A

Brief Summary

Childhood esotropia (ET) is a common cause of visual disability, and its early management is critical for optimal visual and developmental outcomes. The proposed study addresses a need to evaluate botulinum toxin A (BTX-A) as a less invasive, cost-effective alternative to incisional strabismus surgery. If BTX-A is demonstrated to be non-inferior to incisional strabismus surgery in treating ET \>10 to ≤30PD, this could reduce surgical and anesthesia-related risks in infants, lower the economic burden on families and healthcare systems, and reduce disparities in access to care, particularly in underserved settings where surgical resources are limited.

Conditions

Esotropia

Keywords

strabismus; pediatric; botox; botulinum toxin a

Outcome Measures

Primary Outcomes (1)

• Motor Alignment Success

  The primary efficacy outcome will be motor alignment success defined as follows: 1. Success is defined as the absence of failure by 1 year. 2. Failure is defined as any of the following post-randomized treatment (BOTOX or SURGERY) up to 1 year: * ET at distance (determined by a cover/uncover test) with a magnitude of \>10 PD by SPCT at or after the 6-month masked exam. * XT at distance at any time during the exam (determined by a cover/uncover test) with a magnitude of \> 10 PD by SPCT at or after the 6-month masked exam. * Second surgery or injection at any time after the initial surgery/injection through 12 months post-procedure.

  1 Year

Study Arms (2)

Botox

EXPERIMENTAL

Group assigned to receive BTX-A injection

Drug: Botulinum toxin A

Surgery

ACTIVE COMPARATOR

Group assigned to receive Graded bilateral medial rectus recession

Procedure: bilateral medial rectus recession

Interventions

Botulinum toxin A DRUG

BTX-A 4.0 units to each medial rectus

Also known as: Botox

Botox

bilateral medial rectus recession PROCEDURE

Graded bilateral medial rectus recession

Also known as: BMRrec

Surgery

Eligibility Criteria

• Age: 4 Months - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

1. A constant or intermittent ET needing intervention with BTX-A or surgery in the judgement of the investigator meeting one of the criteria below: Infantile Esotropia (IET) * Onset prior to six months of age by parental history. * Angle measuring \>10 to ≤ 30 PD by PACT in the primary position at distance fixation on an accommodative target. Acquired Non-accommodative Esotropia (ANAET) * Onset after six months of age by parental history * Angle decreases less than 10 PD by PACT in the primary position at distance fixation with hyperopic correction. * Residual angle measuring \>10 to ≤ 30 PD by SPCT in the primary position at distance fixation on an accommodative target. Acquired Partially Accommodative Esotropia (APAET) * Onset after six months of age by parental history * Angle decreases 10 PD or more by PACT in the primary position at distance fixation with correction. * Residual angle measuring \>10 to ≤ 30 PD by SPCT in the primary position at distance fixation on an accommodative target. 2. Age at enrollment: * For IET: \> 4 months to \<17 years old * For ANAET or APAET: \>6 months to \<17 years old 3. No myopia ≥ 6.00D SE 4. Current Spectacle Correction at Time of Enrollment * For IET: No spectacle wear prior to enrollment. * For ANAET or APAET: Must be wearing spectacles if significant refractive error as defined below based upon a cycloplegic refraction within 6 months of enrollment. If no significant refractive error, spectacles are at investigator discretion. In either case full hyperopic correction by cycloplegia worn for at least 2 weeks. 5. Requirements for Spectacle Correction (if worn) * If myopia is \< 6.00D SE, then full CR must be worn for at least 2 weeks. * For children \< 5 years of age who have significant hyperopic refractive error (CR ≥ +2.50D SE), full hyperopic correction, determined by CR must have been worn for at least 2 weeks. * For children ≥ age 5 years, with significant hyperopic refractive error who are unable to tolerate correction of full CR due to blur, then maximally-tolerated hyperopic correction determined both with and without cycloplegia is prescribed and must be worn for at least 2 weeks. * For patients who do not have significant hyperopic refractive error (CR \< +2.50D SE), whether to prescribe spectacles is at investigator discretion. However, if the investigator elects to prescribe spectacles, the full tolerated hyperopic correction determined with and without cycloplegia must have been worn for at least 2 weeks. 6. Parent or legal guardian available for follow-up, has home phone (or access to phone), and willing to be contacted by Jaeb Center staff 7. Gestational age \> 34 weeks 8. Birth weight \> 1500 grams 9. No use of atropine within the last two weeks. 10. No history of CNS disease (e.g., IVH, PVL, meningitis, developmental abnormalities of the brain, hydrocephalus, cerebral palsy, hypoxic ischemic encephalopathy) 11. No significant developmental delay in the investigator's judgment (isolated speech delay excepted) 12. No limitation of abduction consistent with paralytic or restrictive myopathy (minor limitation of abduction due to cross fixation is acceptable) 13. No craniofacial malformation affecting the orbits 14. No prior BTX-A injection for strabismus 15. No prior extraocular muscle surgery or intraocular surgery 16. No structural ocular abnormalities (e.g., media opacity, optic atrophy, optic nerve hypoplasia, retinal detachment) 17. No immediate family member (spouse, biological or legal guardian, child, sibling, parent) who is investigative site personnel directly affiliated with this study or who is an employee of the Jaeb Center for Health Research.

Contact the study team to discuss eligibility requirements. They can help determine if this study is right for you.

Sponsors & Collaborators

• Jaeb Center for Health Research: lead
• National Eye Institute (NEI): collaborator
• Pediatric Eye Disease Investigator Group: collaborator

Related Links

• PEDIG Public Website

MeSH Terms

Conditions

Esotropia; Strabismus

Interventions

Botulinum Toxins, Type A

Condition Hierarchy (Ancestors)

Ocular Motility Disorders; Cranial Nerve Diseases; Nervous System Diseases; Eye Diseases

Intervention Hierarchy (Ancestors)

Botulinum Toxins; Metalloendopeptidases; Endopeptidases; Peptide Hydrolases; Hydrolases; Enzymes; Enzymes and Coenzymes; Metalloproteases; Bacterial Proteins; Proteins; Amino Acids, Peptides, and Proteins; Bacterial Toxins; Toxins, Biological; Biological Factors

Central Study Contacts

Raymond T Kraker, MSPH

CONTACT

813-975-8690; rkraker@jaeb.org

PEDIG Protocol Monitor

CONTACT

813-975-8690; pedig@jaeb.org

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: SINGLE
• Who Masked: OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 10, 2026
• First Posted: March 13, 2026
• Study Start (Estimated): January 1, 2027
• Primary Completion (Estimated): January 1, 2030
• Study Completion (Estimated): January 1, 2030
• Last Updated: March 13, 2026

Record last verified: 2026-03

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, ICF
• Time Frame: Data will be made available after publication of each primary manuscript.
• Access Criteria: Users accessing the data must enter an email address.