Treatment of Residual Amblyopia With Donepezil

Phase 1

Completed

• Conditions: Amblyopia
• Interventions: Drug: Donepezil; Other: Patching

• Registration Number: NCT01584076
• Lead Sponsor: Boston Children's Hospital

Brief Summary

Amblyopia is the leading cause of monocular visual impairment in children and adults. Despite conventional treatment with patching or eye drops, many older children and adults do not achieve normal vision in the amblyopic eye.

Donepezil is an acetylcholinesterase inhibitor that increases levels of the neurotransmitter acetylcholine in the brain. Use of acetylcholinesterase inhibitors has been demonstrated by the Hensch lab (Department of Neurology, FM Kirby Neurobiology Center) at Boston Children's Hospital to improve vision and reverse amblyopia in animal models.

The purpose of this study is to evaluate the efficacy of oral donepezil as treatment for residual amblyopia (20/50 - 20/400) in patients 8 years of age and older.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2012-04-23
• Study First Submitted QC: 2012-04-23
• Study First Posted: 2012-04-24
• Study Start: 2012-08
• Primary Completion: 2021-01
• Study Completion: 2021-01
• Status Verified: 2021-06
• Last Update Submitted: 2021-06-16
• Last Update Posted: 2021-06-18

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 18

Inclusion Criteria

1. Age ≥8 years
2. Amblyopia associated with strabismus and/or anisometropia
3. Amblyopic eye visual acuity of 20/50 - 20/400
4. Sound eye visual acuity of ≥20/25
5. For 8 to 17 year olds, current amblyopia treatment of at least 2 hours of daily patching for at least 4 weeks during the pre-enrollment period with no improvement in best-corrected amblyopic eye visual acuity (<5 letters or 1 logMAR line between 2 consecutive visual acuity measurements at least 4 weeks apart while on current treatment)
6. For ≥18 year olds, history of prior amblyopia treatment with patching
7. Wearing optimal optical correction with stable amblyopic eye visual acuity (<5 letters or 1 logMAR line of improvement during 2 consecutive visual acuity measurements at least 4 weeks apart)
8. Complete eye examination within 6 months prior to enrollment
9. Available for at least 6 months of follow-up, have access to a phone, and willing to be contacted by clinical staff
10. Likely to comply with prescribed treatment and unlikely, if applicable, to continue to improve with 2 hours of daily patching alone

Exclusion Criteria

1. Myopia more than -6.00 D spherical equivalent
2. Presence of associated findings that could cause reduced visual acuity
3. Previous intraocular or refractive surgery
4. Strabismus surgery planned within 22 weeks
5. Current vision therapy or orthoptics
6. Treatment with topical atropine within the past 4 weeks
7. Presence of cardiac condition, asthma, obstructive pulmonary disease, seizure disorder, urinary incontinence, and/or peptic ulcer disease receiving concurrent NSAIDs
8. History of gastrointestinal bleeding from peptic ulcer disease
9. Known psychological problems
10. Known skin reaction to patch or bandage adhesives for 8 to 17 year olds
11. Known allergies or contraindications to the use of acetylcholinesterase inhibitors
12. Prior acetylcholinesterase inhibitor treatment
13. Current use of medication for the treatment of ADHD or psychological disorders
14. Inability to swallow pills equivalent in size to the 5 mg donepezil tablet
15. Females who are pregnant, lactating, or intending to become pregnant within the next 6 months

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Donepezil	Patching	-
Donepezil	Donepezil	-

Primary Outcome Measures

Name	Time	Method
Amblyopia Eye Visual Acuity Improvement	22 weeks after enrollment	Study treatment will last for 12 weeks. Primary outcome measure is analysis of the proportion of subjects with improvement in amblyopic eye visual acuity of ≥ 15 letters or 3 logMAR lines at 22 weeks after 10 weeks off study treatment.

Secondary Outcome Measures

Name	Time	Method
Amblyopic Eye Visual Acuity	4, 8, 12, and 22 weeks after enrollment	Analysis of amblyopia eye visual acuity measured at each visit.
Recurrence of Amblyopia after 10 Weeks Off Study Treatment	22 weeks after enrollment	Study treatment will be discontinued after 12 weeks. Amblyopic eye visual acuity at 12 weeks and 22 weeks will be compared. Analysis of the proportion of subjects with recurrence of amblyopia after 10 weeks off study treatment.
Adverse Events	4, 8, 12, and 22 weeks after enrollment	Analysis of the proportion of subjects reporting adverse events.
Adverse Events Requiring Discontinuation of Study Treatment	4, 8, and 12 weeks after enrollment	Analysis of the proportion of subjects requiring discontinuation of study treatment secondary to adverse events.
Completion of Study Treatment	12 weeks after enrollment	Analysis of the proportion of subjects completing study treatment.
Sound Eye Visual Acuity	22 weeks after enrollment	Analysis of sound eye visual acuity at 22 weeks to assess any adverse effect on the occluded eye.

Trial Locations

Locations (3)

Boston Children's Physicians South; 🇺🇸 Weymouth, Massachusetts, United States

Boston Children's Hospital; 🇺🇸 Boston, Massachusetts, United States

Boston Children's Hospital at Waltham; 🇺🇸 Waltham, Massachusetts, United States