Treatment of Residual Amblyopia With Donepezil

NCT ID: NCT01584076

Last Updated: 2021-06-18

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE1
• Total Enrollment: 18 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2012-08-31
• Study Completion Date: 2021-01-31

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.

Conditions

Amblyopia

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Donepezil

Group Type: EXPERIMENTAL

Donepezil

Intervention Type: DRUG

Oral Donepezil Daily

Initial Dosage: Donepezil 5 mg tablets will be used. 1/2 tablet (≈2.5 mg)/day for 8 to 17 year olds OR 1 tablet (5 mg)/day for ≥18 year olds.

Dosage Escalation: Donepezil may be increased by 1/2 tablet (≈2.5 mg)/day every 4 weeks if the amblyopic eye visual acuity has not improved by ≥5 letters or 1 logMAR line to a maximum dosage of 1 1/2 tablets (≈7.5 mg)/day for 8 to 17 year olds OR 2 tablets (10 mg)/day for ≥18 year olds.

Patching

Intervention Type: OTHER

Patching: 2 hours of daily patching will also be prescribed for 8 to 17 year olds only.

Interventions

Donepezil

Oral Donepezil Daily

Initial Dosage: Donepezil 5 mg tablets will be used. 1/2 tablet (≈2.5 mg)/day for 8 to 17 year olds OR 1 tablet (5 mg)/day for ≥18 year olds.

Dosage Escalation: Donepezil may be increased by 1/2 tablet (≈2.5 mg)/day every 4 weeks if the amblyopic eye visual acuity has not improved by ≥5 letters or 1 logMAR line to a maximum dosage of 1 1/2 tablets (≈7.5 mg)/day for 8 to 17 year olds OR 2 tablets (10 mg)/day for ≥18 year olds.

Intervention Type: DRUG

Patching

Patching: 2 hours of daily patching will also be prescribed for 8 to 17 year olds only.

Intervention Type: OTHER

Other Intervention Names

Aricept

Eligibility Criteria

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

• Minimum Eligible Age: 8 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: Yes

Sponsors

Boston Children's Hospital

OTHER

Sponsor Role: lead

Responsible Party

Carolyn Wu

Assistant Professor of Ophthalmology

Responsibility Role: PRINCIPAL_INVESTIGATOR

Principal Investigators

Carolyn Wu, MD

Role: PRINCIPAL_INVESTIGATOR

Boston Children's Hospital

David G. Hunter, MD, PhD

Role: PRINCIPAL_INVESTIGATOR

Boston Children's Hospital

Bharti Gangwani, MD

Role: PRINCIPAL_INVESTIGATOR

Boston Children's Hospital

Locations

Boston Children's Hospital

Boston, Massachusetts, United States

Boston Children's Hospital at Waltham

Waltham, Massachusetts, United States

Boston Children's Physicians South

Weymouth, Massachusetts, United States

Countries

United States

References

Morishita H, Miwa JM, Heintz N, Hensch TK. Lynx1, a cholinergic brake, limits plasticity in adult visual cortex. Science. 2010 Nov 26;330(6008):1238-40. doi: 10.1126/science.1195320. Epub 2010 Nov 11.

Reference Type: BACKGROUND

PMID: 21071629 (View on PubMed)

Other Identifiers

IRB-P00002887

Identifier Type: -

Identifier Source: org_study_id