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Completed

NCT Number: NCT01584076

Treatment of Residual Amblyopia With Donepezil

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.

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Key information

Conditions

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

Age range

8 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1

Primary location

Boston Children's Hospital, Boston, Massachusetts, United States

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Who can participate

Healthy volunteers accepted: Yes

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Age ≥8 years
  • Amblyopia associated with strabismus and/or anisometropia
  • Amblyopic eye visual acuity of 20/50 - 20/400
  • Sound eye visual acuity of ≥20/25
  • 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)
  • For ≥18 year olds, history of prior amblyopia treatment with patching
  • 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)
  • Complete eye examination within 6 months prior to enrollment
  • Available for at least 6 months of follow-up, have access to a phone, and willing to be contacted by clinical staff
  • Likely to comply with prescribed treatment and unlikely, if applicable, to continue to improve with 2 hours of daily patching alone

Exclusion criteria

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

Treatment and study plan

Donepezil

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.

Other names: Aricept

Patching

Other

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

Primary outcomes

  1. Amblyopia Eye Visual Acuity Improvement

    Time frame: 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 outcomes

  1. Amblyopic Eye Visual Acuity

    Time frame: 4, 8, 12, and 22 weeks after enrollment

    Analysis of amblyopia eye visual acuity measured at each visit.

  2. Recurrence of Amblyopia after 10 Weeks Off Study Treatment

    Time frame: 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.

  3. Adverse Events

    Time frame: 4, 8, 12, and 22 weeks after enrollment

    Analysis of the proportion of subjects reporting adverse events.

  4. Adverse Events Requiring Discontinuation of Study Treatment

    Time frame: 4, 8, and 12 weeks after enrollment

    Analysis of the proportion of subjects requiring discontinuation of study treatment secondary to adverse events.

  5. Completion of Study Treatment

    Time frame: 12 weeks after enrollment

    Analysis of the proportion of subjects completing study treatment.

  6. Sound Eye Visual Acuity

    Time frame: 22 weeks after enrollment

    Analysis of sound eye visual acuity at 22 weeks to assess any adverse effect on the occluded eye.

Sponsors and collaborators

Lead sponsor

Boston Children's Hospital

Other

Registry information

Official study title

Recovery From Amblyopia With Cholinesterase Inhibitors

Important dates

Study start
2012
Primary completion
2021
Study completion
2021
First posted
Apr 24, 2012
Registry last updated
Jun 18, 2021

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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