Back to resultsTreatment of Residual Amblyopia With Donepezil
Primary Purpose
Amblyopia
Status
Completed
Phase
Phase 1
Locations
United States
Study Type
Interventional
Intervention
Donepezil
Patching
Sponsored by
About this trial
This is an interventional treatment trial for Amblyopia focused on measuring Amblyopia, Donepezil
Eligibility Criteria
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
Sites / Locations
- Boston Children's Hospital
- Boston Children's Hospital at Waltham
- Boston Children's Physicians South
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Donepezil
Arm Description
Outcomes
Primary Outcome Measures
Amblyopia Eye Visual Acuity Improvement
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
Amblyopic Eye Visual Acuity
Analysis of amblyopia eye visual acuity measured at each visit.
Recurrence of Amblyopia after 10 Weeks Off Study Treatment
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
Analysis of the proportion of subjects reporting adverse events.
Adverse Events Requiring Discontinuation of Study Treatment
Analysis of the proportion of subjects requiring discontinuation of study treatment secondary to adverse events.
Completion of Study Treatment
Analysis of the proportion of subjects completing study treatment.
Sound Eye Visual Acuity
Analysis of sound eye visual acuity at 22 weeks to assess any adverse effect on the occluded eye.
Full Information
NCT ID
NCT01584076
First Posted
April 23, 2012
Last Updated
June 16, 2021
Sponsor
Boston Children's Hospital
1. Study Identification
Unique Protocol Identification Number
NCT01584076
Brief Title
Treatment of Residual Amblyopia With Donepezil
Official Title
Recovery From Amblyopia With Cholinesterase Inhibitors
Study Type
Interventional
2. Study Status
Record Verification Date
June 2021
Overall Recruitment Status
Completed
Study Start Date
August 2012 (Actual)
Primary Completion Date
January 2021 (Actual)
Study Completion Date
January 2021 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
Boston Children's Hospital
4. Oversight
Data Monitoring Committee
Yes
5. Study Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Amblyopia
Keywords
Amblyopia, Donepezil
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 1
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
18 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Donepezil
Arm Type
Experimental
Intervention Type
Drug
Intervention Name(s)
Donepezil
Other Intervention Name(s)
Aricept
Intervention Description
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
Other
Intervention Name(s)
Patching
Intervention Description
Patching: 2 hours of daily patching will also be prescribed for 8 to 17 year olds only.
Primary Outcome Measure Information:
Title
Amblyopia Eye Visual Acuity Improvement
Description
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.
Time Frame
22 weeks after enrollment
Secondary Outcome Measure Information:
Title
Amblyopic Eye Visual Acuity
Description
Analysis of amblyopia eye visual acuity measured at each visit.
Time Frame
4, 8, 12, and 22 weeks after enrollment
Title
Recurrence of Amblyopia after 10 Weeks Off Study Treatment
Description
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.
Time Frame
22 weeks after enrollment
Title
Adverse Events
Description
Analysis of the proportion of subjects reporting adverse events.
Time Frame
4, 8, 12, and 22 weeks after enrollment
Title
Adverse Events Requiring Discontinuation of Study Treatment
Description
Analysis of the proportion of subjects requiring discontinuation of study treatment secondary to adverse events.
Time Frame
4, 8, and 12 weeks after enrollment
Title
Completion of Study Treatment
Description
Analysis of the proportion of subjects completing study treatment.
Time Frame
12 weeks after enrollment
Title
Sound Eye Visual Acuity
Description
Analysis of sound eye visual acuity at 22 weeks to assess any adverse effect on the occluded eye.
Time Frame
22 weeks after enrollment
10. Eligibility
Sex
All
Minimum Age & Unit of Time
8 Years
Accepts Healthy Volunteers
Accepts Healthy Volunteers
Eligibility Criteria
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Carolyn Wu, MD
Organizational Affiliation
Boston Children's Hospital
Official's Role
Principal Investigator
First Name & Middle Initial & Last Name & Degree
David G. Hunter, MD, PhD
Organizational Affiliation
Boston Children's Hospital
Official's Role
Principal Investigator
First Name & Middle Initial & Last Name & Degree
Bharti Gangwani, MD
Organizational Affiliation
Boston Children's Hospital
Official's Role
Principal Investigator
Facility Information:
Facility Name
Boston Children's Hospital
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02115
Country
United States
Facility Name
Boston Children's Hospital at Waltham
City
Waltham
State/Province
Massachusetts
ZIP/Postal Code
02453
Country
United States
Facility Name
Boston Children's Physicians South
City
Weymouth
State/Province
Massachusetts
ZIP/Postal Code
02190
Country
United States
12. IPD Sharing Statement
Plan to Share IPD
No
Citations:
PubMed Identifier
21071629
Citation
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.
Results Reference
background
Learn more about this trial
Treatment of Residual Amblyopia With Donepezil
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