AmblyopiaPhase 1NCT01584076
Treatment of Residual Amblyopia With Donepezil
A Phase 1 study testing Donepezil and Patching in people with Amblyopia, run by Boston Children's Hospital at 3 sites in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 8 years and olderHealthy volunteers can take part
- Conditions the study is for
- Amblyopia
- What is being tested?
- Donepezil and Patching
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 18 participants
- Where
- 3 sitesUnited States
- Study dates
- Aug 2012to Jan 2021
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 8 years and older
- All sexes
- Healthy volunteers accepted
- Child, Adult, Older adult
Who may be able to join
- 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
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- 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
Who cannot join
- 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
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- 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
Trial locations and contacts
3 sites in 1 country.
United States3 sites
Boston Children's Hospital
Boston, Massachusetts, 02115
Boston Children's Hospital at Waltham
Waltham, Massachusetts, 02453
Boston Children's Physicians South
Weymouth, Massachusetts, 02190
About this study
Official title: Recovery From Amblyopia With Cholinesterase Inhibitors
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
- Being tested
- Donepezil, Patching
- Keywords
- Amblyopia, Donepezil
- Medical subject headings
- Amblyopia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
DonepezilDrug
Also called Aricept
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.
PatchingOther
Patching: 2 hours of daily patching will also be prescribed for 8 to 17 year olds only.
| Study group | Role | What they receive |
|---|---|---|
| Donepezil | Experimental | Not described Receives: Drug: Donepezil; Other: Patching |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 22 weeks after enrollment
Secondary outcomes (6)
Amblyopic Eye Visual Acuity
Analysis of amblyopia eye visual acuity measured at each visit.
Measured: 4, 8, 12, and 22 weeks after enrollment
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.
Measured: 22 weeks after enrollment
Adverse Events
Analysis of the proportion of subjects reporting adverse events.
Measured: 4, 8, 12, and 22 weeks after enrollment
Adverse Events Requiring Discontinuation of Study Treatment
Analysis of the proportion of subjects requiring discontinuation of study treatment secondary to adverse events.
Measured: 4, 8, and 12 weeks after enrollment
Completion of Study Treatment
Analysis of the proportion of subjects completing study treatment.
Measured: 12 weeks after enrollment
Sound Eye Visual Acuity
Analysis of sound eye visual acuity at 22 weeks to assess any adverse effect on the occluded eye.
Measured: 22 weeks after enrollment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Apr 2012 (estimated)
- Study startAug 2012
- Primary completionJan 2021
- Study completionJan 2021
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for AmblyopiaStudies of the same condition, open and under way.
- More trials run by Boston Children's HospitalThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 18 participants (actual)
Sponsor and investigators
- Lead sponsor
- Boston Children's Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Carolyn Wu, Boston Children's Hospital
- Investigators
- Carolyn Wu, MD, Principal investigator, Boston Children's HospitalDavid G. Hunter, MD, PhD, Principal investigator, Boston Children's HospitalBharti Gangwani, MD, Principal investigator, Boston Children's Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01584076
- Sponsor's protocol ID
- IRB-P00002887
- Status last verified
- Jun 2021
- First submitted
- 23 Apr 2012
- Last updated
- 18 Jun 2021
- Results posted
- No
- Data monitoring committee
- Yes
- Individual data sharing
- No
Publications
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.
PubMed 21071629
Study at a glance
From the registry record, last updated by the sponsor on 18 Jun 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Recovery From Amblyopia With Cholinesterase Inhibitors
- ClinicalTrials.gov ID
- NCT01584076
- Acronym / sponsor ID
- IRB-P00002887
- Conditions
- Amblyopia
- Keywords
- Amblyopia, Donepezil
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 18 participants (actual)
- Lead sponsor
- Boston Children's HospitalUniversity, hospital or other
- Study start
- Aug 2012 (actual)
- Primary completion
- Jan 2021 (actual)
- Study completion
- Jan 2021 (actual)
- First posted
- 24 Apr 2012 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Donepezil | Experimental | Drug: Donepezil, Other: Patching |
Who may qualify
- Ages
- 8 years and older
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (10 listed)
- 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)
Key exclusion criteria (15 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| DonepezilAlso: AriceptOral 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. | Drug | Donepezil |
| PatchingPatching: 2 hours of daily patching will also be prescribed for 8 to 17 year olds only. | Other | Donepezil |
Primary outcome
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.
6 secondary outcomes
Amblyopic Eye Visual Acuity
Time frame: 4, 8, 12, and 22 weeks after enrollment
Recurrence of Amblyopia after 10 Weeks Off Study Treatment
Time frame: 22 weeks after enrollment
Adverse Events
Time frame: 4, 8, 12, and 22 weeks after enrollment
Adverse Events Requiring Discontinuation of Study Treatment
Time frame: 4, 8, and 12 weeks after enrollment
Completion of Study Treatment
Time frame: 12 weeks after enrollment
Sound Eye Visual Acuity
Time frame: 22 weeks after enrollment
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Jun 2021.
- Background: PubMed 21071629Morishita 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.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Carolyn Wu, MD principal investigator
Boston Children's Hospital
David G. Hunter, MD, PhD principal investigator
Boston Children's Hospital
Bharti Gangwani, MD principal investigator
Boston Children's Hospital
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Donepezil and Patching in people with Amblyopia, run by Boston Children's Hospital at 3 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 10 inclusion and 15 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 18 Jun 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Amblyopia Eye Visual Acuity Improvement (time frame: 22 weeks after enrollment). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01584076, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 18 Jun 2021.