Clinical Trial: Treatment of Residual Amblyopia With Donepezil

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional

Official Title: Recovery From Amblyopia With Cholinesterase Inhibitors

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 Summary:
Sponsor: Boston Children’s Hospital

Current 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.


Original Primary Outcome: Amblyopic Eye Visual Acuity [ Time Frame: 22 weeks after randomization ]

Study treatment (patching and oral medication) will last for 12 weeks. Primary outcome is treatment group comparison of the amblyopia eye visual acuity measured at 22 weeks after 10 weeks off treatment.


Current Secondary Outcome:

  • Amblyopic Eye Visual Acuity [ Time Frame: 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 [ 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.
  • Adverse Events [ Time Frame: 4, 8, 12, and 22 weeks after enrollment ]
    Analysis of the proportion of subjects reporting adverse events.
  • 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.
  • Completion of Study Treatment [ Time Frame: 12 weeks after enrollment ]
    Analysis of the proportion of subjects completing study treatment.
  • 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.


Original Secondary Outcome:

  • Amblyopia Eye Visual Acuity Improvement [ Time Frame: 4, 8, 12, and 22 weeks after randomization ]
    Treatment group comparison of the proportion of subjects with improvement in amblyopic eye visual acuity of ≥ 10 letters.
  • Recurrence of Amblyopia after 10 Weeks Off Treatment [ Time Frame: 12 and 22 weeks after randomization ]
    Study treatment (patching and oral medication) will be discontinued after 12 weeks. Amblyopic eye visual acuity at 12 weeks and 22 weeks after randomization will be compared. Treatment group comparison of the proportion of subjects with recurrence of amblyopia after 10 weeks off treatment.
  • Sound Eye Visual Acuity [ Time Frame: 22 weeks after randomization ]
    Sound eye visual acuity will be evaluated at 22 weeks to assess any adverse effect on the occluded eye.
  • Adverse Events [ Time Frame: 4, 8, 12, and 22 weeks after randomization ]
    Treatment group comparison of the proportion of subjects reporting adverse events.
  • Adverse Events Requiring Discontinuation of Treatment [ Time Frame: 4, 8, and 12 weeks after randomization ]
    Treatment group comparison of the proportion of subjects requiring discontinuation of treatment secondary to adverse events.
  • Completion of Treatment [ Time Frame: 12 weeks after randomization ]
    Treatment group comparison of the proportion of subjects completing treatment.


Information By: Boston Children’s Hospital

Dates:
Date Received: April 23, 2012
Date Started: August 2012
Date Completion: December 2018
Last Updated: February 7, 2017
Last Verified: February 2017