NCT04356716

Sildenafil for Treatment of Choroidal Ischemia

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 22 (actual)
• Sponsor: Columbia University · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

The hypothesis of this study is to determine if there is a benefit afforded by the use of systemic Sildenafil to patients with choroidal and retinal degenerations and dystrophies, such as vitelliform degeneration, dry and reticular age-related macular degeneration (AMD) as well as patients with hereditary and acquired retinal dystrophies such as retinitis pigmentosa and central serous retinopathy.

Detailed description

Age-Related Macular Degeneration (AMD) is a sight-threatening visual disturbance that affects the macula in older ages. It is irreversible if the pigment epithelium is lost (dry AMD), but wet AMD can be arrested or delayed with the use of intraocular injections of one of 3 different compounds. All three drugs are injected into the eye in minute doses of usually 0.1 ml. These are usually injected at 4 to 6 week intervals and treatment may be extended for several years. Different patterns of injection times are followed but usually are monthly for 12 or more months, or monthly for 3 months and then extended observation at one to two month intervals unless vision (visual acuity) declines or Optical Coherence Tomography-angiography (OCT-A) shows recurrence of fluid or increase in size or amount of drusen (deposits of lipofuscin) in the retina. Vitelliform macular degeneration is a disorder that causes visual loss due to drusen in the macula, which have been shown to be identical to the deposits seen in macular degeneration. The goal of therapy in this proposal is to use sildenafil to increase choroidal blood flow to treat dry AMD and slow the progression of visual loss in vitelliform and age-related dry AMD as well as other macular, retinal and choroidal degenerations and dystrophies, as well as reduce or eliminate the number of injections required by slowing down transformation of dry AMD to wet AMD in treated patients.

Conditions

• Choroidal Ischemia
• Vitelliform Macular Dystrophy
• Age-related Macular Degeneration
• Central Serous Retinopathy
• Retinitis Pigmentosa

Interventions

Type	Name	Description
DRUG	Sildenafil	Initial Sildenafil dosage will be weight dependent. Participants will start at 40 mg daily (20mg in the morning, 20 mg in the evening) or 60 mg daily (40mg in the morning and 20 mg in the evening). Sildenafil dosage may be increased to up to 80mg daily (20-40mg in the morning and 20-40mg in the evening) based on the response to lower doses. If the participant has not had improvement after initial treatment, the dose may be increased, at the discretion of the study physician.
OTHER	Standard of Care Sildenafil	Medical record review of participants that receive Sildenafil as part of standard of care.
DIAGNOSTIC_TEST	Ocular Coherence Tomography-Angiography (OCT-A)	Retinal photographs will be taken at each study visit.
OTHER	Visual Acuity (VA)	Visual acuity will be measured with Snellen Eye Chart at each study visit.

Timeline

• Start date: 2014-11-11
• Primary completion: 2020-02-18
• Completion: 2020-02-18
• First posted: 2020-04-22
• Last updated: 2025-07-04
• Results posted: 2025-07-04

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study