Sildenafil for Treatment of Choroidal Ischemia

Phase 2

Active, not recruiting

• Conditions: Vitelliform Macular Dystrophy; Age-related Macular Degeneration; Retinitis Pigmentosa; Central Serous Retinopathy; Choroidal Ischemia
• Interventions: Other: Standard of Care Sildenafil; Drug: Sildenafil; Diagnostic Test: Ocular Coherence Tomography-Angiography (OCT-A); Other: Visual Acuity (VA)

• Registration Number: NCT04356716
• Lead Sponsor: Columbia University

Brief 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.

Study Timeline

• Study Start: 2014-11-11
• Study First Submitted: 2020-04-20
• Study First Submitted QC: 2020-04-20
• Study First Posted: 2020-04-22
• Status Verified: 2023-10
• Last Update Submitted: 2023-10-19
• Last Update Posted: 2023-10-23
• Primary Completion: 2024-12
• Study Completion: 2024-12

Recruitment & Eligibility

• Status: ACTIVE_NOT_RECRUITING
• Sex: All
• Target Recruitment: 25

Inclusion Criteria

• Diagnosis of retinal and choroidal degenerations (reticular or vitelliform AMD or vitelliform-type subretinal drusen) or hereditary or acquired retinal dystrophies (retinitis pigmentosa or central serous retinopathy)

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Exclusion Criteria

• Diagnosis of heart disease requiring use of nitrates
• Inability to be examined monthly or bi-monthly

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Sildenafil	Ocular Coherence Tomography-Angiography (OCT-A)	Participants are prescribed sildenafil 40-80 mg daily.
Sildenafil	Visual Acuity (VA)	Participants are prescribed sildenafil 40-80 mg daily.
Standard of Care Sildenafil	Standard of Care Sildenafil	Medical record review for participants that are prescribed Sildenafil off-label as part of standard of care treatment for disease.
Sildenafil	Sildenafil	Participants are prescribed sildenafil 40-80 mg daily.

Primary Outcome Measures

Name	Time	Method
Change in Choroidal Perfusion	Up to 5 Years	Patients are evaluated for progression of disease/dystrophy via visual acuity (improved/stable/worsened) and appearance of fluid layer and drusen on OCT testing.

Trial Locations

Locations (1)

Columbia University Medical Center, Edward Harkness Eye Institute; 🇺🇸 New York, New York, United States