Sildenafil for Treatment of Choroidal Ischemia

Columbia University

Status and phase: Active, not recruiting; Phase 2

Conditions: Central Serous Retinopathy; Vitelliform Macular Dystrophy; Retinitis Pigmentosa; Age-related Macular Degeneration; Choroidal Ischemia

Treatments: Drug: Sildenafil; Other: Standard of Care Sildenafil; Diagnostic Test: Ocular Coherence Tomography-Angiography (OCT-A); Other: Visual Acuity (VA)

Study type: Interventional

Funder types: Other

Identifiers

NCT04356716

AAAM7406

Details and patient eligibility

About

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.

Full 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.

Enrollment: 25 estimated patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

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)

Exclusion criteria

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

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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25 participants in 2 patient groups

Standard of Care Sildenafil

Active Comparator group

Description:

Medical record review for participants that are prescribed Sildenafil off-label as part of standard of care treatment for disease.

Treatment:

Other: Standard of Care Sildenafil

Sildenafil

Active Comparator group

Description:

Participants are prescribed sildenafil 40-80 mg daily.

Treatment:

Other: Visual Acuity (VA)

Drug: Sildenafil

Diagnostic Test: Ocular Coherence Tomography-Angiography (OCT-A)