Sildenafil for Treatment of Choroidal Ischemia

Sponsor

Columbia University (Other)

Overall Status

Recruiting

CT.gov ID

NCT04356716

Collaborator

(none)

Enrollment

Location

Arms

96.7

Anticipated Duration (Months)

0.3

Patients Per Site Per Month

Study Details

Study Description

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.

Condition or Disease	Intervention/Treatment	Phase
Choroidal Ischemia Vitelliform Macular Dystrophy Age-related Macular Degeneration Central Serous Retinopathy Retinitis Pigmentosa	Drug: Sildenafil Other: Standard of Care Sildenafil Diagnostic Test: Ocular Coherence Tomography-Angiography (OCT-A) Other: Visual Acuity (VA)	Phase 2

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 Design

Study Type:

Interventional

Anticipated Enrollment :

25 participants

Allocation:

Non-Randomized

Intervention Model:

Parallel Assignment

Intervention Model Description:

Participants are assigned to the investigational sildenafil arm with a dose of 40-80mg daily, or the records review arm when taking sildenafil off-label.Participants are assigned to the investigational sildenafil arm with a dose of 40-80mg daily, or the records review arm when taking sildenafil off-label.

Masking:

None (Open Label)

Primary Purpose:

Treatment

Official Title:

Sildenafil for Treatment of Choroidal Ischemia

Actual Study Start Date :

Nov 11, 2014

Anticipated Primary Completion Date :

Dec 1, 2022

Anticipated Study Completion Date :

Dec 1, 2022

Arms and Interventions

Arm	Intervention/Treatment
Active Comparator: Standard of Care Sildenafil Medical record review for participants that are prescribed Sildenafil off-label as part of standard of care treatment for disease.	Other: Standard of Care Sildenafil Medical record review of participants that receive Sildenafil as part of standard of care. Other Names: Viagra Revatio
Active Comparator: Sildenafil Participants are prescribed sildenafil 40-80 mg daily.	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 Names: Viagra Revatio 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.

Arm

Intervention/Treatment

Active Comparator: Standard of Care Sildenafil

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

Other: Standard of Care Sildenafil

Medical record review of participants that receive Sildenafil as part of standard of care.

Other Names:

Viagra

Revatio

Active Comparator: Sildenafil

Participants are prescribed sildenafil 40-80 mg daily.

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 Names:

Viagra

Revatio

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.

Outcome Measures

Primary Outcome Measures

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.

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years and Older

Sexes Eligible for Study:

All

Accepts 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)