Sildenafil for Treatment of Choroidal Ischemia

NCT04356716 | Completed Phase 2 Results FDA Drug

• 1 other identifier: AAAM7406
• Study Type: interventional
• Target: 22
• Locations: 1 country
• Sites: 1

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.

Conditions

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

Keywords

Sildenafil; Viagra; Revatio

Outcome Measures

Primary Outcomes (2)

• Change in Visual Acuity (Sildenafil Group)

  Patients are evaluated for progression of disease/dystrophy via visual acuity (improved/stable/worsened) and appearance of fluid layer and drusen on OCT testing.

  Up to 63 months

• Change in Visual Acuity (Medical Record Review Group)

  Patients are evaluated for progression of disease/dystrophy via visual acuity (improved/stable/worsened) and appearance of fluid layer and drusen on OCT testing.

  22 months

Study Arms (2)

Standard of Care Sildenafil

ACTIVE COMPARATOR

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

Sildenafil

ACTIVE COMPARATOR

Participants are prescribed sildenafil 40-80 mg daily.

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

Interventions

Sildenafil DRUG

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.

Also known as: Viagra, Revatio

Sildenafil

Standard of Care Sildenafil OTHER

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

Also known as: Viagra, Revatio

Standard of Care Sildenafil

Ocular Coherence Tomography-Angiography (OCT-A) DIAGNOSTIC_TEST

Retinal photographs will be taken at each study visit.

Sildenafil

Visual Acuity (VA) OTHER

Visual acuity will be measured with Snellen Eye Chart at each study visit.

Sildenafil

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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)

You may not qualify if:

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

Sponsors & Collaborators

• Columbia University: lead

Study Sites (1)

Columbia University Medical Center, Edward Harkness Eye Institute

New York, New York, 10032, United States

Location

Related Publications (2)

• Coleman DJ, Silverman RH, Rondeau MJ, Lloyd HO, Khanifar AA, Chan RV. Age-related macular degeneration: choroidal ischaemia? Br J Ophthalmol. 2013 Aug;97(8):1020-3. doi: 10.1136/bjophthalmol-2013-303143. Epub 2013 Jun 5.

  PMID: 23740965 BACKGROUND

• Coleman DJ, Lee W, Chang S, Silverman RH, Lloyd HO, Daly S, Tsang SH. Treatment of Macular Degeneration with Sildenafil: Results of a Two-Year Trial. Ophthalmologica. 2018;240(1):45-54. doi: 10.1159/000486105. Epub 2018 Apr 25.

  PMID: 29694963 BACKGROUND

MeSH Terms

Conditions

Vitelliform Macular Dystrophy; Macular Degeneration; Central Serous Chorioretinopathy; Retinitis Pigmentosa

Interventions

Sildenafil Citrate; Visual Acuity

Condition Hierarchy (Ancestors)

Retinal Degeneration; Retinal Diseases; Eye Diseases; Eye Diseases, Hereditary; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Retinal Dystrophies

Intervention Hierarchy (Ancestors)

Sulfonamides; Amides; Organic Chemicals; Sulfones; Sulfur Compounds; Piperazines; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Purines; Heterocyclic Compounds, 2-Ring; Heterocyclic Compounds, Fused-Ring; Vision Tests; Diagnostic Techniques, Ophthalmological; Diagnostic Techniques and Procedures; Diagnosis; Ocular Physiological Phenomena

Results Point of Contact

• Title: Suzanne Daly, RN, BSN
• Organization: Columbia University

sd2798@cumc.columbia.edu

212 305 9535

Study Officials

• Donald Jackson Coleman, MD

  Columbia University

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor of Clinical Ophthalmology

Model Details: 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.

Study Record Dates

• First Submitted: April 20, 2020
• First Posted: April 22, 2020
• Study Start: November 11, 2014
• Primary Completion: February 18, 2020
• Study Completion: February 18, 2020
• Last Updated: July 4, 2025
• Results First Posted: July 4, 2025

Record last verified: 2025-07

Locations

US (1)