Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
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.
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.
Not provided
Not provided
Not provided
Not provided
| Label | Type | Description | Intervention Names |
|---|---|---|---|
| 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. |
|
| Sildenafil | Active Comparator | Participants are prescribed sildenafil 40-80 mg daily. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| 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. |
| Measure | Description | Time Frame |
|---|---|---|
| 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 |
Not provided
Not provided
Inclusion Criteria:
Exclusion Criteria:
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Name | Affiliation | Role |
|---|---|---|
| Donald Jackson Coleman, MD | Columbia University | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Columbia University Medical Center, Edward Harkness Eye Institute | New York | New York | 10032 | United States |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 23740965 | Background | 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. | |
| 29694963 | 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. |
Not provided
Not provided
Not provided
Not provided
Not provided
| ID | Title | Description |
|---|---|---|
| FG000 | Sildenafil | Participants are prescribed sildenafil 40-80 mg daily. |
| FG001 | Medical Record Review | Medical record review for participants that are prescribed Sildenafil off-label as part of standard of care treatment for disease. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
Not provided
Not provided
Not provided
| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot_SAP | Yes | Yes | No | Study Protocol and Statistical Analysis Plan | Apr 8, 2020 |
Not provided
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.
Not provided
Not provided
Not provided
Not provided
|
|
| Standard of Care Sildenafil | Other | Medical record review of participants that receive Sildenafil as part of standard of care. |
|
|
| Ocular Coherence Tomography-Angiography (OCT-A) | Diagnostic Test | Retinal photographs will be taken at each study visit. |
|
| Visual Acuity (VA) | Other | Visual acuity will be measured with Snellen Eye Chart at each study visit. |
|
| COMPLETED |
|
| NOT COMPLETED |
|
Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | Sildenafil | Participants are prescribed sildenafil 40-80 mg daily. |
| BG001 | Medical Record Review | Medical record review for participants that are prescribed Sildenafil off-label as part of standard of care treatment for disease. |
| BG002 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants | Participants |
| ||||||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| ||||||||||||||||||
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||||
| Race (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||||
| Region of Enrollment | Number | participants |
|
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | 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. | Posted | Mean | Standard Deviation | logMAR | Up to 63 months |
|
|
| |||||||||||||||||||||||||||||||||
| Primary | 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. | Posted | Number | logMAR | 22 months |
|
|
Up to 63 months
The subject in the medical review arm was not seen by research staff. No AE data was collected from this subject.
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Sildenafil | Participants are prescribed sildenafil 40-80 mg daily. | 0 | 21 | 0 | 21 | 0 | 21 |
| EG001 | Medical Record Review | Medical record review for participants that are prescribed Sildenafil off-label as part of standard of care treatment for disease. | 0 | 0 | 0 | 0 | 0 | 0 |
Not provided
Not provided
Not provided
Not provided
Not provided
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Suzanne Daly, RN, BSN | Columbia University | 212 305 9535 | sd2798@cumc.columbia.edu |
| Jun 6, 2025 |
| Prot_SAP_000.pdf |
| ID | Term |
|---|---|
| D057826 | Vitelliform Macular Dystrophy |
| D008268 | Macular Degeneration |
| D056833 | Central Serous Chorioretinopathy |
| D012174 | Retinitis Pigmentosa |
| ID | Term |
|---|---|
| D012162 | Retinal Degeneration |
| D012164 | Retinal Diseases |
| D005128 | Eye Diseases |
| D015785 | Eye Diseases, Hereditary |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D058499 | Retinal Dystrophies |
Not provided
Not provided
| ID | Term |
|---|---|
| D000068677 | Sildenafil Citrate |
| D014792 | Visual Acuity |
| ID | Term |
|---|---|
| D013449 | Sulfonamides |
| D000577 | Amides |
| D009930 | Organic Chemicals |
| D013450 | Sulfones |
| D013457 | Sulfur Compounds |
| D010879 | Piperazines |
| D006573 | Heterocyclic Compounds, 1-Ring |
| D006571 | Heterocyclic Compounds |
| D011687 | Purines |
| D006574 | Heterocyclic Compounds, 2-Ring |
| D000072471 | Heterocyclic Compounds, Fused-Ring |
| D014787 | Vision Tests |
| D003941 | Diagnostic Techniques, Ophthalmological |
| D019937 | Diagnostic Techniques and Procedures |
| D003933 | Diagnosis |
| D009799 | Ocular Physiological Phenomena |
Not provided
Not provided
| >=65 years |
|
| Male |
|
| Not Hispanic or Latino |
|
| Unknown or Not Reported |
|
| Asian |
|
| Native Hawaiian or Other Pacific Islander |
|
| Black or African American |
|
| White |
|
| More than one race |
|
| Unknown or Not Reported |
|
| Title | Denominators | Categories |
|---|
| Right eye |
| |||||
| Left eye |
|