Von Hippel-Lindau Syndrome
Conditions
Keywords
Intravitreal Injection, Retinal Capillary Hemangioma
Brief summary
Background: People with Von-Hippel-Lindau (VHL) disease may experience significant vision loss as a result of retinal capillary hemangiomas (RCH), the most common and often earliest manifestation of VHL. Objective: To investigate the safety and possible efficacy of combination investigational treatment with serial intravitreal injections of E10030, a PDGF-B antagonist, and ranibizumab, a VEGF-A antagonist, in participants with severe ocular VHL disease. Design: Three participants with severe ocular VHL disease will receive the combination investigational treatment in one eye and will be followed for 104 weeks. Primary Outcome: The safety of the combination investigational treatment, assessed by tabulation of adverse events reported through Week 52.
Detailed description
Objective: Von Hippel-Lindau (VHL) disease is an autosomal dominant heritable disorder in which multiple benign and malignant neoplasms and cysts of specific histopathologies develop in the kidney, adrenal gland, pancreas, brain, spinal cord, eye, inner ear, epididymis and broad ligament. The disease affects about 7,000 individuals in the United States. Retinal capillary hemangiomas (RCH) are the most common and often the earliest manifestation of VHL disease and may lead to significant vision loss. In some such eyes, inexorable progression of RCH leads to blindness and phthisis bulbi despite aggressive treatment. Levels of vascular endothelial growth factor (VEGF), a potent mediator of angiogenesis and vascular permeability, have been shown to be elevated in multiple cell types deficient in the VHL protein (pVHL). Platelet-derived growth factor (PDGF), which has an important role in stabilization of immature new vessels during angiogenesis, is upregulated in pVHL-defective cell lines and expressed in other pVHL-defective tumors. Anti-VEGF therapy alone had no beneficial effect on ocular VHL disease in two previous phase 1 studies. The objective of this study is to investigate the safety and possible efficacy of combination investigational treatment with serial intravitreal injections of E10030, a PDGF-B antagonist, and ranibizumab, a VEGF-A antagonist, in participants with severe ocular VHL disease. Study Population: Three participants with severe ocular VHL disease will receive the combination investigational treatment in one eye and will be followed for 104 weeks. Design: In this phase I/II, single-center, prospective, open label, non-randomized, uncontrolled, single group trial, one eye of eligible participants will be treated with investigational products, E10030, a PDGF-B antagonist, and ranibizumab, a VEGF-A antagonist. Participants will receive combination investigational treatment consisting of intravitreal injections of E10030 (1.5 mg in 0.05 mL) and ranibizumab (0.5 mg in 0.05 mL) every four weeks from baseline through Week 16 (totaling five treatments) and then every eight weeks through Week 48 (totaling nine treatments from baseline). All participants will be followed for 104 weeks. Outcome Measures: The primary outcome for the study will be safety of the combination investigational treatment, assessed by tabulation of adverse events reported through Week 52. Secondary outcomes will include tabulation of adverse events at Week 104, and the following measures in the study eye at Week 52 and 104: the proportion of participants experiencing reduction in size of at least one RCH in the absence of other ablative treatment (assessed by fundus photography and fluorescein angiography (FA)); the proportion of participants experiencing moderate vision loss (defined as a loss of greater than or equal to 15 letters from baseline on Electronic Visual Acuity (EVA) testing); mean change in visual acuity; change in size of RCH (measured by fundus photography and FA); change in exudation (measured by fundus photography, optical coherence tomography (OCT) and FA); change in epiretinal proliferation, fibrosis or retinal traction (assessed by OCT and fundus photography); proportion of participants undergoing ablative treatment of RCH or ocular surgery; proportion of participants with successful ablative treatment of RCH; and the proportion of participants with appearance of one or more new RCH.
Interventions
DRUGRanibizumab
Intravitreal injections of the commercially-available 10 mg/mL formulation of ranibizumab. Ranibizumab is formulated as a sterile solution (pH 5.5) with histidine, trehalose and polysorbate 20. The vial contains no preservative. Each vial contains 0.5 mL of 10 mg/mL ranibizumab aqueous solution. The intravitreal injection volume of ranibizumab is 50 microliter, which correlates to 0.5 mg of dry ranibizumab.
DRUGE10030
E10030 is not a commercially available drug product, and will be provided by Ophthotech Corp. The drug product is provided as a sterile aqueous solution of E10030 at a concentration of 30 mg (oligo weight)/mL. The solution contains monobasic sodium phosphate monohydrate and dibasic sodium phosphate heptahydrate as buffering agents as well as sodium chloride as a tonicity adjuster. The intravitreal injection volume of E10030 is 50 microliter, which correlates to 1.5 mg of dry E10030.
Sponsors
National Eye Institute (NEI)
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Participant Eligibility Criteria The participant must meet all of the eligibility criteria and none of the
Exclusion criteria
below. INCLUSION CRITERIA: 1. Participant must understand and sign the informed consent. 2. Participant must be 18 years of age or older. 3. Participant must have a diagnosis of VHL disease. In accordance with established criteria for diagnosis, any one of the following will be considered sufficient evidence that VHL disease is present: * A family history of VHL disease plus one or more of the following lesions: RCH, spinal or cerebellar hemangioblastoma, pheochromocytoma, multiple pancreatic cysts, epididymal or broad ligament cystadenomas, multiple renal cysts or renal cell carcinoma before age 60 years. * Presence of two or more hemangioblastomas of the retina or brain or a single hemangioblastoma in association with a visceral manifestation such as kidney or pancreatic cysts; renal cell carcinoma; adrenal or extra-adrenal pheochromocytomas; endolymphatic sac tumors; papillary cystadenomas of the epididymis or broad ligament; or neuroendocrine tumors of the pancreas. * Presence of a known disease-causing germline mutation in the VHL gene. 4. Any female participant of childbearing potential must not be pregnant or breast-feeding, must have a negative pregnancy test at screening and must be willing to undergo pregnancy testing immediately prior to each treatment. 5. Any female participant of childbearing potential and any male participant able to father children must have (or have a partner who has) had a hysterectomy or vasectomy, be completely abstinent from intercourse or must agree to practice two effective methods of contraception throughout the course of the study and for at least two months following the last administration of combination investigational treatment. Acceptable methods of contraception include: * hormonal contraception (i.e., birth control pills, injected hormones, dermal patch or vaginal ring), * intrauterine device, * barrier methods (diaphragm or condom) with spermicide, or * surgical sterilization (hysterectomy, tubal ligation or vasectomy).
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Tabulation of Adverse Events | From Baseline to Week 52 | The total number of adverse events through Week 52. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| The Proportion of Participants Experiencing Reduction in Size of at Least One Retinal Capillary Hemangioma (RCH), in the Absence of Other Ablative Treatment (Assessed by Fundus Photography and Fluorescein Angiography (FA)) | From Baseline to Week 52 | The proportion of participants experiencing a reduction in size of at least one RCH in the study eye, in the absence of other ablative treatment as assessed by fundus photography and fluorescein angiography (FA), between Baseline and Week 52. |
| The Proportion of Participants Experiencing Reduction in Size of at Least One RCH, in the Absence of Other Ablative Treatment (Assessed by Fundus Photography and Fluorescein Angiography [FA]) | From Baseline to Week 104 | The proportion of participants experiencing a reduction in size of at least one RCH in the study eye, in the absence of other ablative treatment as assessed by fundus photography and fluorescein angiography (FA), between Baseline and Week 104. |
| Proportion of Participants Undergoing Ablative Treatment of RCH or Ocular Surgery | From Baseline to Week 52 | The proportion of participants undergoing ablative treatment of RCH or ocular surgery in the study eye between Baseline and Week 52. |
| Proportion of Participants With Successful Ablative Treatment of RCH | From Baseline to Week 52 | The proportion of participants with successful ablative treatment of RCH in the study eye between Baseline and Week 52. |
| Mean Change in Visual Acuity | Baseline and Week 52 | Mean change in visual acuity in the study eye from Baseline as compared to Week 52 as measured using the Electronic Early Treatment of Diabetic Retinopathy Study (ETDRS) Visual Acuity (EVA) Testing protocol. Acuity is measured as letters read using an electronic ETDRS program. |
| Tabulation of Adverse Events | From Baseline to Week 104 | The total number of adverse events, excluding natural progression of disease events, through Week 104. |
| Change in Size of RCH (Measured by Fundus Photography and FA) | From Baseline to Week 52 | Number of participants who experienced increased, decreased, or mixed change in the size of RCH in the study eye between Baseline and Week 52 (measured by fundus photography and FA). |
| Change in Exudation (Measured by Fundus Photography, Optical Coherence Tomography (OCT) and FA) | From Baseline to Week 52 | Number of participants who experienced increased, decreased, or mixed change in exudation in the study eye between Baseline and Week 52 (measured by fundus photography, optical coherence tomography \[OCT\] and FA). |
| Change in Exudation (Measured by Fundus Photography, Optical Coherence Tomography [OCT] and FA) | From Baseline to Week 104 | Number of participants who experienced increased, decreased, or mixed change in exudation in the study eye between Baseline and Week 104 (measured by fundus photography, optical coherence tomography \[OCT\] and FA). |
| Change in Epiretinal Proliferation, Fibrosis or Retinal Traction (Assessed by OCT and Fundus Photography) | From Baseline to Week 52 | Number of participants who experienced increased, decreased, or mixed change in epiretinal proliferation, fibrosis or retinal traction in the study eye between Baseline and Week 52 (assessed by OCT and fundus photography). |
| Proportion of Participants With Appearance of One or More New RCH | From Baseline to Week 52 | The proportion of participants with appearance of one or more new RCH in the study eye between Baseline and Week 52. |
| The Proportion of Participants Experiencing Moderate Vision Loss (Defined as a Loss of Greater Than or Equal to 15 Letters From Baseline on Electronic Visual Acuity [EVA] Testing) | From Baseline to Week 52 | The proportion of participants experiencing moderate vision loss in the study eye (defined as a loss of greater than or equal to 15 letters from baseline on Electronic Visual Acuity \[EVA\] testing) between Baseline and Week 52. |
Countries
United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| E10030 and Ranibizumab Intravitreal injections of E10030 and Ranibizumab | 3 |
| Total | 3 |
Baseline characteristics
| Characteristic | E10030 and Ranibizumab |
|---|---|
| Age, Categorical <=18 years | 0 Participants |
| Age, Categorical >=65 years | 0 Participants |
| Age, Categorical Between 18 and 65 years | 3 Participants |
| Age, Continuous | 44.7 years |
| Ethnicity (NIH/OMB) Hispanic or Latino | 0 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 3 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 0 Participants |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants |
| Race (NIH/OMB) Asian | 0 Participants |
| Race (NIH/OMB) Black or African American | 0 Participants |
| Race (NIH/OMB) More than one race | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants |
| Race (NIH/OMB) White | 3 Participants |
| Sex: Female, Male Female | 1 Participants |
| Sex: Female, Male Male | 2 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 0 / 3 |
| other Total, other adverse events | 3 / 3 |
| serious Total, serious adverse events | 2 / 3 |
Outcome results
Primary
Tabulation of Adverse Events
The total number of adverse events through Week 52.
Time frame: From Baseline to Week 52
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| E10030 and Ranibizumab | Tabulation of Adverse Events | 13 adverse events |
Secondary
Change in Epiretinal Proliferation, Fibrosis or Retinal Traction (Assessed by OCT and Fundus Photography)
Number of participants who experienced increased, decreased, or mixed change in epiretinal proliferation, fibrosis or retinal traction in the study eye between Baseline and Week 104 (assessed by OCT and fundus photography).
Time frame: From Baseline to Week 104
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| E10030 and Ranibizumab | Change in Epiretinal Proliferation, Fibrosis or Retinal Traction (Assessed by OCT and Fundus Photography) | 1 Participants |
| Visual Acuity at Week 52 | Change in Epiretinal Proliferation, Fibrosis or Retinal Traction (Assessed by OCT and Fundus Photography) | 0 Participants |
| Mean Change in Visual Acuity at Week 52 From Baseline | Change in Epiretinal Proliferation, Fibrosis or Retinal Traction (Assessed by OCT and Fundus Photography) | 0 Participants |
| No Change in Size of RCH | Change in Epiretinal Proliferation, Fibrosis or Retinal Traction (Assessed by OCT and Fundus Photography) | 2 Participants |
Secondary
Change in Epiretinal Proliferation, Fibrosis or Retinal Traction (Assessed by OCT and Fundus Photography)
Number of participants who experienced increased, decreased, or mixed change in epiretinal proliferation, fibrosis or retinal traction in the study eye between Baseline and Week 52 (assessed by OCT and fundus photography).
Time frame: From Baseline to Week 52
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| E10030 and Ranibizumab | Change in Epiretinal Proliferation, Fibrosis or Retinal Traction (Assessed by OCT and Fundus Photography) | 1 Participants |
| Visual Acuity at Week 52 | Change in Epiretinal Proliferation, Fibrosis or Retinal Traction (Assessed by OCT and Fundus Photography) | 0 Participants |
| Mean Change in Visual Acuity at Week 52 From Baseline | Change in Epiretinal Proliferation, Fibrosis or Retinal Traction (Assessed by OCT and Fundus Photography) | 0 Participants |
| No Change in Size of RCH | Change in Epiretinal Proliferation, Fibrosis or Retinal Traction (Assessed by OCT and Fundus Photography) | 2 Participants |
Secondary
Change in Exudation (Measured by Fundus Photography, Optical Coherence Tomography (OCT) and FA)
Number of participants who experienced increased, decreased, or mixed change in exudation in the study eye between Baseline and Week 52 (measured by fundus photography, optical coherence tomography \[OCT\] and FA).
Time frame: From Baseline to Week 52
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| E10030 and Ranibizumab | Change in Exudation (Measured by Fundus Photography, Optical Coherence Tomography (OCT) and FA) | 1 Participants |
| Visual Acuity at Week 52 | Change in Exudation (Measured by Fundus Photography, Optical Coherence Tomography (OCT) and FA) | 0 Participants |
| Mean Change in Visual Acuity at Week 52 From Baseline | Change in Exudation (Measured by Fundus Photography, Optical Coherence Tomography (OCT) and FA) | 1 Participants |
| No Change in Size of RCH | Change in Exudation (Measured by Fundus Photography, Optical Coherence Tomography (OCT) and FA) | 1 Participants |
Secondary
Change in Exudation (Measured by Fundus Photography, Optical Coherence Tomography [OCT] and FA)
Number of participants who experienced increased, decreased, or mixed change in exudation in the study eye between Baseline and Week 104 (measured by fundus photography, optical coherence tomography \[OCT\] and FA).
Time frame: From Baseline to Week 104
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| E10030 and Ranibizumab | Change in Exudation (Measured by Fundus Photography, Optical Coherence Tomography [OCT] and FA) | 1 Participants |
| Visual Acuity at Week 52 | Change in Exudation (Measured by Fundus Photography, Optical Coherence Tomography [OCT] and FA) | 0 Participants |
| Mean Change in Visual Acuity at Week 52 From Baseline | Change in Exudation (Measured by Fundus Photography, Optical Coherence Tomography [OCT] and FA) | 1 Participants |
| No Change in Size of RCH | Change in Exudation (Measured by Fundus Photography, Optical Coherence Tomography [OCT] and FA) | 1 Participants |
Secondary
Change in Size of RCH (Measured by Fundus Photography and FA)
Number of participants who experienced increased, decreased, or mixed change in the size of RCH in the study eye between Baseline and Week 52 (measured by fundus photography and FA).
Time frame: From Baseline to Week 52
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| E10030 and Ranibizumab | Change in Size of RCH (Measured by Fundus Photography and FA) | 1 Participants |
| Visual Acuity at Week 52 | Change in Size of RCH (Measured by Fundus Photography and FA) | 0 Participants |
| Mean Change in Visual Acuity at Week 52 From Baseline | Change in Size of RCH (Measured by Fundus Photography and FA) | 0 Participants |
| No Change in Size of RCH | Change in Size of RCH (Measured by Fundus Photography and FA) | 2 Participants |
Secondary
Change in Size of RCH (Measured by Fundus Photography and FA)
Number of participants who experienced increased, decreased, or mixed change in the size of RCH in the study eye between Baseline and Week 104 (measured by fundus photography and FA).
Time frame: From Baseline to Week 104
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| E10030 and Ranibizumab | Change in Size of RCH (Measured by Fundus Photography and FA) | 1 Participants |
| Visual Acuity at Week 52 | Change in Size of RCH (Measured by Fundus Photography and FA) | 0 Participants |
| Mean Change in Visual Acuity at Week 52 From Baseline | Change in Size of RCH (Measured by Fundus Photography and FA) | 0 Participants |
| No Change in Size of RCH | Change in Size of RCH (Measured by Fundus Photography and FA) | 2 Participants |
Secondary
Mean Change in Visual Acuity
Mean change in visual acuity in the study eye from Baseline as compared to Week 52 as measured using the Electronic Early Treatment of Diabetic Retinopathy Study (ETDRS) Visual Acuity (EVA) Testing protocol. Acuity is measured as letters read using an electronic ETDRS program.
Time frame: Baseline and Week 52
Population: Mean visual acuity values are presented for Baseline and Week 52 and the mean change at Week 52 from Baseline for the study eye.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| E10030 and Ranibizumab | Mean Change in Visual Acuity | 64.0 Letters Read | Standard Deviation 26.3 |
| Visual Acuity at Week 52 | Mean Change in Visual Acuity | 59.0 Letters Read | Standard Deviation 21.3 |
| Mean Change in Visual Acuity at Week 52 From Baseline | Mean Change in Visual Acuity | -5.0 Letters Read | Standard Deviation 7 |
Secondary
Mean Change in Visual Acuity
Mean change in visual acuity in the study eye from Baseline as compared to Week 104 as measured using the Electronic Early Treatment of Diabetic Retinopathy Study (ETDRS) Visual Acuity (EVA) Testing protocol. Acuity is measured as letters read using an electronic ETDRS program.
Time frame: Baseline and Week 104
Population: Mean visual acuity values are presented for Baseline and Week 104 and the mean change at Week 104 from Baseline for the study eye.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| E10030 and Ranibizumab | Mean Change in Visual Acuity | 64.0 Letters Read | Standard Deviation 26.3 |
| Visual Acuity at Week 52 | Mean Change in Visual Acuity | 45.0 Letters Read | Standard Deviation 40.1 |
| Mean Change in Visual Acuity at Week 52 From Baseline | Mean Change in Visual Acuity | -19.0 Letters Read | Standard Deviation 14.1 |
Secondary
Proportion of Participants Undergoing Ablative Treatment of RCH or Ocular Surgery
The proportion of participants undergoing ablative treatment of RCH or ocular surgery in the study eye between Baseline and Week 52.
Time frame: From Baseline to Week 52
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| E10030 and Ranibizumab | Proportion of Participants Undergoing Ablative Treatment of RCH or Ocular Surgery | 0 Participants |
Secondary
Proportion of Participants Undergoing Ablative Treatment of RCH or Ocular Surgery
The proportion of participants undergoing ablative treatment of RCH or ocular surgery in the study eye between Baseline and Week 104.
Time frame: From Baseline to Week 104
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| E10030 and Ranibizumab | Proportion of Participants Undergoing Ablative Treatment of RCH or Ocular Surgery | 0 Participants |
Secondary
Proportion of Participants With Appearance of One or More New RCH
The proportion of participants with appearance of one or more new RCH in the study eye between Baseline and Week 104.
Time frame: From Baseline to Week 104
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| E10030 and Ranibizumab | Proportion of Participants With Appearance of One or More New RCH | 1 Participants |
Secondary
Proportion of Participants With Appearance of One or More New RCH
The proportion of participants with appearance of one or more new RCH in the study eye between Baseline and Week 52.
Time frame: From Baseline to Week 52
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| E10030 and Ranibizumab | Proportion of Participants With Appearance of One or More New RCH | 0 Participants |
Secondary
Proportion of Participants With Successful Ablative Treatment of RCH
The proportion of participants with successful ablative treatment of RCH in the study eye between Baseline and Week 104.
Time frame: From Baseline to Week 104
Population: No participants were analyzed for this outcome because none of the participants underwent ablative treatment of RCH. Therefore, no data was collected for this outcome.
Secondary
Proportion of Participants With Successful Ablative Treatment of RCH
The proportion of participants with successful ablative treatment of RCH in the study eye between Baseline and Week 52.
Time frame: From Baseline to Week 52
Population: No participants were analyzed for this outcome because none of the participants underwent ablative treatment of RCH. Therefore, no data was collected for this outcome.
Secondary
Tabulation of Adverse Events
The total number of adverse events, excluding natural progression of disease events, through Week 104.
Time frame: From Baseline to Week 104
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| E10030 and Ranibizumab | Tabulation of Adverse Events | 27 adverse events |
Secondary
The Proportion of Participants Experiencing Moderate Vision Loss (Defined as a Loss of Greater Than or Equal to 15 Letters From Baseline on Electronic Visual Acuity [EVA] Testing)
The proportion of participants experiencing moderate vision loss in the study eye (defined as a loss of greater than or equal to 15 letters from baseline on Electronic Visual Acuity \[EVA\] testing) between Baseline and Week 52.
Time frame: From Baseline to Week 52
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| E10030 and Ranibizumab | The Proportion of Participants Experiencing Moderate Vision Loss (Defined as a Loss of Greater Than or Equal to 15 Letters From Baseline on Electronic Visual Acuity [EVA] Testing) | 0 Participants |
Secondary
The Proportion of Participants Experiencing Moderate Vision Loss (Defined as a Loss of Greater Than or Equal to 15 Letters From Baseline on Electronic Visual Acuity [EVA] Testing)
The proportion of participants experiencing moderate vision loss in the study eye (defined as a loss of greater than or equal to 15 letters from baseline on Electronic Visual Acuity \[EVA\] testing) between Baseline and Week 104.
Time frame: From Baseline to Week 104
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| E10030 and Ranibizumab | The Proportion of Participants Experiencing Moderate Vision Loss (Defined as a Loss of Greater Than or Equal to 15 Letters From Baseline on Electronic Visual Acuity [EVA] Testing) | 2 Participants |
Secondary
The Proportion of Participants Experiencing Reduction in Size of at Least One RCH, in the Absence of Other Ablative Treatment (Assessed by Fundus Photography and Fluorescein Angiography [FA])
The proportion of participants experiencing a reduction in size of at least one RCH in the study eye, in the absence of other ablative treatment as assessed by fundus photography and fluorescein angiography (FA), between Baseline and Week 104.
Time frame: From Baseline to Week 104
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| E10030 and Ranibizumab | The Proportion of Participants Experiencing Reduction in Size of at Least One RCH, in the Absence of Other Ablative Treatment (Assessed by Fundus Photography and Fluorescein Angiography [FA]) | 0 Participants |
Secondary
The Proportion of Participants Experiencing Reduction in Size of at Least One Retinal Capillary Hemangioma (RCH), in the Absence of Other Ablative Treatment (Assessed by Fundus Photography and Fluorescein Angiography (FA))
The proportion of participants experiencing a reduction in size of at least one RCH in the study eye, in the absence of other ablative treatment as assessed by fundus photography and fluorescein angiography (FA), between Baseline and Week 52.
Time frame: From Baseline to Week 52
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| E10030 and Ranibizumab | The Proportion of Participants Experiencing Reduction in Size of at Least One Retinal Capillary Hemangioma (RCH), in the Absence of Other Ablative Treatment (Assessed by Fundus Photography and Fluorescein Angiography (FA)) | 0 Participants |