The Efficacy of Oral Everolimus in Patients With Neovascular Age-related Macular Degeneration

Novartis

Status and phase: Terminated; Phase 2

Conditions: Age-related Macular Degeneration; Choroidal Neo-Vascular Age-onset Macular Degeneration

Treatments: Drug: Everolimus; Drug: Ranibizumab

Study type: Interventional

Funder types: Industry

Identifiers

NCT00857259

CRAD001A2203

EudraCT number: 2008-003550-15

Details and patient eligibility

About

The study will assess the safety and efficacy of Everolimus (RAD001) alone or in combination with Lucentis in patients with neo-vascular age related macular degeneration (AMD)

Enrollment: 16 patients

Sex: All

Ages: 50+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Patients with neovascular Age-Related Macular Degeneration (AMD)
• Best corrected visual acuity ( BCVA) of 20/40 or worse in study eye
• Patients with predominantly classic, minimally classic, or occult choroidal neovascularization in the macula of one eye (the study eye) who have had an inadequate response to VEGF inhibitors in the study eye. Inadequate response is defined as a gain of less than one line of visual acuity and persistent macular edema (central sub-fiel thickness ≥ 300 μm as measured by Optical Coherence Tomography (OCT) despite a minimum of 3 treatments with Lucentis or Avastin

Exclusion criteria

• Any concurrent ocular condition in the study eye that may result in substantial change in vision during the study
• Uncontrolled medical conditions such as cancer, angina, diabetes, viral or fungal infections, impaired lung function, history of stroke
• Patients who have macular edema in the study eye that, in the judgment of the investigator, is unlikely to respond to treatment. Examples of features that may guide the investigator's judgment about unresponsiveness are large regions of geographic atrophy, retinal angiomatous proliferation, or large regions of sub-retinal fibrosis. The presence of one of these features excludes a patient only if the investigator judges the study eye to have irreversible macular edema.
• active bacterial, fungal or viral infections at the time of enrollment, e.g. hepatitis B or C infection. Patients with risk factors for hepatitis B should be tested for hepatitis B viral load and serological markers at screening (a positive HBV-DNA, HBsAg). Patients with risk factors for hepatitis C should be tested using HCVRNA-PCR at screening. A clinical history of hepatitis B or hepatitis C will exclude the patient from the study.

Other protocol-defined inclusion/exclusion criteria may apply

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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16 participants in 3 patient groups

Everolimus 5 mg

Experimental group

Description:

5 mg orally once daily plus sham ocular injection on Day 1 (Baseline) until Day 28

Treatment:

Drug: Everolimus

Ranibizumab 0.5 mg

Active Comparator group

Description:

Ranibizumab intra-vitreal therapy (IVT) 0.5 mg on Day 1 (baseline)

Treatment:

Drug: Ranibizumab

Oral Everolimus (5mg) and Ranibizumab (0.5mg)

Active Comparator group

Description:

Everolimus orally 5 mg once daily plus Ranibizumab Intra-vitreal therapy (IVT) 0.5 mg on day 1 (baseline)

Treatment:

Drug: Ranibizumab

Drug: Everolimus