Clinical trial NCT02222207: Regorafenib Eye Drops: Investigation of Efficacy and Safety in Neovascular Age Related Macular Degeneration
Official title: A Combined Phase IIa / IIb Study of the Efficacy, Safety, and Tolerability of Repeated Topical Doses of Regorafenib Eye Drops, in Treatment-naïve Subjects With Neovascular Age Related Macular Degeneration
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE2
Conditions: Macular Degeneration
Keywords: neovascular Age-related Macular Degeneration
Lead sponsor: Bayer
Sponsor class: INDUSTRY
First posted: 2014-08-21
Enrollment: 52
Sex: ALL
Minimum age (years): 50
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: Part A (Phase IIa):

Primary objectives:

The study part A is designed to investigate whether the use of regorafenib eye drops can help patients with neovascular (wet) Age-Related Macular Degeneration (wAMD) to see better after 4 weeks and 12 weeks after inclusion into this study.

Secondary objectives:

The study will also evaluate the safety and tolerability of the regorafenib eye drops.

Part B (Phase IIb):

Primary objectives:

The study part B is designed to investigate:

* how often the regorafenib eye drops need to be given per day
* whether the use of regorafenib eye drops can help patients with neovascular (wet) Age-Related Macular Degeneration (wAMD) to see better after 4 weeks and 12 weeks after inclusion into this study.

Secondary objectives:

The study will also evaluate how the different dosings of regorafenib eye drops affect patients vision, the safety and the tolerability.


Eligibility criteria: Inclusion Criteria:

* Able to read (or, if unable to read due to visual impairment, be read to verbatim by the person administering the informed consent or a family member) and able to understand the informed consent form (ICF)
* Men and women ≥ 50 years of age
* Active primary subfoveal CNV (Choroidal neovascularization) lesions secondary to AMD (Age-related macular degeneration), including juxtafoveal lesions that affect the fovea as evidenced by FA (Fluorescein angiography) in the study eye and reviewed by the central reading center
* The area of CNV must occupy at least 50% of total lesion in the study eye, as determined by FA review at the central reading center
* Evidence of intraretinal and/or subretinal fluid on OCT (Optical coherence tomography)
* Early Treatment Diabetic Retinopathy Study BCVA of 73 to 25 letters (20/40 to 20/320 Snellen equivalent) in the study eye
* Willing, committed, and able to return for all clinic visits and complete all study related procedures
* Women of childbearing potential and men must agree to use adequate contraception when sexually active. This applies since signing of the ICF until one month after the EOS (end of study) visit. The definition of adequate contraception will be based on the judgment of the investigator.

Exclusion Criteria:

* Concurrent disease in the study eye, other than AMD (e.g., corneal diseases and dystrophies, conjunctival diseases, eye lid abnormalities, or any other diseases of the cornea and macula, or optic nerve abnormality) that could compromise visual acuity, likely require medical or surgical intervention during the study period, would limit the potential to gain or lose vision during study treatment, or could otherwise confound interpretation of the results
* Total lesion size (including neovascularization, scar, blood) \> 12 disc areas (30.5 mm2) as assessed by FA
* Only one functional eye, even if that eye is otherwise eligible for the study
* Prior ocular or systemic treatment or surgery for neovascular AMD in the study eye except dietary supplements or vitamins
* Prior treatment with any systemic anti-VEGF (Vascular endothelial growth factor) agent
* Use of systemic or ocular treatment with an investigational drug within 12 weeks prior to start of study treatment
* Any other condition that would require frequent chronic co-administration of other topical eye medications that would interfere with study drug administration (e.g. contact lens)
* Symptoms or conditions consistent with contraindications listed in the current local label for ranibizumab
* Participation in an investigational study within 30 days prior to start of study treatment that involved treatment with any drug (excluding vitamins and minerals) or device
* Lactating women and women of child-bearing potential with either a positive pregnancy test result or no pregnancy test at screening are excluded. Postmenopausal women must be amenorrheic for at least 12 months in order not to be considered of child bearing potential.

Primary outcomes:
measure: Change From Baseline in Best Corrected Visual Acuity (BCVA) as Measured by Early Treatment Diabetic Retinopathy Study (ETDRS) Letter Score at Study Week 4 for Study Part A
timeFrame: Baseline, Week 4
measure: Change From Baseline in BCVA as Measured by ETDRS Letter Score at Study Week 12 for Study Part A
timeFrame: Baseline, Week 12
Secondary outcomes:
measure: Percentage of Participants With Individual Changes in BCVA of Greater Than Equal to (>=) 0 Letters of Vision From Study Week 4 to Week 12 for Study Part A
timeFrame: Week 4, Week 12
measure: Percentage of Participants With a Loss in BCVA of >= 10 Letters From Baseline to Study Week 12 for Study Part A
timeFrame: Baseline, Week 12
Other outcomes:
Locations:
city: Tucson
country: United States
state: Arizona
city: Arcadia
country: United States
state: California
city: Beverly Hills
country: United States
state: California
city: Campbell
country: United States
state: California
city: Costa Mesa
country: United States
state: California
city: Laguna Hills
country: United States
state: California
city: Mountain View
country: United States
state: California
city: Santa Ana
country: United States
state: California
city: Golden
country: United States
state: Colorado
city: Boynton Beach
country: United States
state: Florida
city: Fort Myers
country: United States
state: Florida
city: Miami
country: United States
state: Florida
city: Plantation
country: United States
state: Florida
city: Sarasota
country: United States
state: Florida
city: Stuart
country: United States
state: Florida
city: Winter Haven
country: United States
state: Florida
city: Decatur
country: United States
state: Georgia
city: New Albany
country: United States
state: Indiana
city: Leawood
country: United States
state: Kansas
city: Wichita
country: United States
state: Kansas
city: Louisville
country: United States
state: Kentucky
city: Eunice
country: United States
state: Louisiana
city: Boston
country: United States
state: Massachusetts
city: Grand Rapids
country: United States
state: Michigan
city: Florissant
country: United States
state: Missouri
city: Teaneck
country: United States
state: New Jersey
city: Asheville
country: United States
state: North Carolina
city: West Mifflin
country: United States
state: Pennsylvania
city: Greenville
country: United States
state: South Carolina
city: Rapid City
country: United States
state: South Dakota
city: Austin
country: United States
state: Texas
city: Houston
country: United States
state: Texas
city: McAllen
country: United States
state: Texas
city: San Antonio
country: United States
state: Texas
city: Temple
country: United States
state: Texas
city: The Woodlands
country: United States
state: Texas
city: Charlottesville
country: United States
state: Virginia
city: Strathfield
country: Australia
state: New South Wales
city: Sydney
country: Australia
state: New South Wales
city: Westmead
country: Australia
state: New South Wales
city: Nedlands
country: Australia
state: Western Australia
city: Parramatta
country: Australia
city: Wien
country: Austria
city: Wien
country: Austria
city: Calgary
country: Canada
state: Alberta
city: Edmonton
country: Canada
state: Alberta
city: Vancouver
country: Canada
state: British Columbia
city: Halifax
country: Canada
state: Nova Scotia
city: London
country: Canada
state: Ontario
city: Mississauga
country: Canada
state: Ontario
city: Montreal
country: Canada
state: Quebec
city: Montreal
country: Canada
state: Quebec
city: Sherbrooke
country: Canada
state: Quebec
city: Osorno
country: Chile
state: Los Lagos
city: Metropolitana
country: Chile
state: Santiago
city: Vitacura
country: Chile
state: Santiago
city: Santiago
country: Chile
city: Bogota
country: Colombia
state: Distrito Capital de Bogotá
city: Hradec Kralove
country: Czech Republic
city: Praha 2
country: Czech Republic
city: Praha 4
country: Czech Republic
city: Praha 7
country: Czech Republic
city: Paris
country: France
state: Cedex 12
city: Boredaux
country: France
city: Lyon
country: France
city: Tübingen
country: Germany
state: Baden-Württemberg
city: München
country: Germany
state: Bayern
city: München
country: Germany
state: Bayern
city: Bonn
country: Germany
state: Nordrhein-Westfalen
city: Köln
country: Germany
state: Nordrhein-Westfalen
city: Münster
country: Germany
state: Nordrhein-Westfalen
city: Mainz
country: Germany
state: Rheinland-Pfalz
city: Dresden
country: Germany
state: Sachsen
city: Dresden
country: Germany
state: Sachsen
city: Leipzig
country: Germany
state: Sachsen
city: Berlin
country: Germany
city: Hamburg
country: Germany
city: Hong Kong
country: Hong Kong
city: Kowloon
country: Hong Kong
city: Budapest
country: Hungary
city: Budapest
country: Hungary
city: Budapest
country: Hungary
city: Debrecen
country: Hungary
city: Pecs
country: Hungary
city: Afula
country: Israel
city: Beer Sheva
country: Israel
city: Haifa
country: Israel
city: Haifa
country: Israel
city: Jerusalem
country: Israel
city: Kfar Saba
country: Israel
city: Petah Tikva
country: Israel
city: Ramat Gan
country: Israel
city: Rehovot
country: Israel
city: Tel Aviv
country: Israel
city: Zerifin
country: Israel
city: Udine
country: Italy
state: Friuli-Venezia Giulia
city: Roma
country: Italy
state: Lazio
city: Roma
country: Italy
state: Lazio
city: Milano
country: Italy
state: Lombardia
city: Milano
country: Italy
state: Lombardia
city: Firenze
country: Italy
state: Toscana
city: Padova
country: Italy
state: Veneto
city: Ancona
country: Italy
city: Bologna
country: Italy
city: Milano
country: Italy
city: Sassari
country: Italy
city: Inba-gun
country: Japan
state: Chiba
city: Miyako-gun
country: Japan
state: Fukuoka
city: Sapporo
country: Japan
state: Hokkaido
city: Sapporo
country: Japan
state: Hokkaido
city: Himeji
country: Japan
state: Hyogo
city: Yamato
country: Japan
state: Kanagawa
city: Yokohama
country: Japan
state: Kanagawa
city: Iga
country: Japan
state: Mie
city: Chofu
country: Japan
state: Tokyo
city: Itabashi-ku
country: Japan
state: Tokyo
city: Ota-ku
country: Japan
state: Tokyo
city: Taito-ku
country: Japan
state: Tokyo
city: Fukuoka
country: Japan
city: Kumamoto
country: Japan
city: Nagasaki
country: Japan
city: Nara
country: Japan
city: Osaka
country: Japan
city: Osaka
country: Japan
city: Seongnam-si
country: Korea, Republic of
state: Gyeonggido
city: Busan
country: Korea, Republic of
city: Incheon
country: Korea, Republic of
city: Seoul
country: Korea, Republic of
city: Seoul
country: Korea, Republic of
city: Seoul
country: Korea, Republic of
city: Seoul
country: Korea, Republic of
city: Bratislava
country: Slovakia
city: Ruzomberok
country: Slovakia
city: Trencin
country: Slovakia
city: Zilina
country: Slovakia
city: Zilina
country: Slovakia
city: L'Hospitalet de Llobregat
country: Spain
state: Barcelona
city: San Cugat del Vallès
country: Spain
state: Barcelona
city: Barakaldo
country: Spain
state: Vizcaya
city: Albacete
country: Spain
city: Barcelona
country: Spain
city: Sevilla
country: Spain
city: Sevilla
country: Spain
city: Valencia
country: Spain
city: Valencia
country: Spain
city: Valladolid
country: Spain
city: Bern
country: Switzerland
city: Lausanne
country: Switzerland
city: Zürich
country: Switzerland
Study officials:
affiliation: Bayer
name: Bayer Study Director
role: STUDY_DIRECTOR
References: