NCT01362348

12 Week Patient Study in Neovascular Age-related Macular Degeneration (AMD)

Terminated Phase 2 Results posted Last updated 21 September 2017

What this trial tests

Phase 2 trial testing pazopanib eye drops in Macular Degeneration in 19 participants. Terminated before completion.

Timeline

7 July 2011

Primary endpoint
16 April 2012

16 April 2012

Quick facts

Lead sponsor	GlaxoSmithKline
Phase	Phase 2
Status	Terminated
Study type	INTERVENTIONAL
Allocation	na
Design	single group
Masking	none
Primary purpose	treatment
Enrollment	19
Start date	7 July 2011
Primary completion	16 April 2012
Estimated completion	16 April 2012
Sites	15 locations across France, United States, Germany

Drugs / interventions tested

• pazopanib eye drops — full drug profile →

Conditions studied

• Macular Degeneration — all drugs for Macular Degeneration →

Sponsor

GlaxoSmithKline — full company profile →

Who can join

50 and older, any sex, with Macular Degeneration. Patients with the condition only — healthy volunteers not accepted.

Results — posted to ClinicalTrials.gov

Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.

Change From Baseline in Central Retinal Lesion Thickness (CRT) as Measured by Optical Coherence Tomography (OCT) at Day 29 Primary · Baseline (Week 0) and Day 29

CRT was the distance between the inner limiting membrane of the retina and the inner border of the retinal pigment epithelium/choriocapillaris band, inclusive of sub retinal fluid, measured in the central 1 millimeter (mm) of the Cube scan. OCT assessments were performed using SPECTRALIS spectral domain OCT. Images were evaluated by investigator for safety monitoring, and by a central reading center for eligibility determination and pharmacodynamics (PD) effects. Observed case (OC) data set was used for analysis in this analysis dataset, a missing assessment at any scheduled time point was con

Group	Value	95% CI
Pazopanib 10 mg/mL QID	37.91	± 89.692

Change From Baseline in Best Correct Visual Acuity (BCVA) as Measured by the Number of Letters Determined by Electronic Early Treatment Diabetic Retinopathy [ETDRS] Study Visual Acuity (EVA) at Day 29 Primary · Baseline (Day -3 to -1) and Day 29

BCVA was measured in the study eye using the EVA chart starting at a test distance of 4 meters. The BCVA score is the number of letters read correctly by the participant. A decrease in the BCVA score indicates a worsening of vision while higher scores indicates improvement of VA. Baseline was defined as the assessments performed between Day -3 to -1. Change from Baseline was calculated by subtracting the Baseline value from the individual post-randomization value at Day 29.

Group	Value	95% CI
Pazopanib 10 mg/mL QID	0.07	± 9.973

Change From Baseline in Central Retinal Lesion Thickness (CRLT) Over Time Secondary · Baseline (Week -3 to -1) Up to Follow-up (Day 102)

CRLT was the manual measurement of the distance between the inner limiting membrane of the retina and the inner border of the choriocapillaris, inclusive of subretinal or sub-retinal pigment epithelium fluid collections and of the thickness of any observable choroidal neovascular membrane or scar tissue, evaluated in the central 1 mm of the Cube scan. OCT assessments were performed using SPECTRALIS spectral domain OCT. Baseline was defined as the assessments performed between Day -3 to -1. Change from Baseline was calculated by subtracting the Baseline value from the individual post-randomizat

CRLT at Week 1

Group	Value	95% CI
Pazopanib 10 mg/mL QID	-3.97	± 93.420

CRLT at Week 2

Group	Value	95% CI
Pazopanib 10 mg/mL QID	-6.39	± 93.420

CRLT at Week 3

Group	Value	95% CI
Pazopanib 10 mg/mL QID	15.15	± 86.646

CRLT at Week 4

Group	Value	95% CI
Pazopanib 10 mg/mL QID	31.72	± 88.289

CRLT at Week 6

Group	Value	95% CI
Pazopanib 10 mg/mL QID	51.22	± 84.679

CRLT at Week 8

Group	Value	95% CI
Pazopanib 10 mg/mL QID	15.48	± 82.304

CRLT at Week 12

Group	Value	95% CI
Pazopanib 10 mg/mL QID	-1.57	± 75.216

CRLT at Follow-up

Group	Value	95% CI
Pazopanib 10 mg/mL QID	-8.42	± 91.346

Change From Baseline in Intraretinal (IR) or Subretinal (SR) Fluid Thickness, Intraretinal Cysts or Serous Retinal Pigment Epithelial Detachment (PED Thickness) Over Time Secondary · Baseline (Week -3 to -1) Up to Follow-up (Day 102)

OCT was used for the determination of retinal morphology changes in the study eye which included assessments of SR fluid (an exudate between the retina and choroid from various sources including the vitreous cavity, subarachnoid space, or abnormal vessels) and PED (retinal pigment epithelium separates from the underlying Bruch's membrane due to the presence of blood, serous exudate, drusen, or a neovascular membrane). Baseline was defined as the assessments performed between Day -3 to -1. Change from Baseline was calculated by subtracting the Baseline value from the individual post-randomizati

SR fluid thickness at Week 1

Group	Value	95% CI
Pazopanib 10 mg/mL QID	-34.79	± 70.781

SR fluid thickness at Week 2

Group	Value	95% CI
Pazopanib 10 mg/mL QID	-29.32	± 71.654

SR fluid thickness at Week 3

Group	Value	95% CI
Pazopanib 10 mg/mL QID	-0.22	± 67.074

SR fluid thickness at Week 4

Group	Value	95% CI
Pazopanib 10 mg/mL QID	5.69	± 67.938

SR fluid thickness at Week 6

Group	Value	95% CI
Pazopanib 10 mg/mL QID	28.05	± 67.192

SR fluid thickness at Week 8

Group	Value	95% CI
Pazopanib 10 mg/mL QID	35.25	± 64.689

SR fluid thickness at Week 12

Group	Value	95% CI
Pazopanib 10 mg/mL QID	1.72	± 57.734

SR fluid thickness at Follow-up

Group	Value	95% CI
Pazopanib 10 mg/mL QID	8.43	± 70.329

Change From Baseline in BCVA Over Time Secondary · Baseline (Week -3 to -1) Up to Follow-up (Day 102)

BCVA was measured in the study eye using the EVA chart starting at a test distance of 4 meters. The BCVA score is the number of letters read correctly by the participant. A decrease in the BCVA score indicates a worsening of vision while higher scores indicates improvement of VA. Baseline was defined as the assessments performed between Day -3 to -1. Change from Baseline was calculated by subtracting the Baseline value from the individual post-randomization value at Day 29.

Week 1

Group	Value	95% CI
Pazopanib 10 mg/mL QID	0.26	± 11.015

Week 2

Group	Value	95% CI
Pazopanib 10 mg/mL QID	0.26	± 11.015

Week 3

Group	Value	95% CI
Pazopanib 10 mg/mL QID	-1.47	± 9.927

Week 4

Group	Value	95% CI
Pazopanib 10 mg/mL QID	0.07	± 9.973

Week 6

Group	Value	95% CI
Pazopanib 10 mg/mL QID	1.76	± 9.345

Week 8

Group	Value	95% CI
Pazopanib 10 mg/mL QID	0.63	± 8.912

Week 12

Group	Value	95% CI
Pazopanib 10 mg/mL QID	-0.85	± 8.069

Follow-up

Group	Value	95% CI
Pazopanib 10 mg/mL QID	-5.49	± 10.388

Change From Baseline in the Area of Choroidal Neovascular (CNV) Size and CNV Total Lesion Complex Size as Measured by Fluorescein Angiography (FA) at Day 29 Secondary · Baseline (Day -3 to -1) and Day 29

CNV was the measurement of the combined classic and occult neovascular lesion including areas of classic neovascularization, late staining of undetermined origin and fibrovascular PED. CNV total lesion complex size was the measurement of the entire lesion including classic and occult neovascular components as well as contagious blood and/or blocked fluorescence and/or serous PED. FA uses fundus photography (FP) to capture images of injected dye circulating throughout the retinal blood vessels to assess leaking, swelling/circulation problems caused by various eye diseases like diabetic retinopa

CNV Size at Week 4

Group	Value	95% CI
Pazopanib 10 mg/mL QID	1.38	± 2.902

CNV total lesion complex size at Week 4

Group	Value	95% CI
Pazopanib 10 mg/mL QID	1.37	± 2.892

Number of Participants With Change in Charactertsics (Atrophy, Pigment, SR Hemorrhage, IR Hemorrhage, SR Fluid and Fibrosis) as Measured by FP Secondary · Day 29

Fundus photography involves capturing of images of the center of the very back inner wall of the eye - the retina, optic nerve, macula and main retinal blood vessels. The parameters assessment were heme SR hemorrhage (absence or presence at the location), heme IR hemorrhage (absence or presence at the location), SR fluid (absence or presence at location), fibrosis (absence or presence at location), atrophy (absence or presence of atrophic changes) and pigment (absence or presence at location). A protocol set of fundus photographs were obtained at Day 29. Images were read by the investigator fo

Atrophy at Week 4

Group	Value	95% CI
Pazopanib 10 mg/mL QID	0

Pigment at Week 4

Group	Value	95% CI
Pazopanib 10 mg/mL QID	12

Heme (SR) at Week 4

Group	Value	95% CI
Pazopanib 10 mg/mL QID	10

Heme (IR) at Week 4

Group	Value	95% CI
Pazopanib 10 mg/mL QID	1

SR fluid at Week 4

Group	Value	95% CI
Pazopanib 10 mg/mL QID	13

Fibrosis at Week 4

Group	Value	95% CI
Pazopanib 10 mg/mL QID	1

Number of Participants Who Received Rescue Medication Secondary · Up to follow-up (Day 102)

At any time during the study, including the follow-up period, rescue treatment (standard of care) was given based on the clinical judgment of the investigator. Rescue treatment was to be strongly considered for participants whose center subfield thickness had increased by \>50 microns from the lowest value on study or whose BCVA decreased by more than 5 letters compared to baseline and who also had persistent fluid by OCT. Data has been reported for the number of participants with their percentages who required any rescue medication administration until follow-up.

Group	Value	95% CI
Pazopanib 10 mg/mL QID	9

Number of Participants With Ocular Adverse Events (AEs), Non-ocular AEs, Serious Ocular AEs and Serious Non-ocular AEs Secondary · Until Follow-up (Day 102)

An AE is defined as any untoward medical occurrence in a participant or clinical investigation participant, temporally associated with the use of a medicinal product, whether or not considered related to the medicinal product. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease (new or exacerbated) temporally associated with the use of a medicinal product. An SAE is defined as any untoward medical occurrence that, at any dose, results in death, is life threatening, requires hospitalization or prolongation of existing hospit

Any Ocular AE

Group	Value	95% CI
Pazopanib 10 mg/mL QID	8

Any Ocular SAE

Group	Value	95% CI
Pazopanib 10 mg/mL QID	0

Any Non-Ocular AE

Group	Value	95% CI
Pazopanib 10 mg/mL QID	9

Any Non-Ocular SAE

Group	Value	95% CI
Pazopanib 10 mg/mL QID	0

Number of Participants With Values of Potential Clinical Concern for Ocular Assessments on General Ophthalmic Examination Secondary · Up to Follow-up (Day 102)

A complete eye examination was performed to include the following: Examination of eyelids and lashes (including meibomian glands), Pupil, motility and confrontation visual field examination, Slit lamp evaluation of anterior ocular structures (including conjunctiva, tear film, cornea with fluorescein staining, anterior chamber, iris, lens, and anterior vitreous), Intraocular pressure (IOP) measurement and Dilated Fundus Examination (Indirect ophthalmoscopy and slit lamp biomicroscopy). Data has been presented in a consolidated format for the total number of participants with values of potential

Group	Value	95% CI
Pazopanib 10 mg/mL QID	0

Number of Participants With Vital Sign Data of Potential Clinical Concern Secondary · Up to Follow-up (Day 102)

Vital sign assessments included systolic blood pressure, diastolic blood pressure and heart rate. The potential clinical concern range for systolic blood pressure was \<85 and \>160 millimeters of mercury, diastolic blood pressure \<45 and \> 100 millimeters of mercury, heart rate \<40 and \>110 beats per minute. Data has been presented in a consolidated format for the total number of participants with values of potential clinical concern for systolic blood pressure, diastolic blood pressure and heart rate until Day 102.

Systolic blood pressure

Group	Value	95% CI
Pazopanib 10 mg/mL QID	2

Diastolic blood pressure

Group	Value	95% CI
Pazopanib 10 mg/mL QID	0

Heart rate

Group	Value	95% CI
Pazopanib 10 mg/mL QID	0

Number of Participants With Clinical Chemistry and Hematology Data of Potential Clinical Concern Secondary · Up to Follow-up (Day 102)

Clinical chemistry parameters included albumin, alkaline phosphatase, alanine amino transferase, aspartate amino transferase, direct bilirubin, total bilirubin, calcium, chloride, carbon dioxide, creatinine, thyroxine (T3 free), gamma glutamyl transferase, glucose, potassium, sodium, total protein, total T3, urea, uric acid while hematology included basophils, eosinophils, hemoglobin, hematocrit, lymphocytes, mean corpuscle hemoglobin concentration, mean corpuscle hemoglobin, mean corpuscle volume, monocytes, segmented neutrophils, total neutrophils, platelet count, red blood cell count, retic

Glucose high

Group	Value	95% CI
Pazopanib 10 mg/mL QID	1

Carbon dioxide low

Group	Value	95% CI
Pazopanib 10 mg/mL QID	2

Lymphocyte low

Group	Value	95% CI
Pazopanib 10 mg/mL QID	1

Platelet count low

Group	Value	95% CI
Pazopanib 10 mg/mL QID	1

Adverse events — posted to ClinicalTrials.gov

Time frame: Up to Follow-up (Day 102). Reporting threshold: 5%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.

Pazopanib 10 mg/mL QID

Serious: 0/19 (0%)

Deaths: 0/19

Other adverse events (22 terms — click to expand)

Reaction	System	Pazopanib 10 mg/mL QID
Instillation site pain	General disorders	—
Age-related macular degeneration	Eye disorders	—
Macular oedema	Eye disorders	—
Ocular Hypertension	Eye disorders	—
Retinal haemorrhage	Eye disorders	—
Vitreous floaters	Eye disorders	—
Nausea	Gastrointestinal disorders	—
Oesophageal obstruction	Gastrointestinal disorders	—
Tooth infection	Infections and infestations	—
Urinary tract infection	Infections and infestations	—
Skeletal injury	Injury, poisoning and procedural complications	—
Platelet count decreased	Investigations	—
Protein urine present	Investigations	—
Red blood cells urine positive	Investigations	—
Urine protein/creatinine ratio increased	Investigations	—
Arthritis	Musculoskeletal and connective tissue disorders	—
Tendonitis	Musculoskeletal and connective tissue disorders	—
Torticollis	Musculoskeletal and connective tissue disorders	—
Depression	Psychiatric disorders	—
Cough	Respiratory, thoracic and mediastinal disorders	—
Dyshponia	Respiratory, thoracic and mediastinal disorders	—
Hypertension	Vascular disorders	—

Data from ClinicalTrials.gov NCT01362348 adverse events section.

Sponsor's own description

The purpose of this 12 week, open-label study is to investigate the safety and efficacy of a single dose regimen of pazopanib eye drop for neovascular AMD.

Publications & conference data

3 peer-reviewed publications reference this trial (live from Europe PMC):

1. Recent Advances in Age-Related Macular Degeneration Therapies.
   Fabre M, Mateo L, Lamaa D, Baillif S, et al · · 2022 · cited 68× · PMID 36014339 · DOI 10.3390/molecules27165089
2. FDA-Approved Kinase Inhibitors in Preclinical and Clinical Trials for Neurological Disorders.
   Lui A, Vanleuven J, Perekopskiy D, Liu D, et al · · 2022 · cited 15× · PMID 36558997 · DOI 10.3390/ph15121546
3. Experimental and clinical tests of FDA-approved kinase inhibitors for the treatment of neurological disorders (update 2024).
   Aliashrafzadeh H, Liu D, De Alba S, Akbar I, et al · · 2025 · PMID 40708570 · DOI 10.37349/eds.2025.1008116