Trial Outcomes & Findings for Regorafenib Eye Drops: Investigation of Efficacy and Safety in Neovascular Age Related Macular Degeneration (NCT NCT02222207)

Results Overview

Participants will be assessed at each clinic visit for best corrected visual acuity using the early treatment diabetic retinopathy study chart. Visual function of the study eye and the fellow eye was assessed using the ETDRS. The participant's ETDRS testing score was recorded in the appropriate eCRF page at each study visit. For participants that dropped out or received rescue treatment the last observation before drop-out or administration of rescue treatment was carried forward. A higher score represents better functioning.

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

52 participants

Primary outcome timeframe

Baseline, Week 4

Results posted on

2016-09-08

Participant Flow

The study was conducted at 27 centers, between 10 October 2014 (first subject first visit) and 17 June 2015 (last subject last visit).

Study was planned to be conducted in 2 parts, Part A and B, in Part A 89 subjects were enrolled, of them 37 were screen failure and 52 were assigned to treatment, 1 subject was excluded from all analysis sets due to protocol deviations and 51 subjects were analyzed. Part B was not initiated and the study terminated following completion of Part A.

Participant milestones

Participant milestones
Measure	Part A Regorafenib Participants self-administered 30 milligram per milliliter (mg/mL), 25 microliter (mcL),1 drop of Regorafenib eye drops, topically thrice daily (TID) to the study eye for 12 weeks.
Overall Study STARTED	52
Overall Study Treated and Valid for Full Analysis Set	51
Overall Study COMPLETED	48
Overall Study NOT COMPLETED	4

Reasons for withdrawal

Reasons for withdrawal
Measure	Part A Regorafenib Participants self-administered 30 milligram per milliliter (mg/mL), 25 microliter (mcL),1 drop of Regorafenib eye drops, topically thrice daily (TID) to the study eye for 12 weeks.
Overall Study Death	1
Overall Study Other reasons	2
Overall Study Adverse Event	1

Baseline Characteristics

Regorafenib Eye Drops: Investigation of Efficacy and Safety in Neovascular Age Related Macular Degeneration

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Part A Regorafenib n=51 Participants Participants self-administered 30 milligram per milliliter (mg/mL), 25 microliter (mcL),1 drop of Regorafenib eye drops, topically thrice daily (TID) to the study eye for 12 weeks.
Age, Continuous	75 years STANDARD_DEVIATION 8.4 • n=93 Participants
Sex: Female, Male Female	29 Participants n=93 Participants
Sex: Female, Male Male	22 Participants n=93 Participants

PRIMARY outcome

Timeframe: Baseline, Week 4

Population: Full Analysis Set (FAS): included participants who received at least one dose of study medication.

Participants will be assessed at each clinic visit for best corrected visual acuity using the early treatment diabetic retinopathy study chart. Visual function of the study eye and the fellow eye was assessed using the ETDRS. The participant's ETDRS testing score was recorded in the appropriate eCRF page at each study visit. For participants that dropped out or received rescue treatment the last observation before drop-out or administration of rescue treatment was carried forward. A higher score represents better functioning.

Outcome measures

Outcome measures
Measure	Part A Regorafenib n=50 Participants Participants self-administered 30 milligram per milliliter (mg/mL), 25 microliter (mcL),1 drop of Regorafenib eye drops, topically thrice daily (TID) to the study eye for 12 weeks.
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	1.18 Score on scale Standard Deviation 7.55 • Interval 7.55 to

PRIMARY outcome

Timeframe: Baseline, Week 12

Population: Full Analysis Set (FAS): included subjects who received at least one dose of study medication.

Participants were assessed at each clinic visit for best corrected visual acuity using the early treatment diabetic retinopathy study chart. Visual function of the study eye and the fellow eye was assessed using the ETDRS protocol. ETDRS testing score was recorded in the appropriate eCRF page at each study visit. For participants that dropped out or received rescue treatment the last observation before drop-out or administration of rescue treatment was carried forward. A higher score represents better functioning.

Outcome measures

Outcome measures
Measure	Part A Regorafenib n=50 Participants Participants self-administered 30 milligram per milliliter (mg/mL), 25 microliter (mcL),1 drop of Regorafenib eye drops, topically thrice daily (TID) to the study eye for 12 weeks.
Change From Baseline in BCVA as Measured by ETDRS Letter Score at Study Week 12 for Study Part A	-2.36 Score on scale Standard Deviation 7.68 • Interval 7.68 to

SECONDARY outcome

Timeframe: Week 4, Week 12

Population: Full Analysis Set (FAS): included subjects who received at least one dose of study medication.

Participants were assessed at each clinic visit for BCVA using the early treatment diabetic retinopathy study chart. For participants that dropped out or received rescue treatment the last observation before drop-out or administration of rescue treatment was carried forward.

Outcome measures

Outcome measures
Measure	Part A Regorafenib n=50 Participants Participants self-administered 30 milligram per milliliter (mg/mL), 25 microliter (mcL),1 drop of Regorafenib eye drops, topically thrice daily (TID) to the study eye for 12 weeks.
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	42 percentage of participants

SECONDARY outcome

Timeframe: Baseline, Week 12

Population: Full Analysis Set (FAS): included subjects who received at least one dose of study medication.

Participants were assessed at each clinic visit for BCVA using the early treatment diabetic retinopathy study chart. For participants that dropped out or received rescue treatment the last observation before drop-out or administration of rescue treatment was carried forward.

Outcome measures

Outcome measures
Measure	Part A Regorafenib n=50 Participants Participants self-administered 30 milligram per milliliter (mg/mL), 25 microliter (mcL),1 drop of Regorafenib eye drops, topically thrice daily (TID) to the study eye for 12 weeks.
Percentage of Participants With a Loss in BCVA of >= 10 Letters From Baseline to Study Week 12 for Study Part A	16 percentage of participants

Adverse Events

Part A Regorafenib

Serious events: 3 serious events

Other events: 11 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Part A Regorafenib n=51 participants at risk Participants self-administered 30 mg/mL, 25 mcL, 1 drop of regorafenib eye drops, topically thrice daily (TID) to the study eye for 12 weeks.
Cardiac disorders Acute myocardial infarction	2.0% 1/51 • From the start of study treatment until 30 days after last dose of study drug treatment (up to Week 16).
Injury, poisoning and procedural complications Ankle fracture	2.0% 1/51 • From the start of study treatment until 30 days after last dose of study drug treatment (up to Week 16).
Investigations Visual acuity tests abnormal	2.0% 1/51 • From the start of study treatment until 30 days after last dose of study drug treatment (up to Week 16).

Other adverse events

Other adverse events
Measure	Part A Regorafenib n=51 participants at risk Participants self-administered 30 mg/mL, 25 mcL, 1 drop of regorafenib eye drops, topically thrice daily (TID) to the study eye for 12 weeks.
Eye disorders Visual acuity reduced	11.8% 6/51 • From the start of study treatment until 30 days after last dose of study drug treatment (up to Week 16).
Investigations Visual acuity tests abnormal	9.8% 5/51 • From the start of study treatment until 30 days after last dose of study drug treatment (up to Week 16).

Additional Information

Therapeutic Area Head

BAYER

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee In mulit centre studies result of study are to be published only through coordination by Bayer in order to combine the results of all centres. Center free to publish its results provided the overall results have not been published within 18 months from study completion (data base lock), subject to the compliance to remaining terms set forth in the PI contract.
Publication restrictions are in place

Restriction type: OTHER