Trial Outcomes & Findings for Exploratory Study of KPI-121 Effect on Intra- or Subretinal Fluid Due to Retinal Vein Occlusion/Diabetic Macular Edema (NCT NCT02245516)

Last Updated: 2020-12-10

Results Overview

Best Corrected Visual Acuity (BCVA) was measured by the Early Treatment Diabetic Retinopathy Study (ETDRS) letter score starting at 4 meters. More letters read correctly results in a higher letter score, which represents better visual acuity.

Recruitment status

COMPLETED

Study phase

Target enrollment

16 participants

Primary outcome timeframe

Day 1 to Day 57

Results posted on

2020-12-10

Participant Flow

Participant milestones

Participant milestones
Measure	KPI-121 1.0% Ophthalmic Suspension KPI-121 1.0% Ophthalmic Suspension dosed QID for 4 weeks in subjects with Retinal Vein Occlusion or Diabetic Macular Edema KPI-121 1.0% Ophthalmic Suspension: KPI-121 drug product will be supplied a 1.0% loteprednol etabonate as a suspension packaged opaque dropper bottles. KPI-121 drug product is a sterile, aqueous, submicron suspension of loteprednol etabonate.	KPI-121 0.25% Ophthalmic Suspension KPI-121 0.25% Ophthalmic Suspension dosed QID for 4 weeks in subjects with Retinal Vein Occlusion or Diabetic Macular Edema KPI-121 0.25% Ophthalmic Suspension: KPI-121 drug product will be supplied a 0.25% loteprednol etabonate as a suspension packaged opaque dropper bottles. KPI-121 drug product is a sterile, aqueous, submicron suspension of loteprednol etabonate.
Overall Study STARTED	9	7
Overall Study COMPLETED	9	4
Overall Study NOT COMPLETED	0	3

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Exploratory Study of KPI-121 Effect on Intra- or Subretinal Fluid Due to Retinal Vein Occlusion/Diabetic Macular Edema

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	KPI-121 1.0% Ophthalmic Suspension n=9 Participants KPI-121 1.0% Ophthalmic Suspension dosed QID for 4 weeks in subjects with Retinal Vein Occlusion or Diabetic Macular Edema KPI-121 1.0% Ophthalmic Suspension: KPI-121 drug product will be supplied a 1.0% loteprednol etabonate as a suspension packaged opaque dropper bottles. KPI-121 drug product is a sterile, aqueous, submicron suspension of loteprednol etabonate.	KPI-121 0.25% Ophthalmic Suspension n=7 Participants KPI-121 0.25% Ophthalmic Suspension dosed QID for 4 weeks in subjects with Retinal Vein Occlusion or Diabetic Macular Edema KPI-121 0.25% Ophthalmic Suspension: KPI-121 drug product will be supplied a 0.25% loteprednol etabonate as a suspension packaged opaque dropper bottles. KPI-121 drug product is a sterile, aqueous, submicron suspension of loteprednol etabonate.	Total n=16 Participants Total of all reporting groups
Age, Customized Age (years)	63.2 years STANDARD_DEVIATION 12.93 • n=5 Participants	65.4 years STANDARD_DEVIATION 10.34 • n=7 Participants	64.2 years STANDARD_DEVIATION 11.54 • n=5 Participants
Sex: Female, Male Female	2 Participants n=5 Participants	2 Participants n=7 Participants	4 Participants n=5 Participants
Sex: Female, Male Male	7 Participants n=5 Participants	5 Participants n=7 Participants	12 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	3 Participants n=5 Participants	1 Participants n=7 Participants	4 Participants n=5 Participants
Race (NIH/OMB) White	6 Participants n=5 Participants	6 Participants n=7 Participants	12 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Region of Enrollment United States	9 Participants n=5 Participants	7 Participants n=7 Participants	16 Participants n=5 Participants

PRIMARY outcome

Timeframe: Day 1 to Day 57

Population: Intent to treat- all subjects randomized

Outcome measures

Outcome measures
Measure	KPI-121 1.0% Ophthalmic Suspension n=9 Participants KPI-121 1.0% Ophthalmic Suspension dosed QID for 4 weeks in subjects with Retinal Vein Occlusion or Diabetic Macular Edema KPI-121 1.0% Ophthalmic Suspension: KPI-121 drug product will be supplied a 1.0% loteprednol etabonate as a suspension packaged opaque dropper bottles. KPI-121 drug product is a sterile, aqueous, submicron suspension of loteprednol etabonate.	KPI-121 0.25% Ophthalmic Suspension n=7 Participants KPI-121 0.25% Ophthalmic Suspension dosed QID for 4 weeks in subjects with Retinal Vein Occlusion or Diabetic Macular Edema KPI-121 0.25% Ophthalmic Suspension: KPI-121 drug product will be supplied a 0.25% loteprednol etabonate as a suspension packaged opaque dropper bottles. KPI-121 drug product is a sterile, aqueous, submicron suspension of loteprednol etabonate.
Best Corrected Visual Acuity (BCVA) by Early Treatment Diabetic Retinopathy Study (ETDRS)	-0.9 ETDRS letters Standard Deviation 7.83	-2.0 ETDRS letters Standard Deviation 2.00

PRIMARY outcome

Timeframe: Day 1 to Day 57

Population: Intent to Treat (all randomized subjects) minus subjects in each treatment group that discontinued the study prior to this time point or otherwise did not have this assessment completed

Change in measurement (in microns) in macular volume as measured by Spectral Domain Optical Coherence Tomography (OCT)

Outcome measures

Outcome measures
Measure	KPI-121 1.0% Ophthalmic Suspension n=9 Participants KPI-121 1.0% Ophthalmic Suspension dosed QID for 4 weeks in subjects with Retinal Vein Occlusion or Diabetic Macular Edema KPI-121 1.0% Ophthalmic Suspension: KPI-121 drug product will be supplied a 1.0% loteprednol etabonate as a suspension packaged opaque dropper bottles. KPI-121 drug product is a sterile, aqueous, submicron suspension of loteprednol etabonate.	KPI-121 0.25% Ophthalmic Suspension n=4 Participants KPI-121 0.25% Ophthalmic Suspension dosed QID for 4 weeks in subjects with Retinal Vein Occlusion or Diabetic Macular Edema KPI-121 0.25% Ophthalmic Suspension: KPI-121 drug product will be supplied a 0.25% loteprednol etabonate as a suspension packaged opaque dropper bottles. KPI-121 drug product is a sterile, aqueous, submicron suspension of loteprednol etabonate.
Macular Volume by SD-OCT	-0.237 mm^3 Standard Deviation 1.1607	-0.377 mm^3 Standard Deviation 0.8190

PRIMARY outcome

Timeframe: Day 1 to Day 57

Population: Intent to Treat (all randomized subjects) minus subjects in each treatment group that discontinued the study prior to this time point or otherwise did not have this assessment completed

Change in measurement (in microns) in the central subfield retinal thickness as measured by Spectral Domain Optical Coherence Tomography (OCT)

Outcome measures

Outcome measures
Measure	KPI-121 1.0% Ophthalmic Suspension n=9 Participants KPI-121 1.0% Ophthalmic Suspension dosed QID for 4 weeks in subjects with Retinal Vein Occlusion or Diabetic Macular Edema KPI-121 1.0% Ophthalmic Suspension: KPI-121 drug product will be supplied a 1.0% loteprednol etabonate as a suspension packaged opaque dropper bottles. KPI-121 drug product is a sterile, aqueous, submicron suspension of loteprednol etabonate.	KPI-121 0.25% Ophthalmic Suspension n=4 Participants KPI-121 0.25% Ophthalmic Suspension dosed QID for 4 weeks in subjects with Retinal Vein Occlusion or Diabetic Macular Edema KPI-121 0.25% Ophthalmic Suspension: KPI-121 drug product will be supplied a 0.25% loteprednol etabonate as a suspension packaged opaque dropper bottles. KPI-121 drug product is a sterile, aqueous, submicron suspension of loteprednol etabonate.
Center Subfield Retinal Thickness by SD-OCT	-8.2 micrometers (μm) Standard Deviation 52.37	4.8 micrometers (μm) Standard Deviation 13.99

PRIMARY outcome

Timeframe: Day 1 to Day 57

Population: Unable to analyze the FA data for efficacy. The efficacy variable defined in the protocol was the mean change from baseline in total area of fluorescein leakage and unfortunately the total area was not collected during the study.

Change in leakage by investigator's assessment of the fluorescein angiogram

Outcome measures

Outcome data not reported

Adverse Events

KPI-121 1.0% Ophthalmic Suspension

Serious events: 0 serious events

Other events: 3 other events

Deaths: 0 deaths

KPI-121 0.25% Ophthalmic Suspension

Serious events: 1 serious events

Other events: 2 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	KPI-121 1.0% Ophthalmic Suspension n=9 participants at risk KPI-121 1.0% Ophthalmic Suspension dosed QID for 4 weeks in subjects with Retinal Vein Occlusion or Diabetic Macular Edema KPI-121 1.0% Ophthalmic Suspension: KPI-121 drug product will be supplied a 1.0% loteprednol etabonate as a suspension packaged opaque dropper bottles. KPI-121 drug product is a sterile, aqueous, submicron suspension of loteprednol etabonate.	KPI-121 0.25% Ophthalmic Suspension n=7 participants at risk KPI-121 0.25% Ophthalmic Suspension dosed QID for 4 weeks in subjects with Retinal Vein Occlusion or Diabetic Macular Edema KPI-121 0.25% Ophthalmic Suspension: KPI-121 drug product will be supplied a 0.25% loteprednol etabonate as a suspension packaged opaque dropper bottles. KPI-121 drug product is a sterile, aqueous, submicron suspension of loteprednol etabonate.
General disorders generalized weakness	0.00% 0/9 • Adverse events were collected from the time a subjects signed the informed consent until they exited the study at the end of Visit 5 (up to 57 days).	14.3% 1/7 • Adverse events were collected from the time a subjects signed the informed consent until they exited the study at the end of Visit 5 (up to 57 days).

Other adverse events

Other adverse events
Measure	KPI-121 1.0% Ophthalmic Suspension n=9 participants at risk KPI-121 1.0% Ophthalmic Suspension dosed QID for 4 weeks in subjects with Retinal Vein Occlusion or Diabetic Macular Edema KPI-121 1.0% Ophthalmic Suspension: KPI-121 drug product will be supplied a 1.0% loteprednol etabonate as a suspension packaged opaque dropper bottles. KPI-121 drug product is a sterile, aqueous, submicron suspension of loteprednol etabonate.	KPI-121 0.25% Ophthalmic Suspension n=7 participants at risk KPI-121 0.25% Ophthalmic Suspension dosed QID for 4 weeks in subjects with Retinal Vein Occlusion or Diabetic Macular Edema KPI-121 0.25% Ophthalmic Suspension: KPI-121 drug product will be supplied a 0.25% loteprednol etabonate as a suspension packaged opaque dropper bottles. KPI-121 drug product is a sterile, aqueous, submicron suspension of loteprednol etabonate.
Eye disorders Increase in floaters	11.1% 1/9 • Number of events 1 • Adverse events were collected from the time a subjects signed the informed consent until they exited the study at the end of Visit 5 (up to 57 days).	0.00% 0/7 • Adverse events were collected from the time a subjects signed the informed consent until they exited the study at the end of Visit 5 (up to 57 days).
Eye disorders Pain	11.1% 1/9 • Number of events 1 • Adverse events were collected from the time a subjects signed the informed consent until they exited the study at the end of Visit 5 (up to 57 days).	0.00% 0/7 • Adverse events were collected from the time a subjects signed the informed consent until they exited the study at the end of Visit 5 (up to 57 days).
Eye disorders Punctate Epithelial Erosions	11.1% 1/9 • Number of events 1 • Adverse events were collected from the time a subjects signed the informed consent until they exited the study at the end of Visit 5 (up to 57 days).	0.00% 0/7 • Adverse events were collected from the time a subjects signed the informed consent until they exited the study at the end of Visit 5 (up to 57 days).
General disorders generalized weakness	0.00% 0/9 • Adverse events were collected from the time a subjects signed the informed consent until they exited the study at the end of Visit 5 (up to 57 days).	14.3% 1/7 • Number of events 1 • Adverse events were collected from the time a subjects signed the informed consent until they exited the study at the end of Visit 5 (up to 57 days).
Eye disorders Soreness	0.00% 0/9 • Adverse events were collected from the time a subjects signed the informed consent until they exited the study at the end of Visit 5 (up to 57 days).	14.3% 1/7 • Number of events 1 • Adverse events were collected from the time a subjects signed the informed consent until they exited the study at the end of Visit 5 (up to 57 days).
General disorders Worsening of Hypercholesterolemia	0.00% 0/9 • Adverse events were collected from the time a subjects signed the informed consent until they exited the study at the end of Visit 5 (up to 57 days).	14.3% 1/7 • Number of events 1 • Adverse events were collected from the time a subjects signed the informed consent until they exited the study at the end of Visit 5 (up to 57 days).

Additional Information

VP, Clinical Development

Kala Pharmaceuticals Inc

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee No submission for publication or public disclosure will be made by the PI until after the publication of the results of the multi-center clinical trial by the Sponsor. Thereafter, PI shall provide KALA with an advance copy of any proposed publication or public disclosure at least sixty (60) days prior to the planned date of submission or disclosure and KALA shall have forty-five (45) days from the date of its receipt to recommend changes.
Publication restrictions are in place

Restriction type: OTHER