Trial Outcomes & Findings for A Study to Investigate the Efficacy and Safety of RG7774 in Patients With Diabetes Mellitus Type 1 or Type 2 With Treatment-Naive Diabetic Retinopathy (NCT NCT04265261)

Last Updated: 2024-09-25

Results Overview

The ETDRS DRSS is a standardized grading test to measure diabetic retinopathy progression, where higher scores indicate a higher risk of vision loss. The DRSS ranges from level 10 (no diabetic retinopathy) to level 85 (advanced diabetic retinopathy)

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

139 participants

Primary outcome timeframe

Week 36

Results posted on

2024-09-25

Participant Flow

Participant milestones

Participant milestones
Measure	Placebo Participants received an oral dose of placebo matched to RG7774 once daily (QD)	Vicasinabin 30 mg QD Participants received 30 mg of oral RG7774 QD	Vicasinabin 200 mg QD Participants received 200 mg of oral RG7774 QD
Overall Study STARTED	47	48	44
Overall Study COMPLETED	37	43	36
Overall Study NOT COMPLETED	10	5	8

Reasons for withdrawal

Reasons for withdrawal
Measure	Placebo Participants received an oral dose of placebo matched to RG7774 once daily (QD)	Vicasinabin 30 mg QD Participants received 30 mg of oral RG7774 QD	Vicasinabin 200 mg QD Participants received 200 mg of oral RG7774 QD
Overall Study Adverse Event	2	0	0
Overall Study Death	1	0	0
Overall Study Lost to Follow-up	4	1	3
Overall Study Non-Compliance with Study Drug	1	1	0
Overall Study Physician Decision	0	0	1
Overall Study Protocol Deviation	0	2	0
Overall Study Withdrawal by Subject	2	1	4

Baseline Characteristics

A Study to Investigate the Efficacy and Safety of RG7774 in Patients With Diabetes Mellitus Type 1 or Type 2 With Treatment-Naive Diabetic Retinopathy

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Placebo n=47 Participants Participants received an oral dose of placebo matched to RG7774 once daily (QD)	Vicasinabin 30 mg QD n=48 Participants Participants received 30 mg of oral RG7774 QD	Vicasinabin 200 mg QD n=44 Participants Participants received 200 mg of oral RG7774 QD	Total n=139 Participants Total of all reporting groups
Age, Continuous	58.9 Years STANDARD_DEVIATION 9.3 • n=5 Participants	57.3 Years STANDARD_DEVIATION 10.0 • n=7 Participants	56.5 Years STANDARD_DEVIATION 10.5 • n=5 Participants	57.6 Years STANDARD_DEVIATION 9.9 • n=4 Participants
Sex: Female, Male Female	17 Participants n=5 Participants	18 Participants n=7 Participants	16 Participants n=5 Participants	51 Participants n=4 Participants
Sex: Female, Male Male	30 Participants n=5 Participants	30 Participants n=7 Participants	28 Participants n=5 Participants	88 Participants n=4 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	14 Participants n=5 Participants	18 Participants n=7 Participants	15 Participants n=5 Participants	47 Participants n=4 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	32 Participants n=5 Participants	30 Participants n=7 Participants	29 Participants n=5 Participants	91 Participants n=4 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	1 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	1 Participants n=4 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Race (NIH/OMB) Asian	2 Participants n=5 Participants	2 Participants n=7 Participants	3 Participants n=5 Participants	7 Participants n=4 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	0 Participants n=4 Participants
Race (NIH/OMB) Black or African American	5 Participants n=5 Participants	0 Participants n=7 Participants	4 Participants n=5 Participants	9 Participants n=4 Participants
Race (NIH/OMB) White	40 Participants n=5 Participants	46 Participants n=7 Participants	35 Participants n=5 Participants	121 Participants n=4 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	2 Participants n=5 Participants	2 Participants n=4 Participants

PRIMARY outcome

Timeframe: Week 36

Population: The mITT population is defined as all participants who give informed consent, are randomized, and receive at least one dose of study treatment (active or placebo). For the mITT population, data were analyzed according to the treatment participants were randomized to.

Outcome measures

Outcome measures
Measure	Placebo n=38 Participants Participants received an oral dose of placebo matched to RG7774 once daily (QD)	Vicasinabin 30 mg QD n=42 Participants Participants received 30 mg of oral RG7774 QD	Vicasinabin 200 mg QD n=35 Participants Participants received 200 mg of oral RG7774 QD
Proportion of Participants With >/= 2-Step Improvement in the Early Treatment Diabetic Retinopathy Study (ETDRS) DR Severity Scale (DRSS) From Baseline at Week 36 Measured in the Study Eye	7.89 Percentage of participants Interval 2.72 to 20.8	9.52 Percentage of participants Interval 3.77 to 22.07	5.71 Percentage of participants Interval 1.58 to 18.61

PRIMARY outcome

Timeframe: Up to 1 year (baseline through follow-up period)

Population: The Safety population is defined as all participants who give informed consent, receive at least one dose of study medication (active or placebo) and were grouped according to the actual treatment received.

Outcome measures

Outcome measures
Measure	Placebo n=47 Participants Participants received an oral dose of placebo matched to RG7774 once daily (QD)	Vicasinabin 30 mg QD n=48 Participants Participants received 30 mg of oral RG7774 QD	Vicasinabin 200 mg QD n=43 Participants Participants received 200 mg of oral RG7774 QD
Percentage of Participants With Adverse Events (AEs)	72.3 Percentage of participants	64.6 Percentage of participants	81.4 Percentage of participants

SECONDARY outcome

Timeframe: Up to Day 277

Vision-threatening DR was defined as anterior segment neovascularization (ASNV), new proliferative diabetic retinopathy (PDR), new diabetic macular edema (DME), and pre-existing DME requiring treatment. Time-to-event was defined as the time where 50% of the population develops a DR vision-threatening event.

Outcome measures

Outcome measures
Measure	Placebo n=47 Participants Participants received an oral dose of placebo matched to RG7774 once daily (QD)	Vicasinabin 30 mg QD n=48 Participants Participants received 30 mg of oral RG7774 QD	Vicasinabin 200 mg QD n=43 Participants Participants received 200 mg of oral RG7774 QD
Time-to-Event for Vision-Threatening DR in the Study Eye	NA Days Interval 257.0 to NA indicates that either the Kaplan-Meier percentile time has not been achieved or that the percentile is at a boundary of the observed range and no upper or lower 95% CI can be found due to insufficient sample size.	267.0 Days Interval 254.0 to NA indicates that either the Kaplan-Meier percentile time has not been achieved or that the percentile is at a boundary of the observed range and no upper or lower 95% CI can be found due to insufficient sample size..	NA Days Interval 260.0 to NA indicates that either the Kaplan-Meier percentile time has not been achieved or that the percentile is at a boundary of the observed range and no upper or lower 95% CI can be found due to insufficient sample size..

SECONDARY outcome

Timeframe: Week 36

This is a descriptive summary of the incidence of new ASNV, new PDR, new DME, and pre-existing DME, all of which indicate disease progression. Each row presents the proportion of participants amongst the overall population for each event.

Outcome measures

Outcome measures
Measure	Placebo n=47 Participants Participants received an oral dose of placebo matched to RG7774 once daily (QD)	Vicasinabin 30 mg QD n=48 Participants Participants received 30 mg of oral RG7774 QD	Vicasinabin 200 mg QD n=43 Participants Participants received 200 mg of oral RG7774 QD
Incidence of New Anterior Segment Neovascularization (ASNV), New Proliferative Diabetic Retinopathy (PDR), New Diabetic Macular Edema (DME), and Pre-Existing DME Requiring Intervention in the Study Eye New ASNV	0 Percentage of participants Interval 0.0 to 9.4	0 Percentage of participants Interval 0.0 to 9.2	0 Percentage of participants Interval 0.0 to 10.2
Incidence of New Anterior Segment Neovascularization (ASNV), New Proliferative Diabetic Retinopathy (PDR), New Diabetic Macular Edema (DME), and Pre-Existing DME Requiring Intervention in the Study Eye New PDR	0 Percentage of participants Interval 0.0 to 9.4	6.3 Percentage of participants Interval 1.6 to 18.2	0 Percentage of participants Interval 0.0 to 10.2
Incidence of New Anterior Segment Neovascularization (ASNV), New Proliferative Diabetic Retinopathy (PDR), New Diabetic Macular Edema (DME), and Pre-Existing DME Requiring Intervention in the Study Eye New DME	0 Percentage of participants Interval 0.0 to 9.4	4.2 Percentage of participants Interval 0.7 to 15.4	0 Percentage of participants Interval 0.0 to 10.2
Incidence of New Anterior Segment Neovascularization (ASNV), New Proliferative Diabetic Retinopathy (PDR), New Diabetic Macular Edema (DME), and Pre-Existing DME Requiring Intervention in the Study Eye Pre-existing DME requiring treatment	4.3 Percentage of participants Interval 0.7 to 15.7	10.4 Percentage of participants Interval 3.9 to 23.4	6.8 Percentage of participants Interval 1.8 to 20.1

SECONDARY outcome

Timeframe: Baseline; Week 36

BCVA was measured by a qualified VA examiner prior to pupil dilation using modified ETDRS Charts 1, 2, and R. The adjusted mean is reported for each group.

Outcome measures

Outcome measures
Measure	Placebo n=47 Participants Participants received an oral dose of placebo matched to RG7774 once daily (QD)	Vicasinabin 30 mg QD n=48 Participants Participants received 30 mg of oral RG7774 QD	Vicasinabin 200 mg QD n=43 Participants Participants received 200 mg of oral RG7774 QD
Change From Baseline in Best Corrected Visual Acuity (BCVA) in the Study Eye at Week 36	0.12 Number of letters Standard Error 0.747	-0.45 Number of letters Standard Error 0.697	-0.22 Number of letters Standard Error 0.744

Adverse Events

Placebo

Serious events: 8 serious events

Other events: 13 other events

Deaths: 1 deaths

Vicasinabin 30 mg QD

Serious events: 3 serious events

Other events: 21 other events

Deaths: 0 deaths

Vicasinabin 200 mg QD

Serious events: 5 serious events

Other events: 15 other events

Deaths: 0 deaths

Serious adverse events

Other adverse events

Other adverse events
Measure	Placebo n=47 participants at risk Participants received an oral dose of placebo matched to RG7774 once daily (QD)	Vicasinabin 30 mg QD n=48 participants at risk Participants received 30 mg of oral RG7774 QD	Vicasinabin 200 mg QD n=43 participants at risk Participants received 200 mg of oral RG7774 QD
Eye disorders Diabetic retinal oedema	6.4% 3/47 • Number of events 3 • Up to 1 year (baseline through follow-up period) All-cause mortality includes the entire study population. SAE and NSAE reporting includes the safety population, which included all participants who gave informed consent and received at least one dose of study medication. Participants in the safety population were grouped according to the actual treatment received.	16.7% 8/48 • Number of events 13 • Up to 1 year (baseline through follow-up period) All-cause mortality includes the entire study population. SAE and NSAE reporting includes the safety population, which included all participants who gave informed consent and received at least one dose of study medication. Participants in the safety population were grouped according to the actual treatment received.	14.0% 6/43 • Number of events 7 • Up to 1 year (baseline through follow-up period) All-cause mortality includes the entire study population. SAE and NSAE reporting includes the safety population, which included all participants who gave informed consent and received at least one dose of study medication. Participants in the safety population were grouped according to the actual treatment received.
Eye disorders Diabetic retinopathy	6.4% 3/47 • Number of events 3 • Up to 1 year (baseline through follow-up period) All-cause mortality includes the entire study population. SAE and NSAE reporting includes the safety population, which included all participants who gave informed consent and received at least one dose of study medication. Participants in the safety population were grouped according to the actual treatment received.	10.4% 5/48 • Number of events 5 • Up to 1 year (baseline through follow-up period) All-cause mortality includes the entire study population. SAE and NSAE reporting includes the safety population, which included all participants who gave informed consent and received at least one dose of study medication. Participants in the safety population were grouped according to the actual treatment received.	2.3% 1/43 • Number of events 2 • Up to 1 year (baseline through follow-up period) All-cause mortality includes the entire study population. SAE and NSAE reporting includes the safety population, which included all participants who gave informed consent and received at least one dose of study medication. Participants in the safety population were grouped according to the actual treatment received.
Eye disorders Vitreous haemorrhage	2.1% 1/47 • Number of events 2 • Up to 1 year (baseline through follow-up period) All-cause mortality includes the entire study population. SAE and NSAE reporting includes the safety population, which included all participants who gave informed consent and received at least one dose of study medication. Participants in the safety population were grouped according to the actual treatment received.	6.2% 3/48 • Number of events 3 • Up to 1 year (baseline through follow-up period) All-cause mortality includes the entire study population. SAE and NSAE reporting includes the safety population, which included all participants who gave informed consent and received at least one dose of study medication. Participants in the safety population were grouped according to the actual treatment received.	4.7% 2/43 • Number of events 3 • Up to 1 year (baseline through follow-up period) All-cause mortality includes the entire study population. SAE and NSAE reporting includes the safety population, which included all participants who gave informed consent and received at least one dose of study medication. Participants in the safety population were grouped according to the actual treatment received.
Gastrointestinal disorders Constipation	6.4% 3/47 • Number of events 4 • Up to 1 year (baseline through follow-up period) All-cause mortality includes the entire study population. SAE and NSAE reporting includes the safety population, which included all participants who gave informed consent and received at least one dose of study medication. Participants in the safety population were grouped according to the actual treatment received.	0.00% 0/48 • Up to 1 year (baseline through follow-up period) All-cause mortality includes the entire study population. SAE and NSAE reporting includes the safety population, which included all participants who gave informed consent and received at least one dose of study medication. Participants in the safety population were grouped according to the actual treatment received.	0.00% 0/43 • Up to 1 year (baseline through follow-up period) All-cause mortality includes the entire study population. SAE and NSAE reporting includes the safety population, which included all participants who gave informed consent and received at least one dose of study medication. Participants in the safety population were grouped according to the actual treatment received.
Infections and infestations COVID-19	2.1% 1/47 • Number of events 1 • Up to 1 year (baseline through follow-up period) All-cause mortality includes the entire study population. SAE and NSAE reporting includes the safety population, which included all participants who gave informed consent and received at least one dose of study medication. Participants in the safety population were grouped according to the actual treatment received.	6.2% 3/48 • Number of events 3 • Up to 1 year (baseline through follow-up period) All-cause mortality includes the entire study population. SAE and NSAE reporting includes the safety population, which included all participants who gave informed consent and received at least one dose of study medication. Participants in the safety population were grouped according to the actual treatment received.	11.6% 5/43 • Number of events 5 • Up to 1 year (baseline through follow-up period) All-cause mortality includes the entire study population. SAE and NSAE reporting includes the safety population, which included all participants who gave informed consent and received at least one dose of study medication. Participants in the safety population were grouped according to the actual treatment received.
Infections and infestations Nasopharyngitis	2.1% 1/47 • Number of events 4 • Up to 1 year (baseline through follow-up period) All-cause mortality includes the entire study population. SAE and NSAE reporting includes the safety population, which included all participants who gave informed consent and received at least one dose of study medication. Participants in the safety population were grouped according to the actual treatment received.	6.2% 3/48 • Number of events 3 • Up to 1 year (baseline through follow-up period) All-cause mortality includes the entire study population. SAE and NSAE reporting includes the safety population, which included all participants who gave informed consent and received at least one dose of study medication. Participants in the safety population were grouped according to the actual treatment received.	0.00% 0/43 • Up to 1 year (baseline through follow-up period) All-cause mortality includes the entire study population. SAE and NSAE reporting includes the safety population, which included all participants who gave informed consent and received at least one dose of study medication. Participants in the safety population were grouped according to the actual treatment received.
Metabolism and nutrition disorders Type 2 diabetes mellitus	2.1% 1/47 • Number of events 1 • Up to 1 year (baseline through follow-up period) All-cause mortality includes the entire study population. SAE and NSAE reporting includes the safety population, which included all participants who gave informed consent and received at least one dose of study medication. Participants in the safety population were grouped according to the actual treatment received.	8.3% 4/48 • Number of events 4 • Up to 1 year (baseline through follow-up period) All-cause mortality includes the entire study population. SAE and NSAE reporting includes the safety population, which included all participants who gave informed consent and received at least one dose of study medication. Participants in the safety population were grouped according to the actual treatment received.	2.3% 1/43 • Number of events 1 • Up to 1 year (baseline through follow-up period) All-cause mortality includes the entire study population. SAE and NSAE reporting includes the safety population, which included all participants who gave informed consent and received at least one dose of study medication. Participants in the safety population were grouped according to the actual treatment received.
Nervous system disorders Headache	6.4% 3/47 • Number of events 5 • Up to 1 year (baseline through follow-up period) All-cause mortality includes the entire study population. SAE and NSAE reporting includes the safety population, which included all participants who gave informed consent and received at least one dose of study medication. Participants in the safety population were grouped according to the actual treatment received.	4.2% 2/48 • Number of events 2 • Up to 1 year (baseline through follow-up period) All-cause mortality includes the entire study population. SAE and NSAE reporting includes the safety population, which included all participants who gave informed consent and received at least one dose of study medication. Participants in the safety population were grouped according to the actual treatment received.	4.7% 2/43 • Number of events 2 • Up to 1 year (baseline through follow-up period) All-cause mortality includes the entire study population. SAE and NSAE reporting includes the safety population, which included all participants who gave informed consent and received at least one dose of study medication. Participants in the safety population were grouped according to the actual treatment received.
Vascular disorders Hypertension	4.3% 2/47 • Number of events 2 • Up to 1 year (baseline through follow-up period) All-cause mortality includes the entire study population. SAE and NSAE reporting includes the safety population, which included all participants who gave informed consent and received at least one dose of study medication. Participants in the safety population were grouped according to the actual treatment received.	6.2% 3/48 • Number of events 3 • Up to 1 year (baseline through follow-up period) All-cause mortality includes the entire study population. SAE and NSAE reporting includes the safety population, which included all participants who gave informed consent and received at least one dose of study medication. Participants in the safety population were grouped according to the actual treatment received.	2.3% 1/43 • Number of events 1 • Up to 1 year (baseline through follow-up period) All-cause mortality includes the entire study population. SAE and NSAE reporting includes the safety population, which included all participants who gave informed consent and received at least one dose of study medication. Participants in the safety population were grouped according to the actual treatment received.

Additional Information

Medical Communications

Hoffmann-La Roche

Phone: 1-800-821-8590

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee The Study being conducted under this Agreement is part of the Overall Study. Investigator is free to publish in reputable journals or to present at professional conferences the results of the Study, but only after the first publication or presentation that involves the Overall Study. The Sponsor may request that Confidential Information be deleted and/or the publication be postponed in order to protect the Sponsor's intellectual property rights.
Publication restrictions are in place

Restriction type: OTHER