Trial Outcomes & Findings for A Study to Evaluate the Efficacy of Oral AKST4290 in Participants With Moderately Severe to Severe Diabetic Retinopathy (CAPRI) (NCT NCT05038020)

NCT ID: NCT05038020

Last Updated: 2023-11-01

Results Overview

The Diabetic Retinopathy Severity Scale (DRSS) divides DR into 13 levels ranging from the absence of retinopathy to severe retinopathy and is used to describe overall DR severity as well as the change in severity over time. Higher scores indicate more severe DR. To be eligible for the study, participants needed to have moderately severe non-proliferative DR (NPDR) (DRSS Level 47) to severe NPDR (DRSS Level 53) in one eye, and at least mild NPDR (DRSS Level 35) to mild proliferative DR (PDR) (DRSS Level 61) in the other eye at baseline. The DRSS score is derived from fundus photography (FP) and fluorescein angiography (FA) findings. The primary efficacy endpoint is the proportion of participants with a ≥ 3-step improvement from baseline on the DRSS score as compared with Week 24.

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

3 participants

Primary outcome timeframe

Baseline to Week 24

Results posted on

2023-11-01

Participant Flow

The study was terminated early by the Sponsor. At the time of discontinuation, three participants were enrolled and none completed the study.

Participant milestones

Participant milestones
Measure
AKST4290
Subjects will receive AKST4290, 400mg twice daily, for 24 weeks AKST4290: Oral AKST4290
Placebo
Subjects will receive matching Placebo, twice daily, for 24 weeks Placebo: Oral Placebo
Overall Study
STARTED
3
0
Overall Study
COMPLETED
0
0
Overall Study
NOT COMPLETED
3
0

Reasons for withdrawal

Reasons for withdrawal
Measure
AKST4290
Subjects will receive AKST4290, 400mg twice daily, for 24 weeks AKST4290: Oral AKST4290
Placebo
Subjects will receive matching Placebo, twice daily, for 24 weeks Placebo: Oral Placebo
Overall Study
Early Termination
3
0

Baseline Characteristics

A Study to Evaluate the Efficacy of Oral AKST4290 in Participants With Moderately Severe to Severe Diabetic Retinopathy (CAPRI)

Baseline characteristics by cohort

Baseline data not reported

PRIMARY outcome

Timeframe: Baseline to Week 24

Population: The study was terminated by the Sponsor. Only 3 participants were enrolled in this study. Based on the low enrollment number, no data is reported here in order to protect and maintain participant privacy/confidentiality.

The Diabetic Retinopathy Severity Scale (DRSS) divides DR into 13 levels ranging from the absence of retinopathy to severe retinopathy and is used to describe overall DR severity as well as the change in severity over time. Higher scores indicate more severe DR. To be eligible for the study, participants needed to have moderately severe non-proliferative DR (NPDR) (DRSS Level 47) to severe NPDR (DRSS Level 53) in one eye, and at least mild NPDR (DRSS Level 35) to mild proliferative DR (PDR) (DRSS Level 61) in the other eye at baseline. The DRSS score is derived from fundus photography (FP) and fluorescein angiography (FA) findings. The primary efficacy endpoint is the proportion of participants with a ≥ 3-step improvement from baseline on the DRSS score as compared with Week 24.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Baseline to Week 24 or 28

Population: The study was terminated by the Sponsor. Only 3 participants were enrolled in this study. Based on the low enrollment number, no data is reported here in order to protect and maintain participant privacy/confidentiality.

The Diabetic Retinopathy Severity Scale (DRSS) divides DR into 13 levels ranging from the absence of retinopathy to severe retinopathy and is used to describe overall DR severity as well as the change in severity over time. Higher scores indicate more severe DR. The DRSS score is derived from fundus photography (FP) and fluorescein angiography (FA) findings. The secondary efficacy endpoint is the proportion of participants with a ≥ 2-step improvement from baseline on the DRSS score as compared with Week 24.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Baseline to Week 28

Population: The study was terminated by the Sponsor. Only 3 participants were enrolled in this study. Based on the low enrollment number, no data is reported here in order to protect and maintain participant privacy/confidentiality.

The secondary efficacy endpoint is the proportion of participants progressing to the following vision-threatening complications that require treatment: CI-DME, PDR, and/or ASNV as assessed by spectral domain optical coherence tomography (SD-OCT), fundus photography (FP), and fluorescein angiography (FA), as appropriate. The central reading center must confirm progression to CI-DME and PDR before treatment is initiated; progression to ASNV, and subsequent treatment, does not require photo documentation.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Baseline to Week 28

Population: The study was terminated by the Sponsor. Only 3 participants were enrolled in this study. Based on the low enrollment number, no data is reported here in order to protect and maintain participant privacy/confidentiality.

The secondary efficacy endpoint is the time to the following vision-threatening event(s) that require treatment: CI-DME, PDR, and/or ASNV

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Baseline to Week 28

Population: The study was terminated early by the Sponsor. At the time of discontinuation, three participants were enrolled, and all participants were allocated to randomized treatment with AKST4290.

Safety was assessed based on the number of participants who reported adverse events of mild, moderate or severe intensities.

Outcome measures

Outcome measures
Measure
AKST4290
n=3 Participants
Subjects will receive AKST4290, 400mg twice daily, for 24 weeks AKST4290: Oral AKST4290
Placebo
Subjects will receive matching Placebo, twice daily, for 24 weeks Placebo: Oral Placebo
To Assess the Overall Safety of AKST429
AE Intensity - Mild
1 Participants
0 Participants
To Assess the Overall Safety of AKST429
AE Intensity - Moderate
2 Participants
0 Participants
To Assess the Overall Safety of AKST429
AE Intensity - Severe
0 Participants
0 Participants

SECONDARY outcome

Timeframe: Baseline to Week 28

Population: The study was terminated by the Sponsor. Only 3 participants were enrolled in this study. Based on the low enrollment number, no data is reported here in order to protect and maintain participant privacy/confidentiality.

The secondary efficacy endpoint is the effect of AKST4290 on diabetic kidney disease as assessed by changes in clinical laboratory values over time (eg, estimated glomerular filtration rate \[eGFR\], urine albumin to creatinine ratio \[UACR\]).

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Baseline to Week 24

Population: The study was terminated by the Sponsor. Only 3 participants were enrolled in this study. Based on the low enrollment number, no data is reported here in order to protect and maintain participant privacy/confidentiality.

The WPAI-GH V2.0 is a 6-question survey used to assess the effects of a participant's health problems (i.e., physical or emotional problems or symptoms) on their ability to work and perform regular activities during the past seven days. The WPAI-GH questions will be analyzed as impairment percentages, in which higher percentages indicate greater impairment and less productivity. The following parameters will be calculated (multiply scores by 100 to express in percentages): * Percent of work time missed due to health: Q2 divided by (Q2 plus Q4) * Percent of impairment while working due to health: Q5 divided by 10 * Percent of overall work impairment due to health: Q2 divided by (Q2 plus Q4) plus \[(1 - (Q2 divided by (Q2 plus Q4 ))) multiplied by (Q5 divided by 10)\] * Percent of activity impairment due to health: Q6 divided by 10

Outcome measures

Outcome data not reported

Adverse Events

AKST4290

Serious events: 0 serious events
Other events: 2 other events
Deaths: 0 deaths

Placebo

Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure
AKST4290
n=3 participants at risk
Subjects will receive AKST4290, 400mg twice daily, for 24 weeks AKST4290: Oral AKST4290
Placebo
Subjects will receive matching Placebo, twice daily, for 24 weeks Placebo: Oral Placebo
Metabolism and nutrition disorders
Diabetes Mellitus
33.3%
1/3 • Number of events 1 • The planned timeframe for adverse event (AE) collection was from the first dose of the study drug until the post-treatment follow-up period (baseline to Week 28). However, the study was terminated early by the sponsor. At the time of termination, 3 participants were enrolled and none completed the study. Enrolled subjects had a safety visit at 30 and 60 days following study drug discontinuation.
Three participants were enrolled and none completed the study. All participants were allocated to randomized treatment with AKST4290, and no participants received placebo.
0/0 • The planned timeframe for adverse event (AE) collection was from the first dose of the study drug until the post-treatment follow-up period (baseline to Week 28). However, the study was terminated early by the sponsor. At the time of termination, 3 participants were enrolled and none completed the study. Enrolled subjects had a safety visit at 30 and 60 days following study drug discontinuation.
Three participants were enrolled and none completed the study. All participants were allocated to randomized treatment with AKST4290, and no participants received placebo.
Eye disorders
Vitreous floaters
33.3%
1/3 • Number of events 1 • The planned timeframe for adverse event (AE) collection was from the first dose of the study drug until the post-treatment follow-up period (baseline to Week 28). However, the study was terminated early by the sponsor. At the time of termination, 3 participants were enrolled and none completed the study. Enrolled subjects had a safety visit at 30 and 60 days following study drug discontinuation.
Three participants were enrolled and none completed the study. All participants were allocated to randomized treatment with AKST4290, and no participants received placebo.
0/0 • The planned timeframe for adverse event (AE) collection was from the first dose of the study drug until the post-treatment follow-up period (baseline to Week 28). However, the study was terminated early by the sponsor. At the time of termination, 3 participants were enrolled and none completed the study. Enrolled subjects had a safety visit at 30 and 60 days following study drug discontinuation.
Three participants were enrolled and none completed the study. All participants were allocated to randomized treatment with AKST4290, and no participants received placebo.
Investigations
Activated partial thromboplastin time prolonged
33.3%
1/3 • Number of events 1 • The planned timeframe for adverse event (AE) collection was from the first dose of the study drug until the post-treatment follow-up period (baseline to Week 28). However, the study was terminated early by the sponsor. At the time of termination, 3 participants were enrolled and none completed the study. Enrolled subjects had a safety visit at 30 and 60 days following study drug discontinuation.
Three participants were enrolled and none completed the study. All participants were allocated to randomized treatment with AKST4290, and no participants received placebo.
0/0 • The planned timeframe for adverse event (AE) collection was from the first dose of the study drug until the post-treatment follow-up period (baseline to Week 28). However, the study was terminated early by the sponsor. At the time of termination, 3 participants were enrolled and none completed the study. Enrolled subjects had a safety visit at 30 and 60 days following study drug discontinuation.
Three participants were enrolled and none completed the study. All participants were allocated to randomized treatment with AKST4290, and no participants received placebo.

Additional Information

Head of Communications

Alkahest, Inc.

Phone: (650) 801-0474

Results disclosure agreements

  • Principal investigator is a sponsor employee
  • Publication restrictions are in place

Restriction type: LTE60