Study of the Safety and Efficacy of APX3330 in Diabetic Retinopathy

NCT ID: NCT04692688

Last Updated: 2023-02-27

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

103 participants

Study Classification

INTERVENTIONAL

Study Start Date

2021-04-08

Study Completion Date

2023-01-25

Brief Summary

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The objective of this study is to evaluate the safety and efficacy of APX3330 to treat diabetic retinopathy (DR) and diabetic macular edema (DME).

Detailed Description

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The objective of this study is to evaluate the efficacy of APX3330 to improve Early Treatment Diabetic Retinopathy Study (ETDRS) Diabetic Retinopathy Severity Score (DRSS) in one hundred (100) subjects with moderately severe to severe NPDR or mild PDR.

Subjects with moderately severe to severe NPDR and mild PDR will be selected for study participation and be screened for study eligibility.

The eligible eye with the highest DRSS, as assessed by the central reading center, will be designated as the study eye for the primary efficacy analysis.

If the subject meets all eligibility criteria, then the subject will be randomized into the study and receive study medication. Blood will be drawn for biomarker analysis.

The total length of subject participation is approximately 26 weeks, with 5 clinic visits, 4 telephone safety calls, and one telephone call follow-up visit.

The execution of the entire study (first subject screen through last randomized subject completed) is expected to be approximately 12 to 15 months.

Conditions

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Diabetic Retinopathy Diabetic Macular Edema NPDR - Non Proliferative Diabetic Retinopathy PDR - Proliferative Diabetic Retinopathy

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

This is a placebo-controlled, double-masked, randomized, Phase 2 study in approximately 100 subjects with moderately severe to severe non-proliferative diabetic retinopathy (NPDR), or mild proliferative diabetic retinopathy (PDR), evaluating safety and efficacy following administration of APX3330 twice daily for 24 weeks.

The study will have a 1:1 randomization (placebo: APX3330). Randomization will be stratified by level of disease severity (NPDR or PDR). Subjects with mild PDR will be capped at 20% for each arm.
Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors
Double-masked

Study Groups

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APX3330

Five 120 mg tablets will be taken by mouth as follows: 3 tablets every morning and 2 tablets every evening.

Group Type EXPERIMENTAL

APX3330

Intervention Type DRUG

APX3330, a small-molecule oral tablet, is a Ref-1 inhibitor that can potentially reduce proinflammatory and hypoxic signaling that contributes to several eye diseases.

Placebo

Five 120 mg tablets will be taken by mouth as follows: 3 tablets every morning and 2 tablets every evening.

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

Placebo tablets are identical to APX3330 tablets except for the absence of the active pharmaceutical ingredient.

Interventions

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APX3330

APX3330, a small-molecule oral tablet, is a Ref-1 inhibitor that can potentially reduce proinflammatory and hypoxic signaling that contributes to several eye diseases.

Intervention Type DRUG

Placebo

Placebo tablets are identical to APX3330 tablets except for the absence of the active pharmaceutical ingredient.

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

1. Males or non-pregnant females ≥ 18 years of age
2. At least one eye with DR graded at least moderately severe to severe NPDR or mild PDR (corresponding to DRSS 47, 53, or 61)
3. BCVA assessed by ETDRS protocol letters score of ≥ 60 letters (Snellen equivalent ≥ 20/63)
4. Body mass index (BMI) between 18 and 40 kg/m2, inclusive

Exclusion Criteria

Ophthalmic:

1. Any prior treatment in the study eye with:

1. Focal or grid laser photocoagulation within the past year or PRP at any time
2. Systemic or intravitreal anti-VEGF agents within the last 6 months
3. Intraocular steroids including triamcinolone and dexamethasone implant within the last 6 months
4. Fluocinolone implant within the last 3 years
2. Active uveitis, vitritis, or infection in either eye including infectious conjunctivitis, keratitis, scleritis, or endophthalmitis.
3. Ocular incisional surgery including cataract surgery in the study eye within 3 months.
4. Clinically significant ocular disease in either eye.
5. Presence of macular or retinal vascular disease including diabetic macular edema, retinopathy from causes other than diabetes, age-related macular degeneration, pattern dystrophy, choroidal neovascularization of any cause, retinal vein occlusion, retinal artery occlusion in the study eye.
6. History of retinal detachment, full-thickness macular hole in the study eye, or idiopathic or autoimmune uveitis in either eye.

Systemic:

1. Known hypersensitivity or contraindication to study drug.
2. Any disease or medical condition that in the opinion of the Investigator would interfere with the study, prevent the subject from successfully participating in the study, or which might confound the study results.
3. Participation in any investigational study within 30 days prior to screening or planning to participate in any other investigational drug or device clinical trials within 30 days of study completion.
4. Resting HR outside the specified range (50-110 beats per minute).
5. Known to be immunocompromised or receiving immunosuppressive therapy.
6. Hypertension with resting diastolic blood pressure (BP) \> 105 mmHg or systolic BP \> 200 mmHg.
7. History of chronic liver disease or presence of elevated alanine aminotransferase (ALT) and aspartate aminotransferase (AST) consistent with such diagnosis.
8. Women of childbearing potential who are pregnant, nursing, planning a pregnancy, or not using a medically acceptable form of birth control.
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

Yes

Sponsors

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Ocuphire Pharma, Inc.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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Clinical Site 9

Phoenix, Arizona, United States

Site Status

Clinical Site 8

Bakersfield, California, United States

Site Status

Clinical Site 5

Beverly Hills, California, United States

Site Status

Clinical Site 11

Palm Desert, California, United States

Site Status

Clinical Site 2

Sacramento, California, United States

Site Status

Clinical Site 24

Walnut Creek, California, United States

Site Status

Clinical Site 19

Miami, Florida, United States

Site Status

Clinical Site 7

Winter Haven, Florida, United States

Site Status

Clinical Site 6

Carmel, Indiana, United States

Site Status

Clinical Site 14

Hagerstown, Maryland, United States

Site Status

Clinical Site 22

Springfield, Massachusetts, United States

Site Status

Clinical Site 17

Grand Blanc, Michigan, United States

Site Status

Clinical Site 12

Albuquerque, New Mexico, United States

Site Status

Clinical Site 15

Shirley, New York, United States

Site Status

Clinical Site 20

Charlotte, North Carolina, United States

Site Status

Clinical Site 1

Rapid City, South Dakota, United States

Site Status

Clinical Site 18

Austin, Texas, United States

Site Status

Clinical Site 16

Bellaire, Texas, United States

Site Status

Clinical Site 10

Fort Worth, Texas, United States

Site Status

Clinical Site 4

McAllen, Texas, United States

Site Status

Clinical Site 3

San Antonio, Texas, United States

Site Status

Clinical Site 23

San Antonio, Texas, United States

Site Status

Clinical Site 13

Southlake, Texas, United States

Site Status

Clinical Site 21

Ogden, Utah, United States

Site Status

Countries

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United States

Other Identifiers

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OPI-APXDR-201 (ZETA-1)

Identifier Type: -

Identifier Source: org_study_id

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