Study of the Safety and Efficacy of APX3330 in Diabetic Retinopathy

NCT04692688 | Completed Phase 2 DMC FDA Drug

• 1 other identifier: OPI-APXDR-201 (ZETA-1)
• Study Type: interventional
• Target: 103
• Locations: 1 country
• Sites: 24

Brief Summary

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

Conditions

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

Keywords

diabetes; diabetic retinopathy; NPDR; PDR

Outcome Measures

Primary Outcomes (1)

• Percent of Subjects with an improvement in Diabetic Retinopathy Severity Score (DRSS)

  Percent of subjects with a ≥ 2-step improvement in DRSS in the study eye

  24 Weeks

Secondary Outcomes (5)

• Percent of Subjects with change in Diabetic Retinopathy Severity Scale (DRSS) Scores

  Up to 24 Weeks

• Mean Change in Diabetic Retinopathy Severity Scale (DRSS) Score

  24 Weeks

• Percent of Subjects without DR/DME Disease Progression

  24 Weeks

• Mean Change in Best-Corrected Visual Acuity (BCVA)

  24 Weeks

• Mean Change in Central Subfield Thickness (CST)

  24 Weeks

Study Arms (2)

APX3330

EXPERIMENTAL

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

Drug: APX3330

Placebo

PLACEBO COMPARATOR

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

Drug: Placebo

Interventions

APX3330 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.

APX3330

Placebo DRUG

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

Placebo

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

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

You may not qualify if:

• Ophthalmic:
• Any prior treatment in the study eye with:
• Focal or grid laser photocoagulation within the past year or PRP at any time
• Systemic or intravitreal anti-VEGF agents within the last 6 months
• Intraocular steroids including triamcinolone and dexamethasone implant within the last 6 months
• Fluocinolone implant within the last 3 years
• Active uveitis, vitritis, or infection in either eye including infectious conjunctivitis, keratitis, scleritis, or endophthalmitis.
• Ocular incisional surgery including cataract surgery in the study eye within 3 months.
• Clinically significant ocular disease in either eye.
• 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.
• History of retinal detachment, full-thickness macular hole in the study eye, or idiopathic or autoimmune uveitis in either eye.
• Systemic:
• Known hypersensitivity or contraindication to study drug.
• 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.
• 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.

Sponsors & Collaborators

• Ocuphire Pharma, Inc.: lead

Study Sites (24)

Clinical Site 9

Phoenix, Arizona, 85014, United States

Location

Clinical Site 8

Bakersfield, California, 93309, United States

Location

Clinical Site 5

Beverly Hills, California, 91607, United States

Location

Clinical Site 11

Palm Desert, California, 92260, United States

Location

Clinical Site 2

Sacramento, California, 95825, United States

Location

Clinical Site 24

Walnut Creek, California, 94598, United States

Location

Clinical Site 19

Miami, Florida, 33143, United States

Location

Clinical Site 7

Winter Haven, Florida, 33880, United States

Location

Clinical Site 6

Carmel, Indiana, 46290, United States

Location

Clinical Site 14

Hagerstown, Maryland, 21740, United States

Location

Clinical Site 22

Springfield, Massachusetts, 01107, United States

Location

Clinical Site 17

Grand Blanc, Michigan, 48439, United States

Location

Clinical Site 12

Albuquerque, New Mexico, 87113, United States

Location

Clinical Site 15

Shirley, New York, 11967, United States

Location

Clinical Site 20

Charlotte, North Carolina, 28210, United States

Location

Clinical Site 1

Rapid City, South Dakota, 57701, United States

Location

Clinical Site 18

Austin, Texas, 78705, United States

Location

Clinical Site 16

Bellaire, Texas, 77030, United States

Location

Clinical Site 10

Fort Worth, Texas, 76104, United States

Location

Clinical Site 4

McAllen, Texas, 78550, United States

Location

Clinical Site 3

San Antonio, Texas, 78240, United States

Location

Clinical Site 23

San Antonio, Texas, 78624, United States

Location

Clinical Site 13

Southlake, Texas, 76092, United States

Location

Clinical Site 21

Ogden, Utah, 84010, United States

Location

MeSH Terms

Conditions

Diabetic Retinopathy; Diabetes Mellitus

Condition Hierarchy (Ancestors)

Retinal Diseases; Eye Diseases; Diabetic Angiopathies; Vascular Diseases; Cardiovascular Diseases; Diabetes Complications; Endocrine System Diseases; Glucose Metabolism Disorders; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Masking Details: Double-masked
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: 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.

Study Record Dates

• First Submitted: December 29, 2020
• First Posted: January 5, 2021
• Study Start: April 8, 2021
• Primary Completion: January 25, 2023
• Study Completion: January 25, 2023
• Last Updated: February 27, 2023

Record last verified: 2023-02

Locations

US (24)