PER-001 for Diabetic Retinopathy

Overseen By Andrew Melie

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: Perfuse Therapeutics, Inc.

Must not be taking: Systemic anti-VEGF

Disqualifiers: Uncontrolled diabetes, Active cancer, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

Duration: 24 weeks

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment called PER-001 for diabetic retinopathy, a condition where diabetes affects the eyes. The study aims to evaluate the effectiveness and safety of PER-001 in improving vision. Participants will receive varying doses of the treatment, while some will receive a "sham" or placebo treatment for comparison. Individuals with moderately severe diabetic eye disease and adequate vision may qualify for this trial. As a Phase 2 trial, this research focuses on assessing the treatment's effectiveness in an initial, smaller group of participants.

Will I have to stop taking my current medications?

The trial does not specify if you need to stop taking your current medications. However, if you are on systemic anti-VEGF treatment, you must have stopped it at least 4 months before starting the trial.

Is there any evidence suggesting that PER-001 is likely to be safe for humans?

Research has shown that PER-001 is generally safe and well-tolerated for people with diabetic retinopathy. In earlier studies, participants reported only mild and temporary side effects, such as vitreous floaters, which appear as small specks or clouds in vision. Beyond this, the treatment did not cause significant issues. Additionally, data from 24 weeks of use indicated that PER-001 was well-tolerated, with no serious safety concerns. These findings suggest that PER-001 is a promising option with a good safety record for treating eye conditions like diabetic retinopathy. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for diabetic retinopathy?

Most treatments for diabetic retinopathy, like anti-VEGF injections, work by slowing down or stopping abnormal blood vessel growth in the retina. PER-001 is unique because it may offer a new mechanism of action by targeting alternative pathways involved in retinal damage. Researchers are excited about PER-001 because it could provide a more effective or faster-acting solution, potentially reducing the frequency of injections or enhancing the overall treatment outcomes for patients with diabetic retinopathy.

What evidence suggests that PER-001 might be an effective treatment for diabetic retinopathy?

Research shows that PER-001 may help treat diabetic retinopathy. Studies have found that it improved eye health and vision in patients and enhanced blood flow in the eye, reducing macular ischemia. Importantly, the treatment was safe and well-tolerated, with no serious side effects reported. In this trial, participants will receive either a low dose or high dose of PER-001, or a sham treatment. PER-001 is being considered as a treatment that might change the course of diabetic retinopathy, not just manage its symptoms. ^{1 2 3 6 7}

Are You a Good Fit for This Trial?

This trial is for adults over 18 with moderate to severe non-proliferative diabetic retinopathy, vision of 20/63 or better, and no recent serious health issues like cancer or heart attack. Pregnant women and those with uncontrolled diabetes, blood pressure, eye infections/inflammation, glaucoma, or a history of certain eye surgeries are excluded.

Inclusion Criteria

My diabetic eye condition is moderately severe to severe.

My vision is 20/63 or better with correction in the eye(s) that may be in the study.

Women who could become pregnant must have a negative pregnancy test before starting the study.

Exclusion Criteria

I had cataract surgery or YAG procedure recently.

I have a specific eye condition affecting my retina or macula.

I have atrial fibrillation that is not well-managed.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive a single administration of PER-001 Intravitreal Implant or sham control

24 weeks

Multiple visits for monitoring

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• PER-001

Trial Overview The study tests PER-001 Intravitreal Implant at two different doses against a sham procedure in people with diabetic retinopathy. It's randomized and single-masked meaning participants don't know if they're getting the real treatment or the sham.

How Is the Trial Designed?

Group I: Phase 2 Cohort B Experimental Treatment 2 Interventions

Cohort B - High Dose or Sham

Group II: Phase 2 Cohort A Experimental Treatment 2 Interventions

Cohort A - Low Dose or Sham

Who Is Running the Clinical Trial?

Perfuse Therapeutics, Inc.

Lead Sponsor

Trials

2

Recruited

60+

Citations

1. modernretina.com modernretina.com/view/asrs-2025-per-001-improved-structure-and-visual-function-in-patients-with-diabetic-retinopathy

ASRS 2025: PER-001 improved structure and visual ... ASRS 2025: PER-001 improved structure and visual function in patients with diabetic retinopathy · Macular ischemia reduction · Decreased macular ...

2. europe.ophthalmologytimes.com europe.ophthalmologytimes.com/view/perfuse-therapeutics-shares-phase-2-clinical-findings-from-glaucoma-and-diabetic-retinopathy-trials-of-per-001

Perfuse Therapeutics shares findings from glaucoma and ... The 24-week data demonstrated that PER-001 was safe and well-tolerated in both trials.

3. clinicaltrials.gov clinicaltrials.gov/study/NCT06003751

A Study of PER-001 in Participants With Diabetic Retinopathy Best-corrected visual acuity (BCVA) of ≥ 60 Early Treatment Diabetic Retinopathy Study (ETDRS) letters (Snellen equivalent of 20/63 or better) at Screening in ...

4. perfusetherapeutics.com perfusetherapeutics.com/perfuse-therapeutics-announces-positive-results-from-phase-2-clinical-trials-in-glaucoma-and-diabetic-retinopathy-patients/

Perfuse Therapeutics Announces Positive Results from ... PER-001 intravitreal implant shows potential to be the first disease-modifying treatment for both glaucoma and diabetic retinopathy ...

5. hcplive.com hcplive.com/view/per-001-implant-safely-improves-ocular-health-in-patients-with-dr

PER-001 Implant Safely Improves Ocular Health in Patients ... PER-001, a sustained-release intravitreal implant, showed safety and efficacy in diabetic retinopathy, with no serious adverse events reported.

6. prnewswire.com prnewswire.com/news-releases/perfuse-therapeutics-announces-positive-visual-structural-and-safety-data-from-a-ph12a-trial-of-per-001-in-glaucoma-patients-at-the-american-glaucoma-society-meeting-302387598.html

Perfuse Therapeutics Announces Positive Visual ... The 24-week data demonstrated that PER-001 was well-tolerated, with only one drug-related adverse event of vitreous floater (mild and transient) ...

7. clinicaltrials.gov clinicaltrials.gov/study/NCT05822245

NCT05822245 | A Study of PER-001 in Participants With ... Phase 2a is a randomized, single-masked (participant) study to further investigate the ocular and systemic safety and tolerability of the two dose levels of ...