A Study of PER-001 in Participants With Open-Angle Glaucoma

A Phase 1/2a Study to Evaluate Safety, Tolerability, and Pharmacodynamic Effect After Single Administration of PER-001 Intravitreal Implant in Participants With Open-Angle Glaucoma

This clinical study has two phases: Phase 1 and Phase 2a. Phase 1 is an open-label, single ascending dose (SAD), while Phase 2a is a randomized, single-masked (participant) with a sham control.

Study Overview

• Status: Active, not recruiting
• Conditions: Open-angle Glaucoma
• Intervention / Treatment: Drug: PER-001 Intravitreal Implant - Low Dose; Drug: PER-001 Intravitreal Implant - High Dose; Drug: PER-001 Intravitreal Implant - Sham

Detailed Description

This clinical study has two phases: Phase 1 and Phase 2a. Phase 1 is an open-label, SAD study to investigate the ocular and systemic safety and tolerability of two dose levels of PER-001, in participants with advanced OAG. Phase 1 will enroll up to approximately 12 participants.

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 PER-001 Intravitreal Implants in participants with progressing glaucoma. Participants who meet entry criteria will be randomized to receive either dose of PER-001 or sham control. In Phase 2a, a total of approximately 24 participants (12 in each Cohort, will be randomized).

• Study Type: Interventional
• Enrollment (Estimated): 36
• Phase: Phase 2; Phase 1

Contacts and Locations

Study Locations

• United States
  • California
    • San Francisco, California, United States, 94158
      • Perfuse Therapeutics, Inc.

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

For Phase 1 and Phase 2a:

• Must be ≥ 18 years of age at the time of signing the informed consent
• A negative pregnancy test for females of childbearing potential at Screening (serum) and Day 1 (urine).
• IOP 6 to 25 mmHg (inclusive) at Screening in potentially eligible eye(s)

Phase 1:

• Best corrected visual acuity (BCVA) LogMAR score 1.0 or better at Screening in at least one eye and prior to randomization at Day 1 in the study eye
• Diagnosis of advanced or severe primary OAG including normal tension, pseudoexfoliation and pigment dispersion

Phase 2:

• BCVA LogMAR score 0.5 or better at Screening in at least one eye and prior to randomization at Day 1 in the study eye
• Primary OAG that is progressing in the study eye

Exclusion Criteria:

• Blood pressure >140/90 mmHg or <90/60 mmHg at Screening
• Any condition which, in the opinion of the investigator, would preclude the participant's ability to comply with study requirements including completion of the study (including but not limited to diagnosis of dementia, Alzheimer's, and/or other neurological disease or physical incapacity)
• Females who are pregnant, nursing, or planning a pregnancy during the study
• Any significant media opacity which precludes clinical evaluation and imaging of the retina
• History of vitrectomy surgery or retinal detachment or macular hole (Stage 3 or 4)
• Retinal laser within 3 months prior to Day 1
• Intraocular surgery, including cataract surgery and Minimally Invasive Glaucoma
• Surgery (MIGS), within 3 months prior to Day 1
• Aphakia or absence of posterior capsule
• Change in IOP lowering therapy within 6 weeks prior to Screening and/or anticipated change in IOP lowering therapy or treatment during the study Worse than mild non-proliferative diabetic retinopathy (Note: stable mild background diabetic retinopathy is permitted)
• Any active uveitis and/or vitritis or history of idiopathic or autoimmune-associated uveitis
• Active infectious conjunctivitis, keratitis, scleritis, or endophthalmitis (Note: mild blepharitis is permitted if stable)
• History of recurrent infectious or inflammatory ocular disease
• Central serous retinopathy
• Non-glaucomatous optic neuropathy

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Sequential Assignment
• Masking: Single

Number of Arms

4

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Phase 1 Cohort A; Cohort A - Low Dose	Drug: PER-001 Intravitreal Implant - Low Dose; PER-001 Low Dose Intravitreal Implant
Experimental: Phase 1 Cohort B; Cohort B - High Dose	Drug: PER-001 Intravitreal Implant - High Dose; PER-001 High Dose Intravitreal Implant
Experimental: Phase 2 Cohort C; Cohort C - Low Dose or Sham	Drug: PER-001 Intravitreal Implant - Low Dose; PER-001 Low Dose Intravitreal Implant; Drug: PER-001 Intravitreal Implant - Sham; PER-001 Intravitreal Sham
Experimental: Phase 2 Cohort D; Cohort D - High Dose or Sham	Drug: PER-001 Intravitreal Implant - High Dose; PER-001 High Dose Intravitreal Implant; Drug: PER-001 Intravitreal Implant - Sham; PER-001 Intravitreal Sham

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Ocular Measurement for Safety - Change from Baseline at Week 24 using Best-corrected Visual Acuity (BCVA)	ETDRS or modified ETDRS chart (Charts 1, 2, and R as applicable) with standardized distance and lighting will be used to calculate the LogMAR score	End of Study(Week 24)
Ocular Measurement for Safety - Change from Baseline at Week 24 using Biomicroscopy	Slit lamp examinations will include evaluation of the lids, conjunctiva, cornea, anterior chamber, iris/pupil, and lens.	End of Study(Week 24)
Ocular Measurement for Safety - Change from Baseline at Week 24 using Intraocular Pressure (IOP)	IOP is measured by a calibrated Goldmann applanation tonometry.	End of Study(Week 24)
Ocular Measurement and Implant Assessment for Safety - Change from Baseline at Week 24 using Dilated Ophthalmoscopy	Dilated ophthalmoscopy examination will include evaluation of the vitreous, macula, retinal vessels, peripheral retina, and optic disc.	End of Study(Week 24)

Collaborators and Investigators

• Sponsor: Perfuse Therapeutics, Inc.
• Investigators: Study Director: Phillip Lai, MD, Perfuse Therapeutics, Inc.

Study record dates

Study Major Dates

• Study Start (Actual): May 25, 2023
• Primary Completion (Actual): April 4, 2025
• Study Completion (Estimated): April 4, 2026

Study Registration Dates

• First Submitted: March 27, 2023
• First Submitted That Met QC Criteria: April 7, 2023
• First Posted (Actual): April 20, 2023

Study Record Updates

• Last Update Posted (Actual): April 15, 2025
• Last Update Submitted That Met QC Criteria: April 10, 2025
• Last Verified: October 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Eye Diseases; Ocular Hypertension; Glaucoma; Glaucoma, Open-Angle
• Other Study ID Numbers: PER001-201

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No