An Open-label Safety, Tolerability and Exploratory Efficacy Clinical Trial of PST-611 in Geographic Atrophy

NCT07705568 | Not Yet Recruiting Phase 2

• 2 other identifiers: PST-611-CT22025-525008-12-00
• Study Type: interventional
• Target: 24
• Locations: 1 country
• Sites: 3

Brief Summary

The goals of this interventional study are (1) to evaluate the safety and tolerability of multiple doses of PST-611 in men and women over the age of 65 with geographic atrophy secondary to age-related macular degeneration and (2) to assess efficacy of multiple doses of PST-611 by assessing retinal morphology and visual function changes over time. Participants will:

• receive one dose every 20 weeks, for a total of 3 doses.
• will be followed up for a total of 52 weeks following the first PST-611 dose.

Conditions

Geographic Atrophy; Age - Related Macular Degeneration (AMD)

Keywords

Geographic atrophy; Age related macular degeneration; PST-611

Outcome Measures

Primary Outcomes (1)

• Frequency and severity of ocular and non-ocular adverse events (Safety and Tolerability)

  Ocular and non-ocular adverse events over time will be recorded

  Screening to week 52

Secondary Outcomes (9)

• Intraocular pressure

  Screening to week 52

• Best corrected visual acuity

  Screening to week 52

• Slit lamp biomicroscopy examination

  Screening to week 52

• Dilated ophthalmoscopy examination

  Screening to week 52

• Color fundus photography

  Screening to week 52

Study Arms (1)

Active treatment

EXPERIMENTAL

This arm consists of 1 dose group receiving multiple dose of PST-611 (one PST-611 dose level for the 3 planned administrations).

Biological: PST-611

Interventions

PST-611 BIOLOGICAL

PST-611 is a naked plasmid DNA encoding human transferrin administered into the ciliary muscle

Active treatment

Eligibility Criteria

• Age: 65 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Older Adult (65+)

You may qualify if:

• Subjects must give written informed consent, be able to make the required trial visits and follow instructions.
• Female and male subjects must be 65 years of age or older.
• In the study eye (SE): cRORA must be present on OCT and attributed to AMD, as evaluated by the Investigator.
• Documented recent history (i.e., over 3 to 12 months prior to first PST-611 administration) of GA lesion area progression, with a yearly growth projection of ≥ 1.6 mm2, in the SE
• GA lesion area prior to the first PST-611 administration must be between 1.25 mm2 and 16 mm2, as assessed by OCT, in SE.
• BCVA must be \< or = 75 ETDRS letters (Snellen \< or = 20/32) in the SE.
• Fixation, either central or eccentric, of both eyes, must be compatible with the performance of the ocular imaging and functional assessments included in the trial, as evaluated by the Investigator.
• If both eyes are eligible, the SE will be selected by the Investigator based on the totality of the clinical evaluation.

You may not qualify if:

• Both eyes (OU): any active intraocular or periocular infection or inflammation (eg, infectious blepharitis, infectious conjunctivitis, keratitis, scleritis, endophthalmitis), or history of intraocular or periocular infection or inflammation in the 12 weeks (84 days) prior to Dose 1.
• SE: any intraocular surgery (including cataract surgery) or intravitreal (IVT) or periocular corticosteroid injection in the 12 weeks (84 days) prior to Dose 1.
• SE: the documented need for more than 2 anti-VEGF IVT treatments per year as well as any anti-VEGF IVT treatment within 3 months prior to Dose 1, and lesion must be clinically inactive.
• SE: media opacity that interferes with fundus imaging or is likely to require surgery during the trial period.
• SE: subject with history of glaucoma filtering surgery (e.g., trabeculectomy or aqueous shunt implant) or who underwent eye surgery in the 12 weeks (84 days) prior to Dose 1.
• SE: subject who has uncontrolled intraocular pressure of ≥ 25 mmHg in the SE at the Screening and Trial Baseline Visits.
• SE: subject with intraocular hypotension (\<6 mmHg) in the SE that in the opinion of the Investigator would interfere with the PST-611 administrations or the evaluation of its safety or efficacy.
• SE: subject with history of scleritis, scleral thinning, cicatrizing conjunctival diseases, severe ocular allergies, severe ocular surface disease, ocular scarring or intraocular hardware (e.g., retained implant device) that could interfere with the PST-611 administrations or the evaluation of its safety.
• SE: any other concurrent ocular surface or intra-ocular condition, including retinal disease other than AMD, which, in the opinion of the Investigator, may pose a safety risk for the PST-611 administrations or interfere with the evaluation of its safety.
• Subject has any condition, which in the opinion of the Investigator, could compromise the subject's safety or adherence to the trial protocol or follow-up.
• Known/suspected hypersensitivity to any standard of care topical or local analgesics/anesthetics or other standard of care treatments used in the preparation of PST-611 administration procedure.
• Treatment with investigational medicinal products in the 12 weeks (84 days) prior to Dose 1.
• Subject previously exposed to any gene therapy product other than the non-viral gene therapy PST-611.

Sponsors & Collaborators

• Eyevensys: lead

Study Sites (3)

CHU de Grenoble-Hôpital Michallon

Grenoble, France, 38043, France

Location

Hôpital Lariboisière

Paris, France, 75010, France

Location

Hôpital Cochin

Paris, France, 75014, France

Location

Related Publications (1)

• Bigot K, Gondouin P, Benard R, Montagne P, Youale J, Piazza M, Picard E, Bordet T, Behar-Cohen F. Transferrin Non-Viral Gene Therapy for Treatment of Retinal Degeneration. Pharmaceutics. 2020 Sep 1;12(9):836. doi: 10.3390/pharmaceutics12090836.

  PMID: 32882879 BACKGROUND

MeSH Terms

Conditions

Geographic Atrophy; Macular Degeneration

Condition Hierarchy (Ancestors)

Retinal Degeneration; Retinal Diseases; Eye Diseases

Study Officials

• Karine Bigot, PhD

  PulseSight Therapeutics

  STUDY DIRECTOR

Central Study Contacts

Karine Bigot, PhD

CONTACT

+33 (0)1 84 79 10 62; karine.bigot@pulsesight.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Parallel enrolment

Study Record Dates

• First Submitted: July 10, 2026
• First Posted: July 15, 2026
• Study Start (Estimated): September 1, 2026
• Primary Completion (Estimated): June 1, 2028
• Study Completion (Estimated): June 1, 2028
• Last Updated: July 16, 2026

Record last verified: 2026-07

Data Sharing

• IPD Sharing: Will not share

Locations

FR (3)