A Safety and Tolerability Clinical Trial of PST-611 in Dry Age-related Macular Degeneration

NCT07024732 | Not Yet Recruiting Phase 1

• 2 other identifiers: PST-611-CT12024-519025-37-00
• Study Type: interventional
• Target: 12
• Locations: 1 country
• Sites: 2

Brief Summary

The goal of this interventional study is to evaluate the safety and tolerability of single ascending doses of PST-611 in men and women over the age of 50 with dry age-related macular degeneration (AMD). The main question it aims to answer is: Is PST-611-CT1 safe for participants? Participants will:

• Receive a single dose of PST-611
• Will be followed up for a total of 16 weeks following PST-611 administration

Conditions

Dry Age Related Macular Degeneration

Keywords

Dry Age Related Macular Degeneration; PST-611

Outcome Measures

Primary Outcomes (1)

• Safety and Tolerability

  Ocular and non-ocular adverse events frequency and severity

  Screening to week 16

Secondary Outcomes (6)

• Intraocular pressure

  Screening to Week 16

• Best corrected visual acuity

  Screening to Week 16

• Slit lamp biomicroscopy examination

  Screening to Week 16

• Dilated ophthalmoscopy examination

  Screening to Week 16

• Color fundus photography

  Screening to Week 16

Study Arms (1)

Active treatment

EXPERIMENTAL

This arm consists of 2 dose groups receiving a single ascending dose of PST-611. These groups will be dosed sequentially, low dose followed by high dose.

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: 50 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), 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 50 years of age or older. Female subjects of childbearing potential must not be pregnant or breast-feeding and must have a negative urine pregnancy test at baseline and throughout the study. They must agree to practice at least one effective method of birth control following administration of study medication.
• In the study eye, at least one of the following must be present at OCT and attributed to AMD, as evaluated by the Investigator: Incomplete RPE and Outer Retinal Atrophy (iRORA), or Complete RPE and Outer Retinal Atrophy (cRORA).
• Best-Corrected Visual Acuity (BCVA), must be 23 ETDRS letters (approximate Snellen equivalent 20/320) or better in the study eye.
• Subject's fellow eye BCVA must be 34 letters (approximate Snellen equivalent 20/200) or better.

You may not qualify if:

• Both eyes: 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 the PST-611 administration.
• Study eye: Any intraocular surgery (including cataract surgery) or intravitreal (IVT) or periocular corticosteroid injection within 12 weeks (84 days) prior to the PST-611 administration.
• Study eye: Any anti-VEGF IVT treatment within 4 weeks (28 days) prior to PST-611 dosing OR subjects who have required and received regular monthly injections of anti-VEGF drugs in the months preceding the trial and would thus have a higher likelihood of requiring and anti-VEGF treatment within 28 days of the PST-611 administration.
• Study eye: media opacity that interferes with fundus imaging or is likely to require surgery during the trial period.
• Study eye: subject with history of glaucoma filtering surgery (e.g. trabeculectomy or aqueous shunt implant) or who underwent eye surgery within 12 weeks (84 days) of the PST-611 administration. Study eye: subject who has uncontrolled intraocular pressure of ≥ 25 mmHg in the SE at the screening and baseline visits.

Sponsors & Collaborators

• Eyevensys: lead

Study Sites (2)

CHU de Grenoble-Hôpital Michallon

Grenoble, 38043, France

Location

Hôpital Cochin

Paris, 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 RESULT

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 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Single ascending dose design with 2 sequential dose groups (low dose group followed by high dose group)

Study Record Dates

• First Submitted: June 5, 2025
• First Posted: June 17, 2025
• Study Start: July 1, 2025
• Primary Completion: December 1, 2025
• Study Completion (Estimated): June 1, 2026
• Last Updated: June 17, 2025

Record last verified: 2025-06

Data Sharing

• IPD Sharing: Will not share

Locations

FR (2)