Clinical trial NCT07024732: A Safety and Tolerability Clinical Trial of PST-611 in Dry Age-related Macular Degeneration
Official title: An Open-label Single Ascending Dose Safety and Tolerability Clinical Trial of PST-611 in Subjects With Dry Age-related Macular Degeneration
Study type: INTERVENTIONAL
Overall status: NOT_YET_RECRUITING
Phase: PHASE1
Conditions: Dry Age Related Macular Degeneration
Keywords: Dry Age Related Macular Degeneration
PST-611
Lead sponsor: Eyevensys
Sponsor class: INDUSTRY
First posted: 2025-06-17
Enrollment: 12
Sex: ALL
Minimum age (years): 50
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SEQUENTIAL
Intervention model description: Single ascending dose design with 2 sequential dose groups (low dose group followed by high dose group)
Primary purpose: TREATMENT

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

Detailed description: The maximum study duration per patient is 28 Weeks (including an up to 12 week screening period + 16 weeks of follow-up after treatment).

The study is a single ascending dose study that investigates two PST-611 dose levels (low and high doses) in 2 successive dose groups. The study will enroll up to 12 participants.

Eligibility criteria: Inclusion Criteria:

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.

Exclusion Criteria:

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.

Primary outcomes:
measure: Safety and Tolerability
timeFrame: Screening to week 16
Secondary outcomes:
measure: Intraocular pressure
timeFrame: Screening to Week 16
measure: Best corrected visual acuity
timeFrame: Screening to Week 16
measure: Slit lamp biomicroscopy examination
timeFrame: Screening to Week 16
measure: Dilated ophthalmoscopy examination
timeFrame: Screening to Week 16
measure: Color fundus photography
timeFrame: Screening to Week 16
measure: Spectral Domain-Optical Coherence Tomography
timeFrame: Screening to Week 16
Other outcomes:
Locations:
city: Grenoble
country: France
facility: CHU de Grenoble-Hôpital Michallon
city: Paris
country: France
facility: Hôpital Cochin
Study officials:
affiliation: PulseSight Therapeutics
name: Karine Bigot, PhD
role: STUDY_DIRECTOR
References:
citation: 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.