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| ID | Type | Description | Link |
|---|---|---|---|
| 2025-525008-12-00 | EU Trial (CTIS) Number |
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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:
The maximum study duration per patient is 64 Weeks (including an up to 12 week screening/baseline period + 52 weeks of follow-up after the first dose). The study is a multiple dose study that investigates one PST-611 dose level for the 3 planned administrations. The study will enroll up to 24 participants.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| 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). |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| PST-611 | Biological | PST-611 is a naked plasmid DNA encoding human transferrin administered into the ciliary muscle |
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| Measure | Description | Time Frame |
|---|---|---|
| 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 |
| Measure | Description | Time Frame |
|---|---|---|
| Intraocular pressure | Intraocular pressure measured in mmHg | Screening to week 52 |
| Best corrected visual acuity | Best corrected visual acuity measured in ETDRS letters |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Karine Bigot, PhD | Contact | +33 (0)1 84 79 10 62 | karine.bigot@pulsesight.com |
| Name | Affiliation | Role |
|---|---|---|
| Karine Bigot, PhD | PulseSight Therapeutics | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| CHU de Grenoble-Hôpital Michallon | Grenoble | France | 38043 | France |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 32882879 | Background | 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. |
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| ID | Term |
|---|---|
| D057092 | Geographic Atrophy |
| D008268 | Macular Degeneration |
| ID | Term |
|---|---|
| D012162 | Retinal Degeneration |
| D012164 | Retinal Diseases |
| D005128 | Eye Diseases |
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Parallel enrolment
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| Screening to week 52 |
| Slit lamp biomicroscopy examination | Changes over time will be recorded | Screening to week 52 |
| Dilated ophthalmoscopy examination | Changes over time will be recorded | Screening to week 52 |
| Color fundus photography | Changes over time will be recorded | Screening to week 52 |
| Spectral Domain-Optical Coherence Tomography | Changes over time will be recorded | Screening to week 52 |
| Quantitative contrast sensitivity function | Changes over time will be recorded | screening to week 52 |
| Area of retinal pigment epithelium (RPE) loss | Changes over time will be recorded. Area of RPE loss will be assessed by deep learning-based analysis of SD-OCT | screening to week 52 |
| Area of photoreceptor degeneration (EZ layer loss) | Changes over time will be recorded. EZ layer loss will be assessed by deep learning-based analysis of SD-OCT | Screening to week 52 |
| Hôpital Lariboisière | Paris | France | 75010 | France |
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| Hôpital Cochin | Paris | France | 75014 | France |
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