Phase 2a, Random, double masked, Placebo-controlled, side by side group, multiple center study evaluating the effectiveness and safety of STN1010904 Ophthalmic Suspension 0.03% and 0.1% compared with Placebo in Subjects with Fuchs endothelial Corneal Dystrophy (FECD) - PHANTOM Study

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Santen Inc.

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Phenomena and Processes [G] - Ocular Physiological Phenomena [G14]

Trial duration

8 Jun 2023 → 26 May 2025

Decision date (initial)

2024-10-04

Transition trial

Yes

Low-intervention

No

Rare-disease indication

No

Vulnerable population

No

Funding sources

SANTEN INCORPORATED, USA

External identifiers

EU CT number

2024-517104-11-00

EudraCT number

2022-000174-25

ClinicalTrials.gov

NCT05376176

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Pharmacogenomic, Dose response, Safety, Efficacy, Therapy

Primary Efficacy Objective:
- To assess the efficacy of two concentrations of STN1010904 ophthalmic suspension (0.03%, and 0.1 %), twice daily dosing when compared to Placebo in subjects diagnosed with FECD

Safety Objective:
- To assess the safety of two concentrations of STN1010904 ophthalmic suspension (0.03%, and 0.1 %), twice daily dosing when compared to Placebo in subjects diagnosed with FECD

Secondary objectives 1

1. To assess the dose response of STN1010904 ophthalmic suspension.

Conditions and MedDRA coding

Fuchs Endothelial Corneal Dystrophy (FECD)

Study design 1 period

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 3

1. Subjects are eligible to be included in the study only if all the following criteria are met at Visit 1 (Screening): 1. Male or female, from 30 to 75 years of age, diagnosed with FECD. 2. Provide signed informed consent prior to any study procedures being performed. 3. Best-corrected visual acuity (BCVA) of +0.2 LogMAR or better (equivalent to ≥ 75 ETDRS letters, or at least Snellen 20/32) in the study eye as measured using an ETDRS chart. 4. Grade 3-5 of the Modified Krachmer scale in the study eye.
2. 5. At least two out of three tomographic features are observed by Pentacam™, evaluated by the Investigator in the study eye: Tomographic Features: Parameter: Loss of parallel isopachs Definition: Any single isopach not being almost circular/oval or parallel to adjacent isopachs within the central 4 mm of the cornea (relative to the pupil center) Parameter: Displacement of the thinnest point of the cornea Definition: Being located outside of the inferotemporal quadrant (centered at the pupil center) or more than 1 mm from the pupil center in any quadrant. Parameter: Focal posterior corneal depression Definition: Any isolated area of depression (negative elevation relative to a sphere with best fit zone of 8 mm with float function) within the central 4 mm of the cornea (relative to the pupil center)
3. 6. Endothelial cells are visible >50% of area in at least one image obtained by noncontact specular microscopy in the central or paracentral-peripheral area in the study eye.

Exclusion criteria 2

1. Ocular Conditions: 1. Monocular vision, or vision worse than +0.7 LogMAR (equivalent to < 50 ETDRS letters, or worse than Snellen 20/100) in the fellow eye; 2. No guttae in either eye (evaluated by slit-lamp and specular microscope). 3. Clinically evident stromal and/or epithelial edema in the study eye (evaluated by slit-lamp microscope). 4. Descemet folds in the study eye (evaluated by slit-lamp microscope). 5. Central cornea thickness is 630μm or more in the study eye (evaluated by Scheimpflug image). 6. Moderate/severe cataract in the study eye (mild cataract or pseudophakic eye is eligible for enrolment). 7. Evidence of any other ocular disease other than FECD in the study eye that may confound the outcome of the study (e.g., active diabetic retinopathy, posterior uveitis, age-related macular degeneration or any other maculopathy, severe myopia >10D, pterygium).
2. Non-Ocular Conditions: 21. Allergy or hypersensitivity to study drug product, fluorescein dye, or other study related procedures/medications. Current or planned participation in any other clinical study involving an investigational product or device within 4 weeks prior to Visit 1 or at any time during this study. 22. History of any disease or condition that in the opinion of the study investigator may put the subject at significant risk, may confound study results, or may interfere significantly with the subject's participation in the study (e.g., recurrent corneal erosion syndrome, uncontrolled cardiovascular disease etc.).

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 2

1. Primary Efficacy Endpoints: - Change from baseline in best corrected visual acuity (BCVA) with contrast level of 100% at Month 18 - Change from baseline in BCVA with contrast level of 10% at Month 18 - Change from baseline in contrast sensitivity with glare light at Month 18
2. Safety Endpoint: - Safety of DE-109D will be assessed by adverse events (AEs), slit-lamp biomicroscopy, indirect ophthalmoscopy, intraocular pressure (IOP), and laboratory tests (serum chemistry, hematology, and urinalysis).

Secondary endpoints 4

1. Secondary Efficacy Endpoints: - Best corrected visual acuity (BCVA) with contrast level of 100% at all postbaseline visits - BCVA with contrast level of 10% at all post-baseline visits - Contrast sensitivity with glare light at all post-baseline visits - Contrast sensitivity without glare light at all post-baseline visits.
2. - Change and percent change from baseline in BCVA with contrast level of 100% at all post-baseline visits - Change and percent change from baseline in BCVA with contrast level of 10% at all post-baseline visits - Change and percent change from baseline in contrast sensitivity with glare light at all post-baseline visits
3. - Change and percent change from baseline in contrast sensitivity without glare light at all post-baseline visits - Change and percent change from baseline in central corneal thickness at all post-baseline visits. - Change and percent change from baseline in endothelial cell density at all post-baseline visits.
4. - Change and percent change in Guttae formation (Modified Krachmer scale) at all post-baseline visits

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 2

Placebo 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Santen Inc.

Sponsor organisation

Santen Inc.

Address

6401 Hollis Street Suite 125

City

Emeryville

Postcode

94608-1462

Country

United States

Scientific contact point

Organisation

Santen Inc.

Contact name

Sushil Panda

Public contact point

Organisation

Santen Inc.

Contact name

Robert Hostutler

Third parties 2

Locations

1 EU/EEA country · 2 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Summary of results Art. 37(4) CTR

Layperson summary Annex V

Documents 11 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

2 submissions — initial application plus substantial / non-substantial modifications