Clinical Trials Register

Summary
EudraCT Number:	2022-000174-25
Sponsor's Protocol Code Number:	101090401IN
National Competent Authority:	France - ANSM
Clinical Trial Type:	EEA CTA
Trial Status:	Ongoing
Date on which this record was first entered in the EudraCT database:	2022-08-16
Trial results

Index
A. PROTOCOL INFORMATION
B. SPONSOR INFORMATION
C. APPLICANT IDENTIFICATION
D. IMP IDENTIFICATION
D.8 INFORMATION ON PLACEBO
E. GENERAL INFORMATION ON THE TRIAL
F. POPULATION OF TRIAL SUBJECTS
G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
P. END OF TRIAL

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A. Protocol Information

A.1

Member State Concerned

France - ANSM

A.2

EudraCT number

2022-000174-25

A.3

Full title of the trial

A Phase IIa, Randomized, Double-Masked, Placebo-Controlled, Parallel-Group, Multicenter Study Assessing the Efficacy and Safety of STN1010904 Ophthalmic Suspension 0.03% and 0.1% Compared with Vehicle in Subjects with Fuchs Endothelial Corneal Dystrophy
(FECD)

A.3.1

Title of the trial for lay people, in easily understood, i.e. non-technical, language

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

A.3.2

Name or abbreviated title of the trial where available

PHANTOM Study

A.4.1

Sponsor's protocol code number

101090401IN

A.5.2

US NCT (ClinicalTrials.gov registry) number

NCT05376176

A.7

Trial is part of a Paediatric Investigation Plan

A.8

EMA Decision number of Paediatric Investigation Plan

B. Sponsor Information
B.Sponsor: 1
B.1.1	Name of Sponsor	SANTEN INCORPORATED
B.1.3.4	Country	United States
B.3.1 and B.3.2	Status of the sponsor	Commercial
B.4 Source(s) of Monetary or Material Support for the clinical trial:
B.4.1	Name of organisation providing support	Santen Inc.
B.4.2	Country	United States
B.5 Contact point designated by the sponsor for further information on the trial
B.5.1	Name of organisation	Santen Inc
B.5.2	Functional name of contact point	Robert Keaney
B.5.3	Address:
B.5.3.1	Street Address	6401 Hollis Street, Suite 125
B.5.3.2	Town/ city	Emeryville
B.5.3.3	Post code	CA 94608
B.5.3.4	Country	United States
B.5.4	Telephone number	+1415268-9052
B.5.6	E-mail	Robert.Keaney@santen.com

D. IMP Identification
D.IMP: 1
D.1.2 and D.1.3	IMP Role	Test
D.2	Status of the IMP to be used in the clinical trial
D.2.1	IMP to be used in the trial has a marketing authorisation	No
D.2.5	The IMP has been designated in this indication as an orphan drug in the Community	No
D.2.5.1	Orphan drug designation number
D.3 Description of the IMP
D.3.1	Product name	STN1010904 Ophthalmic Suspension
D.3.2	Product code	STN1010904 (DE-109D)
D.3.4	Pharmaceutical form	Eye drops
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Ocular use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	Sirolimus
D.3.9.1	CAS number	53123-88-9
D.3.9.2	Current sponsor code	STN1010904 (DE-109D)
D.3.9.3	Other descriptive name	Rapamune
D.3.9.4	EV Substance Code	SUB10537MIG
D.3.10	Strength
D.3.10.1	Concentration unit	% (W/V) percent weight/volume
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	0.1
D.3.11 The IMP contains an:
D.3.11.1	Active substance of chemical origin	Yes
D.3.11.2	Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP)	No
	The IMP is a:
D.3.11.3	Advanced Therapy IMP (ATIMP)	No
D.3.11.3.1	Somatic cell therapy medicinal product	No
D.3.11.3.2	Gene therapy medical product	No
D.3.11.3.3	Tissue Engineered Product	No
D.3.11.3.4	Combination ATIMP (i.e. one involving a medical device)	No
D.3.11.3.5	Committee on Advanced therapies (CAT) has issued a classification for this product	No
D.3.11.4	Combination product that includes a device, but does not involve an Advanced Therapy	No
D.3.11.5	Radiopharmaceutical medicinal product	No
D.3.11.6	Immunological medicinal product (such as vaccine, allergen, immune serum)	No
D.3.11.7	Plasma derived medicinal product	No
D.3.11.8	Extractive medicinal product	No
D.3.11.9	Recombinant medicinal product	No
D.3.11.10	Medicinal product containing genetically modified organisms	No
D.3.11.11	Herbal medicinal product	No
D.3.11.12	Homeopathic medicinal product	No
D.3.11.13	Another type of medicinal product	No
D.IMP: 2
D.1.2 and D.1.3	IMP Role	Test
D.2	Status of the IMP to be used in the clinical trial
D.2.1	IMP to be used in the trial has a marketing authorisation	No
D.2.5	The IMP has been designated in this indication as an orphan drug in the Community	No
D.2.5.1	Orphan drug designation number
D.3 Description of the IMP
D.3.1	Product name	STN1010904 Ophthalmic Suspension
D.3.2	Product code	STN1010904 (DE-109D)
D.3.4	Pharmaceutical form	Eye drops
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Ocular use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	Sirolimus
D.3.9.1	CAS number	53123-88-9
D.3.9.2	Current sponsor code	STN1010904 (DE-109D)
D.3.9.3	Other descriptive name	Rapamune
D.3.9.4	EV Substance Code	SUB10537MIG
D.3.10	Strength
D.3.10.1	Concentration unit	% (W/V) percent weight/volume
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	0.03
D.3.11 The IMP contains an:
D.3.11.1	Active substance of chemical origin	Yes
D.3.11.2	Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP)	No
	The IMP is a:
D.3.11.3	Advanced Therapy IMP (ATIMP)	No
D.3.11.3.1	Somatic cell therapy medicinal product	No
D.3.11.3.2	Gene therapy medical product	No
D.3.11.3.3	Tissue Engineered Product	No
D.3.11.3.4	Combination ATIMP (i.e. one involving a medical device)	No
D.3.11.3.5	Committee on Advanced therapies (CAT) has issued a classification for this product	No
D.3.11.4	Combination product that includes a device, but does not involve an Advanced Therapy	No
D.3.11.5	Radiopharmaceutical medicinal product	No
D.3.11.6	Immunological medicinal product (such as vaccine, allergen, immune serum)	No
D.3.11.7	Plasma derived medicinal product	No
D.3.11.8	Extractive medicinal product	No
D.3.11.9	Recombinant medicinal product	No
D.3.11.10	Medicinal product containing genetically modified organisms	No
D.3.11.11	Herbal medicinal product	No
D.3.11.12	Homeopathic medicinal product	No
D.3.11.13	Another type of medicinal product	No

D.8 Information on Placebo
D.8 Placebo: 1
D.8.1	Is a Placebo used in this Trial?	Yes
D.8.3	Pharmaceutical form of the placebo	Eye drops, solution
D.8.4	Route of administration of the placebo	Ocular use

E. General Information on the Trial

E.1 Medical condition or disease under investigation

E.1.1

Medical condition(s) being investigated

Fuchs Endothelial Corneal Dystrophy (FECD)

Dystrophie cornéenne endothéliale de Fuchs (FECD)

E.1.1.1

Medical condition in easily understood language

Fuchs Endothelial Corneal Dystrophy (FECD)

Dystrophie cornéenne endothéliale de Fuchs (FECD)

E.1.1.2

Therapeutic area

Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

MedDRA Classification

E.1.2 Medical condition or disease under investigation

E.1.2	Version	20.0
E.1.2	Level	LLT
E.1.2	Classification code	10014823
E.1.2	Term	Endothelial corneal dystrophy
E.1.2	System Organ Class	100000004850

E.1.3

Condition being studied is a rare disease

E.2 Objective of the trial

E.2.1

Main objective of the trial

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

E.2.2

Secondary objectives of the trial

To assess the dose response of STN1010904 ophthalmic suspension.

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

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.
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)

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.

Please refer to protocol for a full list of inclusion criteria.

E.4

Principal exclusion criteria

Subjects are excluded from the study if any of the following criteria apply:
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).
Please refer to protocol (section Exclusion Criteria) for a full list of exclusion criteria.
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).
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.).
Please refer to protocol for a full list of exclusion criteria.

E.5 End points

E.5.1

Primary end point(s)

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
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).

E.5.1.1

Timepoint(s) of evaluation of this end point

at Month 18

E.5.2

Secondary end point(s)

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
- 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
- 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
- Change and percent change in Guttae formation (Modified Krachmer scale) at all post-baseline visits

E.5.2.1

Timepoint(s) of evaluation of this end point

post-baseline visit

E.6 and E.7 Scope of the trial

E.6

Scope of the trial

E.6.1

Diagnosis

E.6.2

Prophylaxis

E.6.3

Therapy

Yes

E.6.4

Safety

Yes

E.6.5

Efficacy

Yes

E.6.6

Pharmacokinetic

E.6.7

Pharmacodynamic

E.6.8

Bioequivalence

E.6.9

Dose response

Yes

E.6.10

Pharmacogenetic

E.6.11

Pharmacogenomic

Yes

E.6.12

Pharmacoeconomic

E.6.13

Others

E.7

Trial type and phase

E.7.1

Human pharmacology (Phase I)

E.7.1.1

First administration to humans

E.7.1.2

Bioequivalence study

E.7.1.3

Other

E.7.1.3.1

Other trial type description

E.7.2

Therapeutic exploratory (Phase II)

Yes

E.7.3

Therapeutic confirmatory (Phase III)

E.7.4

Therapeutic use (Phase IV)

E.8 Design of the trial

E.8.1

Controlled

Yes

E.8.1.1

Randomised

Yes

E.8.1.2

Open

E.8.1.3

Single blind

E.8.1.4

Double blind

Yes

E.8.1.5

Parallel group

Yes

E.8.1.6

Cross over

E.8.1.7

Other

E.8.2

Comparator of controlled trial

E.8.2.1

Other medicinal product(s)

E.8.2.2

Placebo

Yes

E.8.2.3

Other

E.8.2.4

Number of treatment arms in the trial

E.8.3

The trial involves single site in the Member State concerned

E.8.4

The trial involves multiple sites in the Member State concerned

Yes

E.8.4.1

Number of sites anticipated in Member State concerned

E.8.5

The trial involves multiple Member States

E.8.6 Trial involving sites outside the EEA

E.8.6.1

Trial being conducted both within and outside the EEA

Yes

E.8.6.2

Trial being conducted completely outside of the EEA

E.8.6.3

If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned

India

United States

France

E.8.7

Trial has a data monitoring committee

E.8.8

Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial

A subject is considered to have completed the study if he/she has completed all phases of the study including Visit 9 (Month 18).

E.8.9 Initial estimate of the duration of the trial

E.8.9.1

In the Member State concerned years

E.8.9.1

In the Member State concerned months

E.8.9.1

In the Member State concerned days

E.8.9.2

In all countries concerned by the trial years

E.8.9.2

In all countries concerned by the trial months

E.8.9.2

In all countries concerned by the trial days

F. Population of Trial Subjects

F.1 Age Range

F.1.1

Trial has subjects under 18

F.1.1.1

In Utero

F.1.1.2

Preterm newborn infants (up to gestational age < 37 weeks)

F.1.1.3

Newborns (0-27 days)

F.1.1.4

Infants and toddlers (28 days-23 months)

F.1.1.5

Children (2-11years)

F.1.1.6

Adolescents (12-17 years)

F.1.2

Adults (18-64 years)

Yes

F.1.2.1

Number of subjects for this age range:

F.1.3

Elderly (>=65 years)

Yes

F.1.3.1

Number of subjects for this age range:

F.2 Gender

F.2.1

Female

Yes

F.2.2

Male

Yes

F.3 Group of trial subjects

F.3.1

Healthy volunteers

F.3.2

Patients

Yes

F.3.3

Specific vulnerable populations

Yes

F.3.3.1

Women of childbearing potential not using contraception

F.3.3.2

Women of child-bearing potential using contraception

Yes

F.3.3.3

Pregnant women

F.3.3.4

Nursing women

F.3.3.5

Emergency situation

F.3.3.6

Subjects incapable of giving consent personally

F.3.3.7

Others

F.4 Planned number of subjects to be included

F.4.1

In the member state

F.4.2

For a multinational trial

F.4.2.1

In the EEA

F.4.2.2

In the whole clinical trial

F.5

Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)

No subsequent intervention is planned for subjects after completing 18 months of treatment.

G. Investigator Networks to be involved in the Trial

N. Review by the Competent Authority or Ethics Committee in the country concerned

Competent Authority Decision

Authorised

Date of Competent Authority Decision

2023-03-17

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2022-10-05

P. End of Trial
P.	End of Trial Status	Ongoing

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