A Phase 3 Double-masked, Randomized, Multicenter Study of the Efficacy and Safety of OCS-01 Eye Drops in Subjects With Diabetic Macular Edema

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Oculis Operations S.a.r.l.

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

completed 29 Jul 2024

Decision date (initial)

2024-05-23

Transition trial

No

Low-intervention

No

Rare-disease indication

No

Vulnerable population

Yes

Funding sources

Oculis Operations Sarl, Switzerland

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Safety, Others, Efficacy

To evaluate the efficacy and safety of OCS-01 as compared to Vehicle at Week 52 in subjects with Diabetic macular edema (DME).

Conditions and MedDRA coding

Diabetic Macular Edema (DME)

Study design 2 periods

Regulatory references

Scientific advice from competent authorities

European Medicines Agency, Federal Institute For Drugs And Medical Devices, Swedish Medical Products Agency

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 8

1. Signed informed consent form before any study-specific procedures are performed.
2. Male or female adult subject aged 18 to 85 years.
3. DME with presence of intraretinal and/or subretinal fluid in the study eye, with CST of ≥310 µm by SD-OCT at screening (Visit 1) (to be confirmed by CRC); CST is not part of the eligibility reconfirmation on Day 1 (Visit 2).
4. BCVA ETDRS letters score >35 letters (>20/200 Snellen equivalent) in the non-study eye at screening (ie, as per definition of monocular blindness).
5. BCVA ETDRS letters score ≤65 (Snellen 20/50) and ≥24 (Snellen 20/320) in the study eye at screening and baseline (Visit 1 and Visit 2).
6. Documented diagnosis of Type 1 or Type 2 diabetes mellitus and an HbA1c of ≤ 10.0% prior to screening (Visit 1) (historic values of HbA1c taken up to 2 months before the screening visit will be permissible otherwise the study site will collect a sample for analysis at screening [Visit 1]).
7. Anti-vascular endothelial growth factor (VEGF) and corticosteroid IVT treatment-naïve (ie, have not received previous treatment with any anti-VEGF and corticosteroid IVT) in the study eye, OR treated with anti-VEGF agents IVT and/or corticosteroids IVT in the study eye in the past and for whom the following washout periods before Day 1 apply: a. Anti-VEGF agents IVT: 3 months b. Periocular or IVT corticosteroids: i. Triamcinolone: 4 months ii. Biodegradable slow-release steroid IVT implant (eg, Ozurdex): 6 months iii. Nonbiodegradable slow-release steroid implant (eg, Iluvien, Retisert, Yutiq): 3 years
8. Negative urine pregnancy test at Visit 1, if women of childbearing potential (WOCBP) those who have experienced menarche and who are not surgically sterilized ([bilateral tubal ligation, hysterectomy or bilateral oophorectomy] or postmenopausal [12 months after last menses]) and must use adequate birth control throughout the study period (refer to Appendix 2 of the protocol).

Exclusion criteria 13

1. Macular edema considered to be because of a cause other than DME. Examples included: The macular edema is considered to be related to ocular surgery, clinical exam and/or OCT suggest that vitreoretinal interface abnormalities disease, acute macular degeneration (AMD), retinal vein occlusion (RVO), uveitis.
2. Decrease in BCVA because of causes other than DME.
3. Known history of significant macular ischemia which would prevent gain in visual acuity in the study eye.
4. Any other ocular disease in the study eye that may cause substantial reduction in BCVA, including retinal detachment, vitreomacular traction, epiretinal membrane, vitreous hemorrhage or fibrosis involving the macula, ocular inflammation (uveitis), other retinal inflammatory or infectious diseases.
5. Active or suspected periocular or ocular infection in the study eye. Mild noninfectious blepharitis is accepted.
6. History of noninfectious uveitis in the study eye.
7. Uncontrolled ocular hypertension or glaucoma in either eye, defined as IOP >22 mmHg while on more than 1 IOP-lowering medication at screening (Visit 1).
8. Subjects who plan to continue using contact lenses (including cosmetic contact lenses) during the study in the study eye.
9. Subjects with high-risk proliferative diabetic retinopathy (PDR) as per CRC assessment at screening (Visit 1) only.
10. Currently enrolled in or have participated in any other clinical study involving a study drug or device, or in any other type of medical research, within 30 days before screening and up to completion of the current study.
11. Use of systemic corticosteroids (ie, oral, IM, IV, intranasal) within 1 month prior to Day 1 and no systemic corticosteroids anticipated throughout the study.
12. Any prior or concomitant systemic anti-VEGF treatment within 6 months prior to Day 1.
13. Any other medical condition that in the opinion of the investigator may affect BCVA, may put the subject at significant risk, may confound the study results, or may interfere significantly with the subject’s participation in the study.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. The primary endpoint is mean change in Best Corrected Visual Acuity (BCVA) Early Treatment Diabetic Retinopathy Study (ETDRS) letters score at Visit 12 (Week 52) compared with baseline.

Secondary endpoints 4

1. The proportion of subjects with a 3-line or greater gain in BCVA assessed with the ETDRS scale at Visit 12 (Week 52) compared with baseline.
2. Other Secondary Efficacy: • Area under the curve (AUC) of BCVA ETDRS letter changes across postbaseline visits up to Visit 12 (Week 52). • Mean change in central subfield thickness (CST) as measured by SD-OCT at Visit 12 (Week 52) compared with baseline.
3. Other Secondary Efficacy:• Mean change in CST as measured by SD-OCT at each postbaseline visit compared with baseline.• Mean change in BCVA ETDRS letters score at each postbaseline visit compared with baseline. • The proportion of subjects with a 3-line or greater gain in BCVA assessed with the ETDRS scale at each postbaseline visit.
4. Safety Endpoints: • Adverse events (AEs) • Intraocular pressure (IOP) • Hemoglobin A1c (HbA1c) • Lens clarity grading • Endothelial cell counts • Hematology • Serum chemistry • Blood pressure

Investigational products

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

Test 1

Placebo 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Oculis Operations S.a.r.l.

Sponsor organisation

Oculis Operations S.a.r.l.

Address

Building D, Epfl Innovation Park Epfl Innovation Park

City

Lausanne

Postcode

1015

Country

Switzerland

Scientific contact point

Organisation

Oculis Operations S.a.r.l.

Contact name

Bastian Dehmel

Public contact point

Organisation

Oculis Operations S.a.r.l.

Contact name

Bastian Dehmel

Third parties 7

Locations

6 EU/EEA countries · 34 investigational sites

By country

Investigational sites

Application history

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