A phase III, randomized, double blind, multiple doses, placebo-controlled parallel-group, adaptive study to evaluate the efficacy of atropine to treat the progression of myopia in children and adolescents from 3 to less than 18 years of age (MODERATO Study)

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Ocus Innovation Ireland Limited

Participant type

Pediatric, Patients

Age range

0-17 years

Gender

Male and Female

Therapeutic area

Diseases [C] - Eye Diseases [C11]

Trial duration

25 Sep 2025 → ongoing

Decision date (initial)

2025-01-13

Transition trial

No

Low-intervention

No

Rare-disease indication

No

Vulnerable population

Yes

External identifiers

EU CT number

2023-510439-13-00

WHO UTN

U1111-1304-3811

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Efficacy, Safety

To evaluate the efficacy of atropine eyedrops at two concentrations, 0.025% and 0.05%, over a 24-month treatment period, in slowing the progression of myopia in children and adolescents from 3 to 18 years old.

Secondary objectives 4

1. To investigate early escape rate in the placebo group (identical looking eyedrops solution but with no medicine in them) at 6 months. This means that to patients who will show significant worsening of myopia at month 6, the investigator will then start to administer atropine eyedrops.
2. To investigate the efficacy of the intervention over a longer period of time in the second 12-months period of the study.
3. To investigate the safety of 0.025% and 0.05% atropine eyedrops in children and adolescents at 3, 6, 12, 18 and at 24 months.
4. To investigate the vision-related quality of life of patients at baseline, 6, 12 and 24 months.

Conditions and MedDRA coding

Myopia

Study design 1 period

Regulatory references

EMA paediatric investigation plan (PIP)

EMEA-002545-PIP01-19

Plan to share IPD

No

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 6

1. Male and female subjects from 3 to less than 18 years of age.
2. Subjects showing myopia with a SER of both eyes at least -0.75 D at baseline.
3. Intraocular pressure ≤ 21 mm Hg in each eye.
4. Parents or legal representative who have been informed about the clinical trial and have signed the informed consent form, with the exception of subjects aged over 16 years only in the UK, who can provide their own consent, according to the local regulations.
5. Women with childbearing potential (WOCBP) who have a negative highly sensitive urine dipstick pregnancy test. Additional pregnancy testing during the clinical trials will be conducted at visits 1, 2, 3, 4, 5, 6.
6. WOCBP or males who are using a highly effective birth control method for contraception. Eligible highly effective contraceptive methods for WOCBP are combined (estrogen and progestogen containing) hormonal contraception associated with inhibition of ovulation (oral, intravaginal, transdermal); progestogen-only hormonal contraception associated with inhibition of ovulation (oral, injectable, implantable); intrauterine device; intrauterine hormone-releasing system. Sexual abstinence represents a highly effective contraceptive method, if in line with the subject's normal habits.

Exclusion criteria 16

1. Anisometropia, meaning a significant difference in refractive power between the two eyes exceeding |1.5| D.
2. Refractive astigmatism (ΔTK) > |1.5| D.
3. Presence of ocular pathologies such as pathological myopia, corneal scars, or other anterior or posterior eye pathologies.
4. History of amblyopia or strabismus.
5. Presence of a history of a retinal dystrophy or systemic disorder that may predispose to severe myopia (e.g., Marfan syndrome, retinitis pigmentosa, Stickler syndrome, retinopathy of prematurity)
6. Abnormal ocular biometry aside from axial length (e.g., keratoconus, lenticonus, spherophakia) or previous intraocular or ocular laser/non-laser surgery.
7. History of glaucoma; anatomic narrow anterior chamber angles.
8. Down syndrome or spastic paralysis.
9. Known intolerances/allergies against atropine eyedrops, or hypersensitivity to any component of the IMP.
10. Pregnancy or breastfeeding.
11. Previous or current alcohol or drug abuse.
12. Mental or emotional instability that might jeopardize the compliance with the trial procedures.
13. Unreliability or lack of cooperation.
14. History of any myopia control treatment within 3 months before inclusion: e.g. peripheral-plus or diffusion-optics-technology glasses, orthokeratology contact lenses, peripheral-plus/multifocal contact lenses, atropine eyedrops.
15. Other reasons why, in the opinion of the investigator, the subjects should not participate in the trial.
16. Patients who have consented to participate in the clinical trial, but do not meet one or more eligibility criteria required for participation in the trial during the screening procedures, and subsequently are not randomly assigned to the study treatment or entered in the study, are considered screening failures.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Mean annual rate of progression of myopia (D/year) based on spherical equivalent (SE) measured by cycloplegic autorefraction through 24 months.

Secondary endpoints 6

1. Number and percentage of early escape patients at 6 months.
2. Mean change in axial length and glasses prescription at 3, 6, 12, 18 and 24 months.
3. Safety at 3, 6, 12, 18 and 24 months, measuring: % children requiring photochromic and/or varifocal lenses counts and percentage of patients reporting any AEs change in photosensitivity index best corrected distance and near visual acuity pupil size and accommodation amplitude
4. Vision-related quality of life.
5. Acceptability of the formulation: it will be assessed with a 3-item questionnaire, scored on a six-point scale.
6. Overall between-group difference from baseline in the proportion of subjects who show < -0.50 D myopia progression (Spherical Equivalent Refraction, SER) at 24 months: Chi-square test, or Fisher's exact test, will be used to test for differences among three treatment groups in the proportion of subjects who show < -0.50 D myopia progression (SER) at 24 months.

Investigational products

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

Test 2

Placebo 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Ocus Innovation Ireland Limited

Sponsor organisation

Ocus Innovation Ireland Limited

Address

20 Harcourt Street

City

Dublin 2

Postcode

D02 H364

Country

Ireland

Scientific contact point

Organisation

Ocus Innovation Ireland Limited

Contact name

Annegret Dahlmann-Noor

Public contact point

Organisation

Ocus Innovation Ireland Limited

Contact name

Annegret Dahlmann-Noor

Third parties 3

Locations

3 EU/EEA countries · 7 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

Documents 67 files

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

Application history

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