A Multicenter, Randomized, Double-Masked, Vehicle-Controlled Study to Assess the Safety and Efficacy of SYD-101 Ophthalmic Solution for the Treatment of Myopia in Children

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Sydnexis Inc.

Participant type

Pediatric, Patients

Age range

0-17 years, 18-64 years

Gender

Male and Female

Therapeutic area

Diseases [C] - Eye Diseases [C11]

Trial duration

23 Oct 2019 → 1 May 2025

Decision date (initial)

2024-04-25

Transition trial

Yes

Low-intervention

No

Rare-disease indication

No

Vulnerable population

Yes

Funding sources

Sydnexis, Inc.

External identifiers

EU CT number

2023-509134-19-00

EudraCT number

2018-004775-13

ClinicalTrials.gov

NCT03918915

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Safety, Efficacy

To evaluate the efficacy of SYD-101 for slowing the progression of myopia in children
To evaluate the safety and tolerability of SYD-101

Conditions and MedDRA coding

Myopia

Study design 2 periods

Regulatory references

Scientific advice from competent authorities

European Medicines Agency, Swedish Medical Products Agency

Plan to share IPD

Yes

IPD plan description

Sydnexis will provide a Lay Language Summary of the clinical trial to study sites, for sites to distribute to study participants as needed. This activity will be completed within 6 months of the 48-month clinical study report being finalized.

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 11

1. Parent/guardian has the ability to understand the study informed consent form (ICF) and agrees to sign the ICF prior to initiation of any protocol-related procedures; participant has the ability to give assent, as applicable, at the time of parental/guardian consent
2. Participant is male or female between 3 and 14 years of age (inclusive) at the time of Screening
3. Participant and parent/guardian are willing and able to comply with study instructions, study visits, and procedures
4. Participant (or parent/guardian) has demonstrated ability to administer artificial eyedrops at the Screening or Baseline visit
5. Participant is in good general health, with no clinically significant findings based on medical history and vital signs, as determined by the investigator at the time of Screening
6. Postmenarchal female participants must have negative urine pregnancy test results
7. Refractive error by cycloplegic autorefraction at the baseline visit: a) Myopia of 0.50 D to 6.00 D (inclusive) b) Astigmatism ≤1.50 D c) Anisometropia ≤1.00 D
8. If the baseline myopia (SE) is <0.75 D, participant must have a history of myopia progression of 0.50 D in the previous 6 to 12 months
9. If baseline myopia (SE) is ≥0.75 D, participant must be wearing refractive correction (single vision eyeglasses or soft, daily-wear, single-vision contact lenses) that meets the following criteria: a) Myopia (SE) corrected to within ±0.50 D of the investigator’s cycloplegic measurement of refractive error b) Cylinder power must be within ±0.50 D of the investigator’s standard refraction technique, which can be based on a cycloplegic or non-cycloplegic refraction c) Cylinder axis must be within ±5 degrees of the axis found on the investigator’s standard refraction when cylinder power is ≥1.00 D or within ±15 degrees when the cylinder power is <1.00 D
10. BCVA of 75 letters (Snellen equivalent 20/32) or better
11. Normal IOP <21 mmHg

Exclusion criteria 13

1. Participant is a female who is pregnant, lactating, or intending to become pregnant within next 4 years
2. Participant has a known allergy or hypersensitivity to atropine or any of the components of SYD-101
3. Participant has history or current evidence of a medical condition predisposing the participant to degenerative myopia (eg, Marfan syndrome, Stickler syndrome) or a condition that may affect visual function or development (eg, diabetes mellitus, chromosome anomaly)
4. One or more biological parents with a history of myopia ≥9.00 D
5. Current use of a monoamine oxidase inhibitor
6. History of, or currently receiving treatment for, any systemic infection or autoimmune disease considered serious by the investigator
7. Participation in an investigational drug or device study within 30 days prior to Screening
8. Evidence of any ocular inflammation or infection in either eye, including blepharitis, conjunctivitis, keratitis, and scleritis
9. History or evidence of the following in either eye: a) Retinopathy of prematurity b) Abnormal refractive anatomy (eg, keratoconus, lenticonus, spherophakia) c) Amblyopia, manifest strabismus, or nystagmus
10. Use of any of the following (previously, currently, or plans to do so in the future): a) Orthokeratology (orthoK), rigid gas-permeable, bifocal, progressive-addition, multi-focal, or other lenses to reduce myopia progression b) Use of atropine, pirenzepine, or other anti-muscarinic agent for myopia
11. History or evidence of any ocular surgery or planned future ocular surgery in either eye
12. History or current evidence of ocular disease in the either eye that, in the opinion of the investigator, may confound assessment of visual acuity and/or refraction
13. Unwillingness or inability to comply with study requirements and restrictions, including but not limited to those specified in Section 5.3 (eg, required conversion from extended wear lenses to daily wear lenses, full-time use of contact lenses or spectacles)

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 2

1. Mean annual progression rate of myopia through month 24
2. Evaluation of AEs and SAEs

Secondary endpoints 15

1. Proportion of participants with myopic progression >0.75 D at or before Month 24
2. Proportion of participants with annual myopia progression rate through Month 24 ≤0.50 D/year
3. Proportion of participants with annual myopia progression rate through Month 24 ≤0.25 D/year
4. Proportion of participants with increase of myopia of >0.50 D at or before Month 24
5. Time to progression of myopia of >0.75 D through Month 24
6. Mean annual progression rate using Month 24 data on Subgroup of participants with refractive history of progression ≥ 0.5D
7. Mean annual progression rate using Month 24 data on Subgroup of participants with refractive history of progression ≥ 0.75D
8. Mean change from baseline in axial length at Month 24 (at sites with the requisite equipment; at least 50% of participants)
9. Mean change from baseline in SE at Month 48 (ie, 12 months after randomized withdrawal)
10. Mean time spent on various activities will be solicited via questionnaire
11. Proportion of agreement to QOL statements
12. Changes from baseline in vital sign measurements
13. Mean change from baseline in pupil diameter. Mean change from baseline in binocular accommodative amplitude . Changes from baseline in findings detected by best-corrected visual acuity (BCVA), biomicroscopy, intraocular pressure (IOP), and ophthalmoscopy. Changes from baseline in corneal endothelial cell count (selected sites only; approximately 25% of study participants).
14. Tolerability to the masked study drug will be solicited via questionnaire
15. Pregnancy test results (female participants of childbearing potential only)

Investigational products

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

Test 2

Placebo 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Sydnexis Inc.

Sponsor organisation

Sydnexis Inc.

Address

445 Marine View Avenue Suite 295

City

Del Mar

Postcode

92014-3926

Country

United States

Scientific contact point

Organisation

Sydnexis Inc.

Contact name

Janet Cheetham

Public contact point

Organisation

Sydnexis Inc.

Contact name

Clinical Trial Information Desk

Third parties 2

Locations

2 EU/EEA countries · 6 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 7 files

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

Application history

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