Multicenter randomized controlled trial of the value of intravitreal anti-VEGF injections as initial then adjuvant treatment in Coats' disease

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Fondation A De Rothschild

Participant type

Pediatric, Patients

Age range

0-17 years

Gender

Male and Female

Therapeutic area

Diseases [C] - Eye Diseases [C11]

Trial duration

6 Nov 2024 → 28 Jan 2026

Decision date (initial)

2024-11-06

Transition trial

Yes

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

Yes

Funding sources

Ministry of solidarity and health

External identifiers

EU CT number

2024-517036-21-00

EudraCT number

2018-003863-67

ClinicalTrials.gov

NCT03940690

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Efficacy

To compare the efficacy of treatment with intravitreal (IVT) injection of anti-VEGF (bevacizumab, Avastin®) on the stage of Coats' disease, versus reference treatment with laser photocoagulation, 6 months after initiation of study treatment.

Secondary objectives 3

1. To compare the efficacy of IVT injections of anti-VEGF (bevacizumab, Avastin®) as initial treatment and then adjuvant to laser photocoagulation sessions versus laser photocoagulation treatment alone, at 2 months, 4 months and 9 months after initiation of study treatment.
2. To evaluate the safety of intravitreal injections of bevacizumab
3. To measure the agreement between the two independent expert ophthalmologist assessors of the stage of Coats' disease at each assessment (at 0, 1, 2, 4, 6 and 9 months)

Conditions and MedDRA coding

Coats' disease

Study design 1 period

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 6

1. Patient ≤ 16 years
2. Coats disease confirmed by fundus examination and fluorescein angiography
3. Stages 2 or 3 on fundus examination (Shields classification)
4. Naïve to any ocular treatment on the eye affected by Coats' disease
5. Beneficiary of a Social Security scheme
6. Free, informed, written consent of both holders of parental authority

Exclusion criteria 10

1. Other ocular pathology on the eye affected by Coats' disease
2. Bilateral forms of the disease
3. History of hypersensitivity to bevacizumab
4. History of hypersensitivity to Chinese Hamster Ovary (CHO) cell products or to other human or humanized recombinant antibodies
5. Allergic reaction to previous fluorescein retinal angiography
6. Active or suspected periocular infection
7. Contraindication to treatments used for general anesthesia (Propofol, halogenated gases (Sevoflurane) and morphine derivatives (Sufentanil))
8. Cardiovascular, hemorrhagic and gastrointestinal risks
9. Premature infant under 37 weeks corrected age
10. Pregnancy or breastfeeding

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Proportion of patients with improvement in at least one stage of the disease according to the Shields classification (Am J Ophthalmol, 2001), 6 months after initiation of study treatment

Secondary endpoints 7

1. Comparison, between the 2 groups, of the proportion of patients with an improvement of at least one stage of the Shields classification assessed on multimodal retinal imaging by two independent experts at 2 months, 4 months and 9 months after initiation of study treatment
2. Comparison between the 2 groups of each of the items defining the Shields stage at 2, 4, 6 and 9 months after initiation of study treatment: presence of telangiectasia, presence of exudates, presence of retinal detachment; glaucoma, bulbar phthisis
3. Comparison between the 2 groups of visual acuity (expressed in logMAR) at 4, 6, and 9 months after initiation of study treatment, in children of verbal age (from 3 years)
4. Comparison between the 2 groups of macular thickness variation (in micrometers), the presence of serous detachment, and the maximum thickness of fibrosis observed on optical coherence tomography at 2, 4, 6, and 9 months after initiation of study treatment in children for whom it was performed
5. Comparison between the 2 groups in terms of the number of patients requiring additional treatment (i.e., an additional laser session beyond the third session or surgical treatment) 9 months after initiation of study treatment
6. Comparison between the 2 groups in terms of the number of events and adverse effects (i.e., progression to stage 5 (phthisis); need for surgical treatment; cataract; vitreoretinal fibrosis; tractional retinal detachment, as well as any serious adverse event detected by the investigator leading to discontinuation of treatment)
7. Calculation of the kappa coefficient, measuring the agreement between the two independent expert ophthalmologist evaluators at each evaluation of Coats disease stage at 0, 1, 2, 4, 6, and 9 months after initiation of study treatment

Investigational products

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

Test 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Fondation A De Rothschild

Sponsor organisation

Fondation A De Rothschild

Address

29 Rue Manin

City

Paris

Postcode

75019

Country

France

Scientific contact point

Organisation

Fondation A De Rothschild

Contact name

Clinical research Department

Public contact point

Organisation

Fondation A De Rothschild

Contact name

Clinical research Department

Locations

1 EU/EEA country · 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

Documents 10 files

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

Application history

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