RETINO 2018: conservative treatment for retinoblastoma: efficacy of the new management strategies and visual outcome

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Institut Curie

Participant type

Pediatric, Patients

Age range

0-17 years

Gender

Male and Female

Therapeutic area

Diseases [C] - Neoplasms [C04]

Trial duration

25 Mar 2021 → ongoing

Decision date (initial)

2024-05-31

Transition trial

Yes

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

Yes

External identifiers

EU CT number

2024-514025-30-00

EudraCT number

2019-005039-94

ClinicalTrials.gov

NCT04681417

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Efficacy

- Study 1: To evaluate the efficacy of IAC in term of local control of the disease in patients eligible for IAC (randomized phase II study);
- Study 2: To assess the visual function, based on World Health Organization (WHO) criteria, in patients eligible for other conservative treatments (IV chemotherapy in association with local ophthalmologic treatments or local ophthalmological treatment without IV chemotherapy; this is a minimally invasive interventional study).

Conditions and MedDRA coding

Ocular conservative treatment for retinoblastoma

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 10

1. Overall study inclusion criteria: Newly diagnosed RB.
2. Overall study inclusion criteria: RB with at least one eye eligible for conservative management
3. Overall study inclusion criteria: Patients likely to be compliant with the study requirements and visits, including late follow-up.
4. Overall study inclusion criteria: Patients not previously treated with chemotherapy or radiotherapy for this or any other cancer.
5. Overall study inclusion criteria: Patients with no contraindication to the proposed treatments.
6. Overall study inclusion criteria: Informed consent signed by parents or legal representative
7. Overall study inclusion criteria: French Social Security System coverage.
8. Study 1 inclusion criteria:Children aged from 6 months to 6 years.
9. Study 1 inclusion criteria: RB in at least one eye, deemed manageable with IAC in one side and without IV chemotherapy: a. Unilateral RB classified as group B, C (if vitreous seeds < 3 mm), D or E with no invasion of the anterior segment, and without massive tumor of more than 2/3 of the eye, eligible for conservative management, or b. Bilateral RB but with very unbalanced lesions between the two eyes: one eye group D or E without invasion of the anterior segment or massive tumor of more than 2/3 of the eye, eligible for IAC, and the other eye eligible for local treatment only (without IAC).
10. Study 2 inclusion criteria: RB eligible for conservative management although not manageable with IAC: a. Unilateral RB in children below 6 months of age, classified as Group A, B, C or D, with or without vitreous seeding, compatible with conservative management, or b. Bilateral RB classified as group A, B, C D, or E without invasion of the anterior segment, and/or massive tumors of more than 2/3 of the eye and eligible for conservative management.

Exclusion criteria 10

1. Overall study non-inclusion criteria: RB not eligible for conservative management : a. Extra-ocular extension of the disease, or b. Group E eyes with invasion of the anterior segment, and/or massive tumors of more than 2/3 of the eye.
2. Overall study non-inclusion criteria: Patient older than 6 years of age.
3. Overall study non-inclusion criteria:Patients with another associated disease contra indicating systemic chemotherapy.
4. Overall study non-inclusion criteria: Previously treated retinoblastoma by chemotherapy
5. Overall study non-inclusion criteria: Patients already treated for another malignant disease
6. Overall study non-inclusion criteria: Patient with any psychological, familial, sociological or geographical condition potentially hampering compliance with the study protocol and follow-up schedule.
7. Overall study non-inclusion criteria: Patients whose parents have not accepted the treatment regimen after explanation of it.
8. Overall study non-inclusion criteria: Contraindication to study drug mentioned in SmPC (Summary of Products Characteristics) of the study drugs
9. Overall study non-inclusion criteria: Inclusion in another experimental anti-cancer drug therapy
10. Study 1 non-inclusion criteria: Any contraindication or concomitant disease that would preclude the Study 1 treatment procedure and could delay treatment.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 2

1. Study 1: Rate of eye preservation in the investigational arm (Topotecan + Melphalan) and in the reference arm (Melphalan) 24 months after the date of randomization.
2. Study 2: Percentage of patients with major, mild or no impairment of visual function according to WHO criteria, i.e. normal bilateral visual acuity (≥ 6/10) or mild bilateral visual impairment (3/10 to 5/10) according to tumor location and extension, when the patient will have 6 years of age and at least 24 months of follow-up after study inclusion.

Investigational products

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

Test 6

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Institut Curie

Sponsor organisation

Institut Curie

Address

26 Rue D Ulm

City

Paris

Postcode

75005

Country

France

Scientific contact point

Organisation

Institut Curie

Contact name

Livia LUMBROSO - LE ROUIC

Public contact point

Organisation

Institut Curie

Contact name

Livia LUMBROSO - LE ROUIC

Locations

1 EU/EEA country · 28 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Application history

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