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Study on Retinoblastoma Treatment: Evaluating the Effectiveness of Topotecan, Vincristine Sulfate, and Etoposide in Patients Eligible for Conservative Therapy

Verified siteRegistered drugNo placebo
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What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial is focused on studying the treatment of retinoblastoma, a type of eye cancer that primarily affects young children. The study aims to evaluate the effectiveness of different treatment strategies to preserve vision and control the disease. The treatments being studied include a combination of medications such as Topotecan, Melphalan, Vincristine Sulfate, Etoposide, Carboplatin, and Melphalan Hydrochloride. These medications are administered in various forms, including solutions for infusion and injection, to determine the best approach for managing retinoblastoma.

The purpose of the study is to assess how well these treatments work in controlling the disease and preserving vision. The study is divided into two parts. The first part focuses on the use of intra-arterial chemotherapy (IAC), which involves delivering chemotherapy directly to the eye, to see how well it controls the disease. The second part evaluates the visual function of patients who receive other conservative treatments, such as intravenous (IV) chemotherapy combined with local eye treatments or local treatments alone, which are less invasive.

Participants in the study will receive one of the treatment options and will be monitored over time to see how well the treatments work in preserving the eye and vision. The study will also look at the visual outcomes based on criteria set by the World Health Organization (WHO). The goal is to find the most effective treatment strategy for children with retinoblastoma, ensuring the best possible visual outcomes while controlling the disease.

The research process

The trial runs in 7 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Joining the study

    Upon joining the study, the patient is assessed to ensure they meet the inclusion criteria. This includes being newly diagnosed with retinoblastoma (RB) and having at least one eye eligible for conservative management.

    The patient must not have received prior chemotherapy or radiotherapy for any cancer and must have no contraindications to the proposed treatments.

  2. Step 2

    Treatment allocation

    The patient is assigned to one of two studies based on their eligibility. Study 1 involves intra-arterial chemotherapy (IAC) for patients with RB manageable with this method. Study 2 involves other conservative treatments, potentially including intravenous (IV) chemotherapy.

  3. Step 3

    Study 1 treatment

    In Study 1, patients receive IAC with Topotecan and Melphalan. The goal is to evaluate the local control of the disease and the rate of eye preservation 24 months after randomization.

  4. Step 4

    Study 2 treatment

    In Study 2, patients may receive IV chemotherapy in combination with local ophthalmologic treatments or local treatments alone. The focus is on assessing visual function based on World Health Organization (WHO) criteria.

  5. Step 5

    Medication administration

    Medications used include Topotecan (solution for infusion), Vincristine (solution for injection), Etoposide (concentrate for infusion), Carboplatin (solution for injection/infusion), and Melphalan (solution for injection/infusion).

    The administration routes vary: intra-arterial for Topotecan and Melphalan, and intravenous for Vincristine, Etoposide, and Carboplatin.

  6. Step 6

    Follow-up and assessment

    Patients undergo regular follow-up visits to monitor treatment efficacy and side effects. Visual function is assessed according to WHO criteria, with evaluations continuing until the patient reaches 6 years of age and has at least 24 months of follow-up.

  7. Step 7

    Study completion

    The study is estimated to conclude by January 20, 2034. The primary endpoints include the rate of eye preservation in Study 1 and the percentage of patients with varying levels of visual impairment in Study 2.

Who can join the trial?

11 criteria

  • The patient must have a newly diagnosed condition called **RB** (retinoblastoma).
  • The patient must have at least one eye that can be treated without removing it.
  • The patient should be able to follow the study's requirements and attend all visits, including follow-ups.
  • The patient must not have received chemotherapy or radiotherapy for this or any other cancer before.
  • The patient should not have any medical reasons that would prevent them from receiving the proposed treatments.
  • The parents or legal representative of the patient must sign a document called an **informed consent**, which means they agree to the patient participating in the study.
  • The patient must be covered by the French Social Security System.
  • For Study 1, the patient should be a child aged between 6 months and 6 years.
  • For Study 1, the patient must have RB in at least one eye that can be managed with a treatment called **IAC** (Intra-Arterial Chemotherapy) without using another treatment called **IV chemotherapy** (Intravenous Chemotherapy).
  • For Study 2, the patient should have RB that can be managed without IAC, such as:
    • Unilateral RB (in one eye) in children under 6 months, classified as Group A, B, C, or D, with or without small particles in the eye called **vitreous seeding**, and suitable for treatment without removing the eye.
    • Bilateral RB (in both eyes) classified as Group A, B, C, D, or E, without affecting the front part of the eye, and/or with large tumors covering more than two-thirds of the eye, but still suitable for treatment without removing the eye.

Who cannot join the trial?

1 criterion

  • There are no specific reasons listed that would prevent a patient from participating in this study.
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Investigated drugs

  • IAC (Intra-Arterial Chemotherapy)

    is a treatment method where chemotherapy drugs are delivered directly into the artery that supplies blood to the eye. This approach aims to control the disease locally by targeting the tumor more precisely, potentially reducing side effects compared to systemic chemotherapy.

  • IV Chemotherapy (Intravenous Chemotherapy)

    involves administering chemotherapy drugs through a vein. This method allows the medication to travel through the bloodstream to reach cancer cells throughout the body. In this trial, it is used in combination with local eye treatments to manage retinoblastoma.

  • Local Ophthalmologic Treatments

    refer to various procedures applied directly to the eye to treat retinoblastoma. These treatments can include laser therapy, cryotherapy, or other methods that focus on the tumor in the eye, aiming to preserve vision and control the disease with minimal invasiveness.

What is already known about the treatment

Intra-arterial Chemotherapy (IAC) – This medication is administered directly into the artery supplying the eye, specifically for treating retinoblastoma, a type of eye cancer. It is currently being studied in clinical trials to evaluate its effectiveness in controlling the disease locally. The main therapeutic indication is for ocular conservative treatment of retinoblastoma. At the molecular level, IAC works by delivering chemotherapy drugs directly to the tumor, minimizing exposure to the rest of the body and potentially reducing side effects. It is classified under targeted chemotherapy treatments.

Investigated diseases

Retinoblastoma – Retinoblastoma is a rare type of eye cancer that typically develops in early childhood, affecting the retina, which is the light-sensitive lining at the back of the eye. It can occur in one or both eyes and may lead to vision problems or loss of vision if not managed. The disease often presents as a white reflection in the pupil, known as leukocoria, and may also cause eye redness or swelling. Retinoblastoma can progress rapidly, potentially spreading to other parts of the body if not treated. The condition is usually diagnosed in children under the age of five and can be hereditary or occur sporadically. Early detection and intervention are crucial for preserving vision and preventing further complications.
Trial detailsLast updated 7 Oct 2026
Age0-17PhasePhase IITrial ID2024-514025-30-00Protocol codeIC 2019-05Estimated enrolment225 patientsSponsorInstitut Curie

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