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Study on Retinoblastoma Treatment Using Etoposide, Carboplatin, and Melphalan for Patients with Specific Eye Conditions

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 focuses on the conservative treatment of patients with retinoblastoma, a rare type of eye cancer that typically affects young children. The study involves several medications, including etoposide, carboplatin, melphalan, and vincristine sulfate. These medications are used in different combinations and methods to treat the disease. The purpose of the study is to evaluate the effectiveness of these treatments in preserving the eye without the need for external beam radiation.

The study is divided into three parts. In the first part, patients with certain types of retinoblastoma receive intravenous chemotherapy with etoposide and carboplatin, followed by a treatment that combines chemotherapy and heat therapy, known as chemothermotherapy. The second part involves patients with more advanced retinoblastoma receiving melphalan through a method called intra-arterial chemotherapy, where the drug is delivered directly to the eye's blood supply. The third part of the study includes patients with more severe cases, who receive a combination of three drugs, including vincristine sulfate, along with local treatments like laser therapy and cryotherapy, which uses extreme cold to destroy cancer cells.

The study aims to determine how well these treatments work in saving the eye and preventing the need for radiation therapy. It also looks at the side effects of the treatments and the risk of the cancer coming back. The study will follow patients over time to monitor their response to the treatments and any potential long-term effects. The ultimate goal is to find effective ways to treat retinoblastoma while preserving the patient's vision and quality of life.

The research process

The trial runs in 5 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Initial treatment phase

    The treatment begins with the administration of intravenous chemotherapy. This involves the use of two medications: etoposide and carboplatin. These medications are given through a vein, which is known as intravenous use.

    The purpose of this phase is to start treating the retinoblastoma and to evaluate the effectiveness of the treatment in preserving the eye without the need for external radiation.

  2. Step 2

    Chemothermotherapy phase

    Following the initial chemotherapy, a treatment called chemothermotherapy is administered. This phase does not include laser treatment on day 8.

    The goal is to continue treating the retinoblastoma while reducing the number of laser sessions needed.

  3. Step 3

    Intra-arterial chemotherapy phase

    For certain patients, melphalan is administered directly into the artery that supplies the eye. This is known as intra-arterial chemotherapy.

    This method is used to treat specific cases of retinoblastoma and is combined with local treatments such as laser treatment, cryoapplication, or radioactive plaques.

  4. Step 4

    Local treatment phase

    Local treatments may include laser treatment, cryoapplication, or the use of radioactive plaques. These treatments are aimed at preserving the eye and are used in combination with chemotherapy.

    In some cases, melphalan is injected directly into the eye, known as intravitreal use, to further treat the retinoblastoma.

  5. Step 5

    Follow-up phase

    Regular follow-up appointments are scheduled to monitor the response to treatment and to check for any signs of relapse.

    These follow-ups include eye examinations, which may be performed under general anesthesia for younger children, and assessments of any side effects from the treatments.

Who can join the trial?

12 criteria

  • Patients must not have been previously treated with chemotherapy or radiotherapy for this tumor or any other cancer.
  • Patients must be able to have long-term follow-up care.
  • Parents or legal representatives must provide written informed consent for the child to participate.
  • Patients must have social security coverage.
  • There should be no medical reasons preventing the use of the study treatments.
  • For Study 1 and Study 3, children must be between 0 to 6 years old.
  • For Study 1, patients with unilateral retinoblastoma in groups A, B, or C, or bilateral retinoblastoma in groups A, B, or C, are eligible. This excludes patients with a threat to the macula (the central part of the retina) and those with both eyes in group D or only one remaining eye.
  • For Study 1, patients less than six months old with unilateral retinoblastoma in groups B, C, or D, or bilateral retinoblastoma with one eye in group D and the other eye treatable without chemotherapy, are eligible.
  • For Study 2, patients with bilateral retinoblastoma that is very different in each eye, with one eye in group D treatable with intra-arterial chemotherapy using Melphalan, and the other eye treatable with local treatments without chemotherapy, are eligible.
  • For Study 2, children must be between 6 months to 6 years old.
  • For Study 2, patients with unilateral or bilateral retinoblastoma in group B, C, or D are eligible.
  • For Study 3, children with bilateral retinoblastoma in group D or with only one eye that can be treated conservatively are eligible.

Who cannot join the trial?

4 criteria

  • Patients with **bilateral group D** retinoblastoma, which means the cancer is in both eyes and is more advanced.
  • Patients with **bilateral macular threat**, meaning the cancer is threatening the central part of the retina in both eyes.
  • Patients who are not suitable for **local treatment** in one eye, which means treatments like laser or cryotherapy cannot be used effectively.
  • Patients who are part of a **vulnerable population**, which refers to groups that may need special protection or care.
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Investigated drugs

  • VP16

    is a medication used in the trial as part of the intravenous chemotherapy regimen. It is used to help treat retinoblastoma by targeting and killing cancer cells, which can help preserve the eye without the need for external beam radiation.

  • Carboplatin

    is another medication used in the intravenous chemotherapy regimen. It works by interfering with the DNA of cancer cells, preventing them from growing and dividing. This helps in treating retinoblastoma and aims to preserve the eye.

  • Melphalan

    is administered intra-arterially in the trial. It is delivered directly to the ophthalmic artery to target the cancer cells in the eye more effectively. This method helps in treating retinoblastoma while minimizing the need for external radiation.

  • Laser Treatment

    is a local therapy used in combination with chemotherapy. It involves using a focused beam of light to destroy cancer cells in the eye, helping to preserve vision and avoid more invasive treatments.

  • Cryoapplication

    is a local treatment that involves freezing cancer cells to kill them. It is used alongside chemotherapy to enhance the treatment's effectiveness in preserving the eye.

  • I125 Radioactive Plaques

    are small devices placed on the eye to deliver targeted radiation to the tumor. This local treatment is used in combination with chemotherapy to help control the cancer while preserving the eye.

What is already known about the treatment

  • Melphalan

    Melphalan is administered intra-arterially, specifically targeting the ophthalmic artery, to treat retinoblastoma, a type of eye cancer. It is currently being studied for its effectiveness in preserving the eye without the need for external beam radiation. The main therapeutic indication is for retinoblastoma, particularly in cases where local treatments are possible. At the molecular level, Melphalan works by cross-linking DNA strands, which inhibits cancer cell replication and leads to cell death. It is classified as an alkylating agent in pharmacology.

  • Carboplatin

    Carboplatin is given intravenously as part of a chemotherapy regimen for retinoblastoma, aiming to preserve the eye without using external beam radiation. It is used in combination with other treatments to enhance its effectiveness. The drug works by binding to DNA and interfering with the cancer cell's ability to divide and grow. Carboplatin is classified as a platinum-based chemotherapy agent.

  • VP16 (Etoposide)

    Etoposide, also known as VP16, is administered intravenously and is used in combination with other drugs to treat retinoblastoma. It is part of a conservative treatment approach to avoid external beam radiation. Etoposide acts by inhibiting the enzyme topoisomerase II, which is essential for DNA replication, leading to cancer cell death. It is classified as a topoisomerase inhibitor in pharmacology.

Investigated diseases

Retinoblastoma – Retinoblastoma is a rare type of eye cancer that typically develops in early childhood, usually before the age of five. It originates in the retina, the light-sensitive lining at the back of the eye. The disease can affect one or both eyes and may cause symptoms such as a white color in the pupil, eye redness, or vision problems. As the cancer progresses, it can lead to further complications, including the potential spread to other parts of the body. The progression of retinoblastoma can vary, with some cases remaining localized to the eye while others may become more aggressive. Early detection and intervention are crucial in managing the disease and preserving vision.
Trial detailsLast updated 7 Oct 2026
Age0-17PhasePhase IITrial ID2024-517275-21-00Protocol codeIC 2011-05Estimated enrolment133 patientsSponsorInstitut Curie

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