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Carboplatin with drug combination for children with unilateral retinoblastoma undergoing enucleation: study of tumor biomarkers and relapse risk

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

The study focuses on retinoblastoma, a rare eye cancer that usually affects children and can require removal of the affected eye (enucleation). Participants receive chemotherapy that may be given before or after surgery and includes the drugs vincristine, topotecan, etoposide, cyclophosphamide, carboplatin and thiotepa. The main purpose is to determine whether tissue and blood markers can predict the risk of the disease returning.

Patients who need enucleation are enrolled and their tumor tissue, blood, eye fluid and spinal fluid are collected for analysis. After surgery, additional chemotherapy may be given, and the children are followed for several years to monitor for any return of cancer or other health events. Information from the samples and imaging studies is used to look for patterns that relate to outcomes, while safety of the treatment is also observed.

The research process

The trial runs in 9 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Baseline Evaluation

    You will undergo an eye examination and imaging tests, including an MRI scan, to assess the size and location of the tumor.

    Samples of blood, cerebrospinal fluid (CSF), and aqueous humor (AH) will be collected to measure biomarkers before any treatment begins.

  2. Step 2

    Decision on Eye Removal (Enucleation)

    If the tumor cannot be treated with medication alone, the study protocol calls for removal of the affected eye, known as enucleation.

    If the tumor may respond to medication first, you will receive chemotherapy before the eye is removed.

  3. Step 3

    Neoadjuvant Chemotherapy (If Required)

    You will receive a series of chemotherapy drugs through infusion or injection. The medicines and their doses are:

    Vincristine sulfate: 1.5 mg per square meter of body‑surface area, given by infusion.

    Topotecan: 0.4 mg, given by injection.

    Etoposide: 300 mg per square meter, given by infusion.

    Cyclophosphamide: 1950 mg per square meter, given by infusion.

    Carboplatin: 500 mg per square meter, given by infusion.

    Thiotepa: 300 mg per square meter, given by infusion.

    The exact schedule (how often the drugs are given) and the total number of cycles are defined by the study protocol.

  4. Step 4

    Surgical Removal of the Eye (Enucleation)

    After the chemotherapy phase (or immediately if chemotherapy is not needed), you will have surgery to remove the affected eye.

    During the procedure, the tumor tissue is collected for detailed laboratory analysis.

  5. Step 5

    Pathology and Biomarker Analysis

    The removed tumor is examined under a microscope to identify histological risk factors.

    Laboratory tests will also look for molecular subtypes and other biomarkers in the tumor tissue.

  6. Step 6

    Adjuvant Chemotherapy

    Following surgery, additional chemotherapy may be given to reduce the risk of tumor returning. The same medicines listed for neoadjuvant therapy are used:

    Vincristine sulfate (1.5 mg/m²), Topotecan (0.4 mg), Etoposide (300 mg/m²), Cyclophosphamide (1950 mg/m²), Carboplatin (500 mg/m²), and Thiotepa (300 mg/m²).

    The frequency and total duration of these treatments are specified in the study protocol.

  7. Step 7

    Orbital Irradiation (If Indicated)

    In some cases, targeted radiation therapy to the eye socket may be recommended to further lower the chance of relapse.

    The details of the radiation dose and schedule are determined by the study investigators.

  8. Step 8

    Follow‑Up Visits

    You will attend regular follow‑up appointments that include eye examinations, imaging (such as MRI), and collection of blood, CSF, and AH samples to monitor for any signs of tumor return.

    These visits continue for the duration of the study to assess relapse‑free survival and overall health.

  9. Step 9

    Study Completion

    At the end of the study period, a final assessment will be performed to evaluate long‑term outcomes, including survival rates and any late side effects.

Who can join the trial?

17 criteria

  • Written informed consent (and assent if the child is old enough) must be signed according to the hospital or country rules.
  • The child must have unilateral retinoblastoma (cancer in one eye only) that fits one of the following descriptions:
    • Very large eye (called buphthalmos) or high pressure inside the eye (raised intraocular pressure) – stage cT3c.
    • Infection‑like inflammation of the eye socket without germs (aseptic orbital cellulitis) – stage cT3e.
    • Evidence on scans that the tumor has spread outside the eye to the optic nerve or surrounding tissue – stage cT4a.
    • Visible tumor outside the eye causing the eye to bulge (proptosis) or a mass in the eye socket – stage cT4b.
    • Extensive non‑familial (not inherited) tumor inside one eye that needs the eye to be removed (primary enucleation).
    • The child must not have received any chemotherapy or radiotherapy for retinoblastoma or any other cancer before joining the study.
    • There must be no medical reason that would stop the planned treatment (no contraindications).
    • The child must have adequate organ function, meaning:
      • Blood (hematopoietic) function: enough white blood cells (neutrophils greater than 1.0 × 10⁹ per liter) and enough platelets (greater than 100 × 10⁹ per liter) to help fight infection and stop bleeding.
      • Kidney (renal) function: blood test for creatinine must be less than 1.5 times the normal upper limit for the child’s age, and the kidney’s filtering ability should be normal (checked with the Schwartz formula).
      • Ear safety: hearing test score must be lower than Grade II on the Brock ototoxicity grading scale (means no serious hearing damage).
      • Heart function: an ultrasound of the heart (echocardiography) must show normal left‑ventricular performance.
      • Liver function (Study A only): bilirubin level ≤ 1.5 × upper limit, and liver enzymes ALT, AST less than 3 × upper limit, alkaline phosphatase (ALP) less than 2.5 × upper limit for age.
      • If the patient could become pregnant, a pregnancy test must be negative within 72 hours before the first dose of study medication.
      • Women who could become pregnant must agree to use a highly effective birth‑control method during screening, treatment, and for at least six months after the last dose. It is also recommended that the male partner use a condom with spermicide.
      • For Study B, the patient must be able to stay in the study for long‑term follow‑up.
      • The patient must be covered by national health insurance (or the equivalent system required by the country).

Who cannot join the trial?

12 criteria

  • Having retinoblastoma in both eyes, a family history of the disease, or a tumor in a third location (bilateral, familial or trilateral retinoblastoma).
  • Not meeting any of the study’s required inclusion characteristics (non‑compliance with inclusion criteria).
  • Having uncontrolled medical problems, mental health issues, family or social situations, or living far away that could make it hard to follow the study plan (uncontrolled medical, psychological, familial, sociological or geographical conditions).
  • Being enrolled in another experimental (interventional) study within one year after finishing treatment in this study, unless the cancer returns early (participation in another interventional study).
  • Not having a high‑quality orbital and brain MRI scan (diagnostic‑quality MRI) before eye removal surgery (enucleation).
  • Having a single‑eye retinoblastoma that needs chemotherapy before eye removal because of very enlarged eye (buphthalmos), infection‑like inflammation around the eye (aseptic orbital cellulitis) or high pressure inside the eye (raised intraocular pressure).
  • Having cancer that has grown outside the eye (extraocular extension).
  • Imaging that suggests the tumor may have reached behind the optic nerve (retrolaminar extension) and could affect the surgical cut line (surgical margin).
  • Imaging that suggests the tumor has spread beyond the white part of the eye (extrascleral extension).
  • Having cancer cells confirmed in lymph nodes through lab tests (lymph node involvement).
  • Having a single‑eye retinoblastoma that could be treated without removing the eye (amenable to conservative treatment).
  • Having cancer that has already spread to other parts of the body at diagnosis (metastatic extension), including lymph nodes, distant organs such as bone, lung, liver, or the brain beyond the optic nerve (such as optic chiasm invasion or meningeal disease).
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Investigated drugs

  • Vincristine

    is a chemotherapy medicine given by infusion. In this study it is used before eye removal to help shrink the retinoblastoma tumor by stopping cancer cells from dividing, making the surgery easier and reducing the chance of the disease coming back.

  • Topotecan

    is a chemotherapy drug given by injection. It works by interfering with the DNA of cancer cells, which can stop the tumor from growing. In the trial it is part of the pre‑surgery treatment to try to reduce the size of the retinoblastoma and improve outcomes after the eye is removed.

  • Etoposide

    is a chemotherapy agent administered through an infusion. It damages the DNA of rapidly growing cells, helping to kill cancer cells. In this trial it is used as part of the neoadjuvant (pre‑surgery) chemotherapy regimen to shrink the eye tumor before enucleation.

  • Cyclophosphamide

    is a chemotherapy drug given by infusion. It works by preventing cancer cells from multiplying. In the study it is combined with other medicines to treat the retinoblastoma before the eye is surgically removed, aiming to lower the risk of the cancer returning.

  • Carboplatin

    is a chemotherapy medicine given by infusion. It damages the DNA of cancer cells, helping to stop tumor growth. In this trial it is used before enucleation to reduce the size of the retinoblastoma and to improve the chances of long‑term remission.

  • Thiotepa

    is a chemotherapy drug delivered by infusion. It works by interfering with the DNA of cancer cells, leading to their death. In the study it is part of the pre‑operative chemotherapy plan to help shrink the eye tumor and support better surgical and survival outcomes.

What is already known about the treatment

  • Vincristine

    Vincristine is given as a sterile solution that is slowly injected into a vein (IV infusion). It is an established chemotherapy drug that has been used for many years and is well described in medical literature. It is mainly used to treat various cancers, including childhood solid tumors such as retinoblastoma. It works by stopping the building blocks of DNA from joining together, which blocks the cancer cells from dividing, and it belongs to the class of antimitotic (vinca alkaloid) agents.

  • Topotecan

    Topotecan is supplied as a concentrate that is mixed with fluid and then injected into a vein (IV injection). It is an approved cancer medicine with extensive clinical data and is listed in standard drug references. It is used to treat several solid tumors, including eye cancers like retinoblastoma when other options are needed. It blocks an enzyme called topoisomerase I, preventing DNA from being copied correctly, and it is classified as a topoisomerase inhibitor chemotherapy.

  • Etoposide

    Etoposide comes as a liquid that is diluted and given by IV infusion. It is a well‑known chemotherapy drug that has been studied for decades and is included in many treatment guidelines. It is used for many cancers, such as lung cancer, testicular cancer, and retinoblastoma in pediatric protocols. It works by stopping an enzyme needed for DNA replication, causing cancer cells to die, and it belongs to the class of topoisomerase II inhibitor agents.

  • Cyclophosphamide

    Cyclophosphamide is provided as a solution that is diluted and administered through an IV infusion. It is a long‑standing chemotherapy agent with a large body of research supporting its use. It is used to treat a wide range of cancers, including blood cancers and solid tumors like retinoblastoma when combined with other drugs. It acts as an alkylating agent, adding chemical groups to DNA that break the strands, and it is classified as an alkylating chemotherapy.

  • Carboplatin

    Carboplatin is supplied as a liquid that is mixed with fluid and given by IV infusion. It is an approved cancer drug that has been widely studied and is part of many standard treatment regimens. It is used for cancers such as ovarian, lung, and pediatric eye tumors such as retinoblastoma. It works by attaching to DNA and forming cross‑links that stop the cells from copying their genetic material, and it belongs to the class of platinum‑based chemotherapy agents.

  • Thiotepa

    Thiotepa is a powder that is reconstituted into a solution for IV infusion. It is an established chemotherapy drug with a solid record in medical literature. It is used for several cancers, including brain tumors and retinoblastoma in certain treatment plans. It interferes with DNA by adding chemical groups that prevent the strands from separating, and it is classified as an alkylating agent.

Investigated diseases

Retinoblastoma - Retinoblastoma is a cancer that begins in the retina, the light‑sensing tissue at the back of the eye. It typically appears in early childhood and may cause the eye to enlarge or develop a white pupil. As the tumor grows it can extend into nearby structures such as the optic nerve. The disease may affect one eye (unilateral) or both eyes (bilateral). Progression is tracked by changes in tumor size and spread beyond the eye.
Trial detailsLast updated 8 Nov 2026
Age0-17PhasePhase IITrial ID2024-515962-15-00Protocol codeIC 2023-09Estimated enrolment50 patientsSponsorInstitut Curie

sourced from the EU Clinical Trials Register and site verification

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