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Not yet recruitingRare disease

Phase I/II Study of Dinutuximab Beta, Irinotecan, and Sirolimus in Children, Adolescents, and Adults with Relapsed or Refractory Bone Sarcoma

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 rare bone cancers such as osteosarcoma, chondrosarcoma, Ewing sarcoma, Ewing‑like tumors with rare fusions, and CIC‑DUX4 bone sarcoma. Participants will receive a combination of three medications: dinutuximab beta, which is given through a vein over several days, irinotecan given by vein on specific days, and the pill form of sirolimus taken daily.

The purpose of the trial is to find the safest amount of the first drug to use together with the other two and to see how well the disease can be kept from growing for at least 16 weeks. Treatment is organized in 28‑day cycles; during each cycle the infusion of the first medication runs continuously for four days at the start and again in the middle of the cycle, the second medication is given on the first and fifteenth days, and the oral medication is taken every day. Patients are monitored with regular imaging scans and blood tests, and terms such as “continuous infusion” (medicine delivered slowly over many hours), “maximum tolerated dose” (the highest amount that does not cause unacceptable side effects), “dose‑limiting toxicity” (side effects that stop dose increases), and “progression‑free survival” (time the disease does not get worse) are explained in simple language throughout the study.

The research process

The trial runs in 6 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    First day of treatment

    On day 1 a continuous intravenous infusion of dinutuximab beta is started and is given for four consecutive days.

    On the same day an intravenous infusion of irinotecan is administered.

    A tablet of sirolimus is taken by mouth each day, beginning on day 1.

  2. Step 2

    Continuation of infusion

    The infusion of dinutuximab beta continues without interruption through day 4.

    The daily oral dose of sirolimus is taken each day while the infusion is running.

  3. Step 3

    Mid‑cycle treatment

    On day 15 a second continuous intravenous infusion of dinutuximab beta is started and is given for four days (days 15 to 18).

    On day 15 an intravenous infusion of irinotecan is also administered.

    The daily oral dose of sirolimus continues without interruption.

  4. Step 4

    Completion of first 28‑day course

    The first 28‑day treatment course ends after the day‑18 infusion of dinutuximab beta is finished and the daily oral sirolimus has been taken through day 28.

    During this first course the medical team assesses any dose‑limiting toxicities, which are side effects that are severe enough to stop increasing the dose.

  5. Step 5

    Subsequent treatment cycles

    If the treatment continues, each new 28‑day cycle repeats the same schedule: a four‑day infusion of dinutuximab beta starting on day 1, another four‑day infusion starting on day 15, intravenous irinotecan on days 1 and 15, and daily oral sirolimus throughout the cycle.

  6. Step 6

    Progression‑free survival assessment

    At or before 16 weeks after the first dose of study drugs, imaging tests are performed to determine whether the disease has progressed. this assessment follows standard criteria for measuring tumor response.

Who can join the trial?

20 criteria

  • Must have a tissue sample (biopsy) that shows one of the following bone cancers: osteosarcoma, Ewing sarcoma, chondrosarcoma, CIC‑DUX4 bone sarcoma, or Ewing‑like tumor with rare fusions; the cancer must have come back (relapsed) and be able to be removed now or possibly after the study treatment.
  • Women who could become pregnant must have a negative pregnancy test taken within 72 hours before the first dose.
  • Women who are sexually active and could become pregnant must use a highly effective birth‑control method during the study and for at least 6 months after the last dose; men who are sexually active must use condoms during the study and for at least 3 months after the last dose.
  • Written permission (informed consent) must be signed by the patient (and by a parent or legal guardian if the patient is a minor) and the patient must give age‑appropriate agreement (assent) before any study procedures.
  • The patient must be able to attend study visits, follow the study plan, and manage any side effects that occur.
  • The patient must be covered by a social security system or similar benefit as required in their country.
  • The cancer must be refractory (not responding) or relapsed and must not have spread to the brain (no brain metastasis).
  • The patient may have had between 1 and 3 previous treatments for metastatic or locally advanced sarcoma.
  • Age must be at least 6 years and no more than 40 years at the time of enrollment.
  • Performance status (how well the patient can function) must be at least 70 % using the Lansky Play score for children 16 years or younger, or the Karnofsky score for patients older than 16 years.
  • Life expectancy must be expected to be at least 4 months.
  • For Phase I participants: imaging (CT‑scan, MRI, or PET‑scan) done 7‑28 days before the first dose must show measurable disease that is getting worse.
  • For Phase II participants: imaging done 7‑28 days before the first dose must show measurable disease that meets one of these size rules – a tumor at least 10 mm on CT, a chest finding at least 20 mm on X‑ray, a lymph node at least 10 mm in short axis, or bone/marrow involvement seen on PET.
  • Heart function must be adequate, with a shortening fraction of at least 29 % (more than 35 % if younger than 3 years) and a left ventricular ejection fraction of at least 50 % on an echocardiogram performed 7‑28 days before the first dose.
  • Blood and organ tests done within 21 days before the first dose must show:
    • Absolute neutrophil count (a type of white blood cell) ≥ 1.0 × 10⁹/L.
    • Platelet count ≥ 100 × 10⁹/L.
    • INR (a clotting test) ≤ 1.5 and normal aPTT (another clotting test).
    • Serum creatinine (kidney function) ≤ 1.5 times the normal upper limit, or kidney clearance ≥ 60 mL/min/1.73 m².
    • Total bilirubin (liver function) ≤ 1.5 times the normal upper limit (≤ 3.0 times if the patient has Gilbert’s syndrome).
    • Liver enzymes ALT and AST ≤ 2.5 times the normal upper limit (or ≤ 5 times if the liver is involved by tumor).

Who cannot join the trial?

21 criteria

  • You do not have any tumor that can be seen and measured for the study (no visible or evaluable target tumor).
  • You have type I diabetes or diabetes that is not well‑controlled (blood sugar level after fasting is more than 1.5 times the normal upper limit).
  • You have an active viral infection such as hepatitis B, hepatitis C, HIV, or any other serious infection that is not under control (grade 2 or higher on a standard side‑effect scale).
  • You have a serious wound, ulcer, or bone fracture that has not healed at the time of enrollment.
  • You have any ongoing treatment‑related side effect that is grade 2 or higher, except for low white‑blood‑cell count (lymphopenia), hair loss (alopecia), ear damage (ototoxicity), or nerve damage (peripheral neuropathy).
  • You received any cancer‑directed medication within 21 days before the first dose, or within five times the drug’s half‑life (the time it takes for half of the drug to leave your body), whichever is shorter.
  • You had a high‑dose chemotherapy with stem‑cell rescue (myeloablative therapy) within 8 weeks before the first dose.
  • You underwent non‑palliative radiation therapy (radiation intended to cure or control disease) within 21 days before the first dose.
  • You had major surgery or a serious injury within 21 days before the first dose. (Procedures such as feeding tube placement, brain‑shunt surgery, endoscopic brain surgery, tumor biopsy, or placing a central line are not considered major surgery, but they still require at least a 48‑hour gap before starting the study drug.)
  • You have a history of another cancer or any medical condition that the study doctor believes could put you at risk or affect the study results.
  • You are currently taking blood thinners that work by blocking vitamin K (Vitamin K antagonists).
  • You have previously taken drugs that block a protein called mTOR (mTOR inhibitors).
  • You are taking medicines that strongly affect the liver enzyme CYP450 3A4, which can speed up or slow down the study drugs.
  • You have used corticosteroid steroids (such as prednisone) within 14 days before the first dose.
  • You have a gastrointestinal (GI) problem that could prevent you from absorbing oral medication properly, such as chronic inflammatory bowel disease, ulcerative disease, uncontrolled nausea, vomiting, diarrhea, or a condition that causes poor nutrient absorption.
  • You have serious uncontrolled heart disease, including any irregular heartbeat (arrhythmia), heart failure that is worse than class II on the NYHA scale, unstable chest pain (angina), or new‑onset chest pain.
  • You have uncontrolled high blood pressure (hypertension): for patients 17 years old or younger, blood pressure above the 95th percentile for your age and weight that is not controlled with one medication; for patients older than 17, systolic pressure over 150 mmHg or diastolic pressure over 90 mmHg that is not controlled with one medication.
  • You have had a blood clot or blockage (thrombotic or embolic event) in the past six months.
  • You have interstitial lung disease, a condition that causes scarring and inflammation of the lung tissue.
  • You have uncontrolled high cholesterol or triglycerides (fasting cholesterol over 3 g/L or 7.75 mmol/L and triglycerides more than 2.5 times the upper normal limit).
  • You have a bleeding or clotting disorder that affects how your blood normally clots (abnormalities of coagulation or hemostasis).
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Investigated drugs

  • Dinutuximab beta

    is a laboratory‑made protein called a monoclonal antibody. It is designed to find and attach to a special marker (called GD2) that is found on many bone tumor cells. By binding to this marker, the drug helps the body’s immune system recognize and attack the cancer. In this study it is given through a vein as a continuous infusion over several days, and it is combined with other medicines to try to improve treatment results.

  • Sirolimus

    is a medicine that blocks a cell‑growth pathway known as mTOR. When this pathway is turned off, cancer cells may grow more slowly or stop growing. Sirolimus is taken by mouth every day during the treatment cycle. It works together with the other drugs to try to keep the tumor from spreading.

  • Irinotecan

    is a chemotherapy drug that interferes with the DNA inside cancer cells, making it harder for them to multiply. It is given through a vein on specific days of the treatment cycle. In this trial, irinotecan is used together with dinutuximab beta and sirolimus to attack the tumor from several angles.

What is already known about the treatment

  • Din​utuximab beta

    This medication is given as a solution for infusion that is administered intravenously, usually as a continuous infusion over four days. It is an approved drug for high‑risk neuroblastoma and is now being studied in clinical trials for relapsed bone tumors. Dinutuximab beta works by attaching to the GD2 protein on cancer cells, which then signals the immune system to attack those cells. It belongs to the class of monoclonal antibodies used as immunotherapy.

  • Sirolimus

    Sirolimus is taken by mouth in tablet form and is absorbed through the digestive system. It is an established immunosuppressant approved for preventing organ‑transplant rejection and is also explored as a cancer‑treatment option. The drug blocks the mTOR pathway, a key signal that tells cells to grow and divide, thereby slowing tumor growth. It is classified as an mTOR inhibitor and immunosuppressive agent.

  • Irinotecan hydrochloride trihydrate

    This agent is supplied as a powder that is reconstituted for intravenous infusion. It is an approved chemotherapy drug for colorectal cancer and is frequently used off‑label in trials for sarcomas. Irinotecan is changed inside the body to SN‑38, which stops the enzyme topoisomerase I from repairing DNA, leading to cancer‑cell death. It is categorized as a topoisomerase I inhibitor and a cytotoxic chemotherapy.

Investigated diseases

  • Osteosarcoma

    A malignant tumor that originates in the bone‑forming cells, most often affecting the long bones around the knee or shoulder. It typically begins as a painful swelling that may be mistaken for a sports injury. As the tumor grows, it destroys normal bone tissue and can expand into surrounding soft tissue. Cancer cells may break away and travel through the bloodstream, most commonly reaching the lungs. The disease often progresses quickly, requiring prompt monitoring of its size and spread.

  • Chondrosarcoma

    A cancer that arises from cartilage‑producing cells, usually found in the pelvis, shoulder, or ribs. It often presents as a deep, aching pain and a hard mass in the affected bone. The tumor slowly erodes the surrounding bone and can extend into nearby muscles. In some cases, cancer cells spread to other organs, especially the lungs. Growth is generally slower than in osteosarcoma, but the tumor can become larger over time.

  • Ewing sarcoma

    A fast‑growing cancer that develops in the bone or soft tissue of children and young adults. It commonly appears as a painful lump in the thigh, pelvis, or chest wall. The tumor rapidly invades bone and can break through the cortex into surrounding tissue. Cancer cells frequently travel through the blood to the lungs and other bones. The disease often spreads early, making regular imaging important.

  • Ewing‑like tumor with rare fusions

    A rare type of bone‑derived cancer that carries unusual genetic rearrangements different from classic Ewing sarcoma. It presents similarly with pain and swelling in the affected bone. The tumor can enlarge quickly and may extend into nearby soft tissue. Like other small‑round‑cell tumors, it has the potential to spread to the lungs and other skeletal sites. Because of its rarity, its behavior is monitored closely for changes in size and spread.

  • CIC‑DUX4 bone sarcoma

    A newly recognized malignant bone tumor driven by a CIC‑DUX4 gene fusion. It usually manifests as a painful, enlarging mass in the long bones or pelvis. The cancer grows aggressively, destroying bone and infiltrating surrounding muscles. Cells can disseminate through the bloodstream, often reaching the lungs. Ongoing observation focuses on tumor growth and any new areas of involvement.

Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase ITrial ID2026-525463-40-00Protocol code9422Estimated enrolment89 patientsSponsorLes Hopitaux Universitaires De Strasbourg

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