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Treatment Study of Arsenic Trioxide, Tretinoin, and Gemtuzumab Ozogamicin for Children and Adolescents with Acute Promyelocytic Leukemia

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What is this trial about?

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

This study looks at acute promyelocytic leukemia (APL) in children and adolescents. APL is a blood cancer that starts in very early white blood cells. The purpose of the study is to learn how well treatment works and how safe it is for young people with newly diagnosed APL. The treatment uses arsenic trioxide together with tretinoin, and in some patients with higher-risk disease it also includes gemtuzumab ozogamicin. Other medicines listed in the study materials include cytarabine, methotrexate, and methylprednisolone, which may be used as part of the treatment plan or support care. Some medicines are given by mouth and others are given into a vein or into the fluid around the spine, depending on the treatment step.

The study follows a treatment course made up of several phases. At first, treatment is given to bring the leukemia under control. After that, more treatment is given over time to help keep the disease from returning. Some children may receive only the main drug combination, while others may receive an added medicine based on the type of APL and the level of risk. During the study, doctors watch for side effects and monitor how the disease responds to treatment.

The study is designed for newly diagnosed APL in children and adolescents and compares treatment approaches for different risk groups. The study also examines whether the treatment causes any serious problems and how long children stay in the hospital during therapy.

The research process

"\n \n {\n \"timeline\": [\n {\n \"step\": \"study treatment start\",\n \"step_number\": \"1\",\n \"details\": [\n \"After joining the study, you begin treatment for acute promyelocytic leukemia (APL), a type of blood cancer.\",\n \"The treatment uses ATRA and ATO. ATRA means all-trans retinoic acid, and ATO means arsenic trioxide.\",\n \"If your disease is standard risk, the study uses the combination of ATO and ATRA.\",\n \"If your disease is high risk, the study explores a combination of ATRA, ATO, and GO. GO means gemtuzumab ozogamicin.\"\n ]\n },\n {\n \"step\": \"medicines during treatment\",\n \"step_number\": \"2\",\n \"details\": [\n \"Tretinoin (ATRA) is taken by mouth as soft capsules at a dose of 25 mg/m² each day. m² means square meter of body surface area. The source data does not specify the full duration.\",\n \"Arsenic trioxide (ATO) is given into a vein at a dose of 0.15 mg/kg. kg means kilogram of body weight. The source data does not specify the full frequency or duration.\",\n \"For some patients, gemtuzumab ozogamicin (GO) is given into a vein at a dose of 3 mg/m². The source data does not specify the full frequency or duration.\",\n \"Some medicines are also listed for use by injection into the fluid around the spinal cord, called intrathecal use. These include cytarabine 30 mg, methotrexate 12 mg, and methylprednisolone acetate 10 mg. The source data does not specify when or how often these are given.\"\n ]\n },\n {\n \"step\": \"treatment phases and follow-up during the study\",\n \"step_number\": \"3\",\n \"details\": [\n \"The study follows the results of treatment over time, including whether complete remission is reached. Complete remission means that signs of the disease are no longer seen by standard tests.\",\n \"The study also checks whether molecular remission is reached after three consolidation courses. Consolidation courses are treatment phases given after the first treatment phase to help keep the disease under control.\",\n \"The study monitors whether the disease returns, whether there are side effects, and whether any early death occurs during treatment.\",\n \"The study follows the main outcome for up to 2 years from diagnosis and also aims to measure results at 3 years.\"\n ]\n },\n {\n \"step\": \"study end\",\n \"step_number\": \"4\",\n \"details\": [\n \"The study ends after the planned treatment and follow-up assessments are completed.\",\n \"The trial has an estimated recruitment period from 2019-10-19 to 2027-10-31.\"\n ]\n }\n ]\n }\n \n"

Who can join the trial?

6 criteria

  • Have a new diagnosis of acute promyelocytic leukemia (APL), confirmed by finding the PML/RARα fusion gene in the leukemia cells. A fusion gene is an abnormal joining of two genes.
  • Be younger than 18 years old.
  • Have written informed consent from a parent or legal guardian. Informed consent means permission given after the study has been explained.
  • If the participant is female and can become pregnant, have a negative pregnancy test using a blood test called beta-HCG.
  • If the participant can have children, be willing to avoid becoming pregnant or causing a pregnancy during the study and for 3 months after the last dose of the study medicine.
  • Be willing to work with the study doctor on the most suitable way to prevent pregnancy during the study period.

Who cannot join the trial?

14 criteria

  • If the diagnosis of APL is later found to be incorrect because the PML/RARα rearrangement is not present, the patient cannot stay in the study. This rearrangement is a specific change in the leukemia cells that confirms this type of leukemia.
  • Significant liver dysfunction, meaning the liver is not working well enough, including:
    • Bilirubin higher than 3 mg/dL. Bilirubin is a substance made when the body breaks down red blood cells.
    • ALT or AST higher than 5 times the normal value. These are liver enzymes, which are proteins that can rise when the liver is injured or inflamed.
    • Kidney function that is too poor, shown by creatinine higher than 2 times the normal value for age. Creatinine is a waste product that helps show how well the kidneys are working.
    • Heart rhythm problems or other major heart findings, including:
      • Congenital long QT syndrome, a condition present from birth that affects the heart’s electrical recovery time.
      • A history of or current ventricular or atrial tachyarrhythmia, which means an abnormally fast heart rhythm from the lower or upper chambers of the heart.
      • Resting bradycardia lower than 50 beats per minute. Bradycardia means a slow heart rate.
      • QTc longer than 450 msec on the screening ECG. QTc is a measure of the heart’s electrical timing, and an ECG is a test that records the heart’s activity.
      • L-FEV lower than 50% or LV-FS lower than 28%. These are heart function measurements that show how well the heart pumps.
      • Neuropathy, which means nerve damage that can cause pain, numbness, tingling, or weakness.
      • A concurrent active malignancy, meaning another cancer is present at the same time.
      • Uncontrolled life-threatening infections, meaning a severe infection that is not being kept under control.
      • Being pregnant or breastfeeding.
      • Having already received a different treatment before entering the study, because APL was not suspected at first or because ATRA and/or ATO were not available. ATRA and ATO are study medicines used in this trial.
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Investigated drugs

  • Aracytin

    is a brand name for cytarabine. In this trial, it is given by injection into the fluid around the brain and spinal cord to help treat leukemia cells that may be hiding in the central nervous system.

  • Cytarabine Accord

    is another brand of cytarabine. It is also given into the spinal fluid and is used to help control leukemia cells in the brain and spinal cord area.

  • Depo-Medrol

    is a brand name for methylprednisolone acetate. It is given into the spinal fluid as part of the treatment plan, likely to help reduce inflammation and support the intrathecal therapy used in the study.

  • Methotrexate

    is a medicine used to treat cancer. In this trial it is given into the spinal fluid to help prevent or treat leukemia cells in the central nervous system.

  • Arsenic trioxide

    is one of the main study medicines. It is given through a vein and helps kill leukemia cells. It is a key part of treatment for acute promyelocytic leukemia in this study.

  • Vesanoid

    is a brand name for tretinoin. It is taken by mouth and helps leukemia cells mature into more normal blood cells, which is an important part of treating acute promyelocytic leukemia.

What is already known about the treatment

  • cytarabine

    Cytarabine is given as a liquid injection or infusion, and in this trial it is used by the intrathecal route, which means it is placed into the fluid around the brain and spinal cord. It is an established anti-cancer medicine that is widely used in medical practice and well described in the medical literature. It is mainly used to treat blood cancers such as leukemia, and it works by blocking cancer cells from making new DNA, which stops them from growing and dividing. It belongs to the antimetabolite group of anti-cancer medicines, specifically a pyrimidine analog.

  • methylprednisolone acetate

    Methylprednisolone acetate is given as an injection suspension, and in this trial it is used intrathecally, meaning it is delivered into the fluid around the brain and spinal cord. It is a well-known corticosteroid medicine that is commonly used in medicine and has been widely discussed in the medical literature. It is used to reduce inflammation and to help control swelling and irritation, including in conditions where the immune system and inflammation need to be suppressed. It belongs to the corticosteroid class of medicines and works by lowering the production of substances in the body that cause inflammation.

  • methotrexate

    Methotrexate is given as a liquid injection, and in this trial it is used intrathecally, so it is placed into the fluid around the brain and spinal cord. It is a long-established anti-cancer medicine that is widely used in medicine and strongly supported by medical literature. It is mainly used to treat leukemia and other cancers, and it works by blocking an enzyme needed for cells to make DNA, which slows or stops the growth of rapidly dividing cells. It belongs to the antimetabolite group of anti-cancer medicines, specifically a folate antagonist.

  • arsenic trioxide

    Arsenic trioxide is given as an intravenous infusion, meaning it is delivered directly into a vein. It is an established anti-cancer medicine and is well known in modern medical practice and literature, especially for acute promyelocytic leukemia. It is used to treat this type of leukemia because it helps cancer cells die and lose their ability to keep growing, partly by affecting key control systems inside the cell. It belongs to the anti-cancer medicine group and is classified as a targeted or other anti-neoplastic agent.

  • tretinoin

    Tretinoin is taken by mouth as a soft capsule. It is a well-established medicine in cancer care and is widely described in the medical literature, especially for acute promyelocytic leukemia. It is used to help immature leukemia cells mature into normal blood cells instead of continuing to grow as cancer cells. It belongs to the retinoid group of medicines, and it works by turning on signals in the cell that guide normal cell growth and development.

  • gemtuzumab ozogamicin

    Gemtuzumab ozogamicin is given as an intravenous infusion, meaning it is delivered directly into a vein. It is a newer anti-cancer medicine that is recognized in current medical practice and described in the medical literature, mainly for certain types of leukemia. It is used to target leukemia cells and deliver a cell-killing substance into them, which helps destroy the cancer cells more specifically. It belongs to the group of antibody-linked anti-cancer medicines, also called antibody-drug conjugates.

Investigated diseases

Acute promyelocytic leukemia - Acute promyelocytic leukemia is a fast-growing type of blood cancer that starts in immature white blood cells called promyelocytes. It develops when these abnormal cells multiply in the bone marrow and crowd out normal blood cells. As the disease progresses, the number of abnormal cells rises and the blood and bone marrow become increasingly filled with these cells. This can lead to worsening bone marrow failure and spread of leukemia cells in the blood. In children and adolescents, it follows the same basic pattern of rapid cell buildup and progression.
Trial detailsLast updated 7 Oct 2026
Age0-17PhasePhase IITrial ID2024-518669-83-00Protocol codeICC APL Study 02Estimated enrolment99 patientsSponsorAssociazione Italiana Ematologia Oncologia Pediatrica

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