Not recruitingRare disease

Study of gemtuzumab ozogamicin and chemotherapy combinations in children with newly diagnosed acute myeloid leukemia, high-risk myelodysplastic syndrome, or myeloid 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

This clinical trial studies treatments for Acute Myeloid Leukemia (AML), high-risk Myelodysplastic Syndrome (MDS), and Myeloid Sarcoma in children under 18 years of age. The study tests different combinations of cancer medicines including Gemtuzumab Ozogamicin, Cytarabine, Mitoxantrone, Fludarabine, Busulfan, and Cyclophosphamide. These medications are given through an intravenous line (IV) directly into the bloodstream.

The main purpose of this study is to find the best treatment combination and dosing for children with these blood cancers. The study will compare different chemotherapy combinations and evaluate how well they work in treating the disease. It will also test different ways of preparing patients for stem cell transplantation, a procedure that replaces damaged bone marrow with healthy cells.

The study will track how patients respond to treatment by monitoring their blood and bone marrow. Researchers will look at how long patients stay free of disease, how well they tolerate the treatments, and any side effects that may occur. The study will continue to follow patients for up to 10 years to understand the long-term effects of these treatments.

The research process

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

Step 1
Initial diagnosis and trial entry
The trial is for patients under 18 years old with acute myeloid leukemia (AML), high-risk myelodysplastic syndrome, or isolated myeloid sarcoma
Before starting treatment, your heart function will be checked to ensure it meets required standards
A pregnancy test is required for female patients who can become pregnant
Step 2
First phase of treatment (Induction)
You will receive one of two treatment combinations through intravenous infusion:
Option 1: mitoxantrone with cytarabine
Option 2: liposomal daunorubicin with cytarabine
The medicine gemtuzumab ozogamicin may be added to your treatment
Your bone marrow will be examined after this phase to check treatment response
Step 3
Second phase of treatment (Consolidation)
If your disease responds well to initial treatment, you will receive one of two consolidation treatments:
Option 1: High-dose cytarabine
Option 2: Fludarabine combined with cytarabine
Step 4
Stem cell transplant (if needed)
Some patients will need a stem cell transplant
If required, you will receive one of two preparation treatments:
Option 1: Strong preparation with busulfan and cyclophosphamide
Option 2: Gentler preparation with fludarabine and busulfan
Your blood cell counts will be monitored regularly during recovery
Step 5
Follow-up period
Regular check-ups will continue to monitor your health
Heart function will be checked for up to 10 years
Additional health monitoring will continue until the end of the study in 2033

Who can join the trial?

10 criteria

Patient must be under 18 years old at the time of entering the trial
Must have a new diagnosis of acute myeloid leukemia (AML), high-risk myelodysplastic syndrome (with more than 10% abnormal cells in bone marrow), or isolated myeloid sarcoma
No previous chemotherapy or biological therapy for these conditions, except as allowed in the protocol
Must have normal heart function (measured as fractional shortening ≥28% or ejection fraction ≥55%)
Must have normal kidney function (measured by a test called creatinine clearance)
Must have normal liver function (measured by bilirubin and liver enzyme tests)
Must be physically fit enough to receive the study treatment
For female patients who can become pregnant, must have a negative pregnancy test
Patients who can have children must agree to use effective birth control during treatment and for 12 months after the last treatment
Patient and/or parent/legal guardian must provide written informed consent

Who cannot join the trial?

13 criteria

Age below 16 years old
Previous chemotherapy treatment for acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS)
Known allergies to any of the study medications (gemtuzumab ozogamicin, cytarabine, mitoxantrone, liposomal daunorubicin)
Severe heart problems, including heart failure or significant heart rhythm abnormalities
Severe liver dysfunction (poor liver function)
Severe kidney dysfunction (poor kidney function)
Active uncontrolled infections
Pregnancy or breastfeeding
Participation in other clinical trials within the last 30 days
Mental conditions that prevent understanding of the study requirements
Active second cancer (other than treated non-melanoma skin cancer or cervical carcinoma in situ)
HIV positive status
Active hepatitis B or C infection

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Verified sites

All sites with verified contact details – recruitment status may not be available; ask directly

Assistance Publique Hopitaux De Marseille
Verified
Marseille, France
Assistance Publique Hopitaux De Paris
Verified
Paris, France
CHRU De Nancy
Responsive
Vandoeuvre Les Nancy, France

Trial locations

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Investigated drugs

Gemtuzumab ozogamicin
is a targeted therapy medication used to treat acute myeloid leukemia. It works by attaching to cancer cells and delivering a substance that can kill them.
Cytarabine
is a chemotherapy medication that works by interfering with DNA synthesis, which helps stop cancer cells from growing and dividing.
Mitoxantrone
is a chemotherapy medication that works by blocking an enzyme called topoisomerase II, which cancer cells need to divide and grow.
Liposomal daunorubicin
is a special form of chemotherapy medication enclosed in tiny fat particles, which helps deliver the medicine more effectively to cancer cells while potentially reducing side effects.
Fludarabine
is a chemotherapy medication that works by slowing or stopping the growth of cancer cells.
Busulfan
is a chemotherapy medication used before bone marrow transplants to prepare the body by destroying abnormal cells in the bone marrow.

What is already known about the treatment

Based on the clinical trial data, here are the medication descriptions: Gemtuzumab ozogamicin - A targeted cancer medication administered intravenously that combines an antibody with a chemotherapy drug, specifically designed to treat acute myeloid leukemia (AML). This medication works by attaching to CD33 proteins found on leukemia cells and delivering toxic substances directly to cancer cells, while minimizing damage to healthy cells. It belongs to the class of antibody-drug conjugates and is typically used in combination with standard chemotherapy during induction treatment. Mitoxantrone - An anthracenedione chemotherapy drug given through intravenous infusion, used in the treatment of various cancers including acute myeloid leukemia. This medication works by interfering with DNA repair and synthesis in cancer cells, preventing them from multiplying. It belongs to the class of antineoplastic agents and is often used in combination with other chemotherapy drugs like cytarabine. Cytarabine - A chemotherapy medication administered intravenously that is fundamental in treating acute myeloid leukemia and other blood cancers. It works by interfering with DNA synthesis during cell division, preventing cancer cells from growing and multiplying. This antimetabolite drug can be given in different doses, including high-dose regimens for consolidation therapy. Liposomal daunorubicin - An advanced form of daunorubicin chemotherapy enclosed in tiny fat particles (liposomes), administered through intravenous infusion. This anthracycline medication works by damaging the DNA of cancer cells and preventing them from multiplying, while the liposomal formulation helps reduce side effects and improve drug delivery to cancer cells.

Investigated diseases

Acute Myeloid Leukemia (AML)
A blood and bone marrow cancer that develops when immature blood cells called myeloid cells begin growing abnormally. These abnormal cells build up in the bone marrow and interfere with the production of normal blood cells. The disease progresses rapidly, affecting the production of red blood cells, white blood cells, and platelets.
High-Risk Myelodysplastic Syndrome (MDS)
A group of blood disorders where bone marrow fails to produce enough healthy blood cells. In high-risk MDS, more than 10% of cells in the bone marrow are immature cells called blasts. The condition causes the bone marrow to produce abnormal blood cells that don't develop properly and can't function normally.
Myeloid Sarcoma
A rare condition where myeloid cells form a tumor mass outside the bone marrow. The abnormal myeloid cells can develop in various parts of the body, forming solid tumors in soft tissues or bones. This condition can occur on its own (de novo) or develop as a result of other blood disorders (secondary).