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

3 1 1 1

What is this study about?

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.

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

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

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

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

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 Study?

  • 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 Study?

  • 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

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name City Country Status
University Hospital Of Clermont-Ferrand Clermont Ferrand France
Centre Hospitalier Universitaire De Lille Lille France
Centre Hospitalier Regional Et Universitaire De Brest Brest France
Oncopole Claudius Regaud Toulouse France
CHU Grenoble Alpes La Tronche France

Other Sites

Site Name City Country Status
Centre Hospitalier Universitaire Rouen Rouen France
Centre Hospitalier Universitaire De Poitiers Poitiers France
Robert Debre University Hospital Paris France
Centre Hospitalier Universitaire De Montpellier Montpellier France
Assistance Publique Hopitaux De Paris Paris France
Centre Hospitalier Universitaire De Nantes Nantes France
Centre Hospitalier Universitaire De Nice Nice France
Centre Hospitalier Universitaire Amiens Picardie Amiens France
Centre Hospitalier Universitaire De Rennes Rennes France
Centre Hospitalier Lyon Sud Pierre Benite France
Centre Hospitalier Universitaire De Caen Normandie Caen France
Centre Hospitalier Universitaire De Bordeaux Bordeaux France
University Of Bordeaux Talence France
Our Lady’s Childrens Hospital Dublin Ireland
Cwypdm Hwavuppvehi Uuirckqzxvxqc Rxped Reims France
Cqgtbs Hqilaspslbs Ep Urqtooqrnfpke Dc Lwytzqh Limoges France
Cyrbjy Hlozeqntzee Umfwzgwpjygrw Dw Dittg Dijon France
Araxkhcmod Ppqsvxxs Hxzjncdo Dj Pgpkn Paris France
Akbttyhbvj Plgefpvv Hhhkwbxr Dc Meskbrpgn Marseille France
Bvyemvhb Uxcrxysdtr Hmdyvtjs Ckhbvh Besançon France
Clkidv Hzwpjpsuxld Rueztojd Uqdyhecsqecbu Df Tdvdh Tours France
Ckpw Dp Nlfbl Vandoeuvre Les Nancy France
Ctxzrq Hijpfrjkzdf Rzrazssf Dqmhumpvdpbejx Angers France
Ilcvhlxd dl Czekbrfagejs Hwhrbkttqhs Uslhiqiyygxot dq Sovto Ejxykaw (fynoegr Saint Priest En Jarez France
Hfphkkxj Urzkfjeftdtswl Sltnxxwwgo &pyozia Hsmajht da Hpbfuupecmw STRASBOURG, Alsace France

Want to learn more about this study or check if you can participate? Contact us.

Trial status

Country Status Recruitment Start
France France
Not recruiting
31.01.2025
Ireland Ireland
Not recruiting
31.01.2025

Trial locations

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.

Cyclophosphamide is a chemotherapy medication that works by damaging the DNA of cancer cells, which prevents them from multiplying.

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).

Trial ID:
2024-516112-21-00
Protocol code:
RG_14-088
NCT ID:
NCT02724163
Trial Phase:
Therapeutic confirmatory (Phase III)

Other Trials to Consider

  • Venetoclax added to fludarabine, cytarabine and gemtuzumab ozogamicin (drug combination) in children with relapsed acute myeloid leukemia

    Recruiting

    3 1 1 1
    Investigated diseases:
    Austria Belgium Czechia Denmark Finland France +8
  • Study of Revumenib and Chemotherapy for Patients with Newly Diagnosed Acute Myeloid Leukemia with NPM1 Gene Mutation

    Recruiting

    3 1 1
    Investigated diseases:
    Austria Belgium Czechia France Germany Greece +7