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Fludarabine with drug combination for older or frail patients with acute myeloid leukemia undergoing haploidentical stem cell transplant

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 patients with acute myeloid leukemia who are older or considered frail and therefore cannot tolerate very strong chemotherapy. The treatment plan uses a combination of medicines given through a vein, including fludarabine and treosulfan, followed by a transplant of blood‑forming stem cells from a half‑matched donor, known as haploidentical stem cell transplantation. After the transplant, patients receive additional medication such as cyclophosphamide, along with other drugs that help keep the immune system from attacking the new cells, for example ciclosporin and mycophenolate mofetil.

The purpose of the study is to evaluate how well this approach works in preventing the leukemia from returning. Participants first receive the chemotherapy drugs to prepare their bodies, then undergo the stem‑cell transplant, and afterward take medicines to reduce the risk of the donor cells causing problems. Follow‑up visits are scheduled over several months to monitor recovery and any side effects.

Reduced intensity conditioning means a milder pre‑transplant chemotherapy regimen designed to be easier on older or weaker patients. Haploidentical stem cell transplantation uses a donor who shares only half of the genetic markers, often a family member, making a transplant possible when a fully matched donor is unavailable. Post‑transplant cyclophosphamide is given shortly after the transplant to help prevent graft‑versus‑host disease, a condition where the new immune cells attack the patient’s own tissues. The study tracks how long patients stay free of disease, how quickly blood counts recover, and the overall safety of the treatment.

The research process

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

Step 1
Initial evaluation
After enrollment, a series of medical tests are performed to confirm eligibility and to establish a baseline. these may include blood work, imaging, and assessment of overall health.
Step 2
Start conditioning regimen
The conditioning phase begins with two intravenous medicines:
fludarabine is given by infusion at a dose of 60 mg per administration.
treosulfan is given by infusion at a dose of 20 g per administration.
Both drugs are intended to prepare the body for the upcoming stem cell transplant.
Step 3
Haploidentical stem cell transplantation
Once the conditioning drugs have been administered, the donor’s stem cells are infused into the bloodstream. this procedure is called haploidentical stem cell transplantation and is performed in a specialized treatment area.
Step 4
Post‑transplant cyclophosphamide
After the stem cell infusion, an intravenous dose of cyclophosphamide is given at 40 mg per kilogram of body weight. this medication helps reduce the risk of the immune system attacking the new cells.
Step 5
Oral immunosuppression
To further prevent graft‑versus‑host disease, two oral medicines are started:
ciclosporin is taken by mouth at a dose of 3 mg per kilogram of body weight.
mycophenolate mofetil is taken by mouth at a dose of 3 g.
These medications are usually taken daily for several weeks or months, as directed by the treatment team.
Step 6
Follow‑up monitoring
Regular clinic visits are scheduled to check blood counts, organ function, and signs of complications such as infection or graft‑versus‑host disease.
The medical team will also assess how well the transplant is working and whether any adjustments to medication are needed.

Who can join the trial?

7 criteria

You are between 60 and 75 years old, or you are 18‑59 years old but cannot receive the usual high‑intensity chemotherapy (called a MAC regimen) because other health problems give you a score of 3 or higher on the HCT‑CI (a tool that measures other illnesses).
You have acute myeloid leukemia (AML) that meets the 2022 ELN classification and need a stem‑cell transplant from another person (allo‑HSCT). This includes being in the first complete remission (CR1) with intermediate or unfavorable risk, or being in CR1 with favorable risk only if a tiny amount of disease (minimal residual disease, MRD) is still detectable, or being in a second remission (CR2) or later, including cases where the disease returned at a molecular level after the first remission.
At the time you join the study, less than 5% of your bone‑marrow cells are leukemia cells (called blasts), meaning you are in a state called CR, CRi, CRh, or MLFS after prior treatment.
You have a plan for an allo‑HSCT using a haploidentical donor (a family member who is a half‑match).
You must be covered by a health‑care system (such as health insurance or national coverage).
You must sign an informed consent form before any study procedures begin, showing that you understand and agree to take part.
Your overall health status must be good enough, with an ECOG score of less than 2 or a Karnofsky Index of 60% or higher (both are ways doctors measure how well you can carry out daily activities).

Who cannot join the trial?

16 criteria

Heart pumping ability less than 40% (your heart’s left ventricle is not pumping enough blood).
Pregnant women, women who could become pregnant without reliable birth control, or women who are breastfeeding.
Adults who are under legal protection such as guardianship, curatorship, or court‑ordered protection.
Inability to attend required medical visits because of where you live, social circumstances, or psychological reasons.
Liver enzymes (ASAT and ALAT) more than 2.5 times the normal upper limit (indicates significant liver stress or damage).
DLCOc less than 40% (a lung test showing poor ability to transfer oxygen into the blood).
Having acute promyelocytic leukemia (a specific type of blood cancer different from AML).
Currently receiving an experimental drug in another clinical trial.
Known allergy (hypersensitivity) to the medicines treosulfan, fludarabine, cyclophosphamide, or any of their ingredients.
Being positive for HIV (human immunodeficiency virus).
Having had a previous allogeneic stem cell transplant (transplant from a donor).
Having an uncontrolled infection that spreads throughout the body (fungal, bacterial, or viral) that has not gotten better despite treatment.
Having hepatitis B (HBV) or hepatitis C (HCV) with a detectable amount of virus in the blood or being on antiviral medication.
Having any other active or uncontrolled cancer.
Kidney function (renal clearance) less than 50 mL per minute (the kidneys are not filtering blood well enough).
Any severe, uncontrolled medical condition that the doctor believes makes using treosulfan, fludarabine, or cyclophosphamide unsafe (a contraindication).

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Verified sites

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

Verified
Assistance Publique Hopitaux De Paris
Paris, France
Verified
Besancon University Hospital Center
Besançon, France
Responsive
CHRU De Nancy
Vandoeuvre Les Nancy, France

Trial locations

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Investigated drugs

Ciclosporin
is an oral medication that helps suppress the immune system. In this study it is given as part of the standard background treatment to reduce the risk that the body will reject the donor stem cells after transplantation.
Mycophenolate mofetil
is another oral drug that weakens the immune response. It is also used in the background regimen to help prevent the immune system from attacking the transplanted stem cells.
Fludarabine
is given by IV infusion. It is a chemotherapy drug that attacks rapidly dividing cells, including cancer cells. In this trial it is a key part of the test conditioning regimen, helping to prepare the patient’s bone marrow for the new stem cells.
Cyclophosphamide
is administered intravenously. It is a chemotherapy agent that also reduces immune activity. In this study it is used after the stem cell transplant (post‑transplant cyclophosphamide) to lower the chance of graft‑versus‑host disease.
Treosulfan
is provided as an IV infusion. It is a chemotherapy drug used in the test conditioning regimen to help destroy any remaining leukemia cells and make space in the bone marrow for the donor stem cells.

What is already known about the treatment

CICLOSPORIN
Taken by mouth, this tablet works by blocking a protein called calcineurin, which reduces activity of immune cells; it is an established immunosuppressant widely used to prevent organ rejection and treat some autoimmune disorders; its main use is in transplant patients; it belongs to the class of calcineurin inhibitors and is well documented in medical literature.
MYCOPHENOLATE MOFETIL
Administered orally as a capsule, it inhibits an enzyme needed for DNA building blocks, lowering the proliferation of immune cells; it is a standard drug for preventing rejection after organ transplants and for certain autoimmune diseases; it is a proven therapy with extensive research; it is classified as an antimetabolite immunosuppressant.
FLUDARABINE PHOSPHATE
Given as an intravenous solution for injection or infusion, this chemotherapy drug mimics a building block of DNA and stops cancer cells from copying their DNA; it is approved for several blood cancers, including use in conditioning regimens before stem‑cell transplants; it is a well‑studied nucleoside analog in oncology; it belongs to the class of antimetabolite chemotherapeutic agents.
CYCLOPHOSPHAMIDE
Delivered by intravenous infusion, it adds an alkyl group to DNA, causing breaks that kill rapidly dividing cells; it is a long‑standing chemotherapy used for many cancers and as part of transplant conditioning; it has a long history of clinical use and extensive literature; it is classified as an alkylating agent.
TREOSULFAN
Provided as an intravenous infusion solution, this drug also adds alkyl groups to DNA, leading to cell death, and is used primarily to prepare patients for stem‑cell transplantation; it is an approved, orphan‑designated medication for certain high‑risk blood cancers and is increasingly studied in reduced‑intensity regimens; it is a member of the alkylating agent class.

Investigated diseases

Acute Myeloid Leukemia - Acute Myeloid Leukemia (AML) is a cancer of the blood and bone marrow that originates from the rapid growth of abnormal myeloid cells. These immature cells accumulate in the marrow, crowding out normal blood‑forming cells and leading to low counts of red cells, white cells, and platelets. As the disease advances, the abnormal cells spill into the bloodstream and can spread to other organs such as the spleen, liver, and central nervous system. The uncontrolled proliferation causes the marrow to become increasingly filled with leukemic blasts, worsening blood shortages. Over time, patients may experience increasing fatigue, infections, bruising, and anemia as the disease progresses.