Not yet recruitingRare disease

Safety and feasibility study of CARCIK‑CD123/33 cell therapy in adult and pediatric patients with relapsed or refractory AML and BPDCN

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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 two rare blood cancers: Acute Myeloid Leukemia and Blastic Plasmacytoid Dendritic Cell Neoplasm. These conditions can return after treatment or may not respond to standard therapies. The investigational treatment is an experimental cell therapy called CARCIK-CD123/33. This therapy uses immune cells that are modified in the laboratory to recognize and attack cancer cells, and it is given to patients through an intravenous infusion, which means the medicine is delivered directly into a vein.

The purpose of the study is to determine whether the infusion of this engineered cell therapy can be administered safely and is feasible for further testing.

Participants who meet the study requirements will first undergo a screening process to confirm eligibility. Those who proceed will receive a single infusion of the cell therapy, after which they will be monitored closely for at least four weeks for any side effects and for early signs that the cancer may be responding. Follow‑up visits continue for several months, during which blood tests and other routine examinations are performed to track safety and any changes in the disease.

The research process

The trial runs in 7 steps – from screening to follow-up. Each step says what happens and what the team monitors.

Step 1
Screening and baseline assessments
After joining the study you will undergo screening procedures to confirm eligibility. this includes a review of your medical history, physical examination, and laboratory tests such as blood work and bone marrow sampling.
The results of these tests are used to establish baseline measurements before any study treatment is given.
Step 2
Pre‑infusion preparation
Once eligibility is confirmed, the study team will prepare the experimental cell product. the carcik‑cd123/33 cells are manufactured specifically for you based on the protocol.
Step 3
Administration of the investigational cell therapy
On the scheduled infusion day (day 0) you will receive the carcik‑cd123/33 product by intravenous infusion. the exact dose is defined by the study protocol and is given as a single infusion.
The infusion is performed in a clinical setting where vital signs are continuously monitored.
Step 4
Immediate post‑infusion observation
After the infusion you will remain in the clinic for several hours. staff will watch for any immediate reactions, check your blood pressure, heart rate, and temperature, and address any discomfort.
Step 5
Safety monitoring during the first 28 days
For the next 28 days you will attend regular clinic visits. at each visit blood samples are taken to look for dose limiting toxicity (serious side effects that may limit further dosing) and other adverse events.
The study team will also perform physical examinations and may repeat laboratory tests to track any changes.
Step 6
Response evaluation at day 28
On day 28 a formal assessment of your disease response is performed. doctors compare the new test results with the baseline to determine whether the leukemia has responded to the therapy.
This evaluation includes blood work, bone marrow analysis, and any imaging that may be required.
Step 7
Long‑term follow‑up
After the 28‑day assessment you will continue to have scheduled follow‑up visits. these visits may occur every few weeks to months, depending on the protocol.
During follow‑up the team will monitor the persistence of the infused cells in your blood, check for disease progression, and record overall survival information.

Who can join the trial?

12 criteria

Must be able and willing to give written informed consent (or have a parent or legal guardian do so) and follow the study rules.
The doctor must think you will live at least 12 weeks more.
Women who could become pregnant must have a negative pregnancy test, agree to avoid pregnancy by either staying abstinent or using reliable effective contraception (such as a hormonal pill, injection, implant, intrauterine device, or surgical sterilization) without interruption from screening until at least 12 months after the cell infusion, and must not breast‑feed during the study and for at least 12 months after the infusion.
Men must either stay abstinent or use a condom when having sexual contact with a pregnant woman or a woman who could become pregnant for 12 months after the cell infusion.
Age must be between 6 months and 17 years for children, or 18 to 75 years for adults.
You must have active acute myeloid leukemia or blastic plasmacytoid dendritic cell neoplasm that is present in the bone marrow or blood, and no cure is available with current standard treatments.
You need a family member or unrelated donor who matches at least half of the HLA genes (called haploidentical, meaning 4 out of 8 matches) and is willing to donate blood or cells for making the CARCIK‑CD123/33 therapy.
You must have a donor for a future allogeneic stem cell transplant (including cord blood) and be eligible to receive that transplant.
Your leukemia cells must show the proteins CD123 and CD33 on their surface, proven by a test called flow cytometry.
If you have previously received drugs that target CD123 or CD33, at least a 2‑week washout period (time without the drug) must have passed before you can join the study.
You must have an ECOG performance status of 2 or less (for people 16 years or older) or a Lansky score greater than 50 (for children under 16). ECOG measures how well you can carry out daily activities (lower numbers are better), and Lansky is a similar score used for children.
Your major organs (heart, liver, kidneys, etc.) must be working well enough, as judged by the study doctors (satisfactory organ function).

Who cannot join the trial?

19 criteria

Having a current serious infection caused by viruses, bacteria, or fungi (for example, flu‑like illness, pneumonia, or a severe fungal infection).
Having other important diseases that are not under control and could interfere with the study, such as diabetes mellitus (high blood sugar), lung problems like asthma or chronic obstructive lung disease, or autoimmune diseases where the immune system attacks the body.
Receiving any standard cancer treatment with radiation or chemotherapy (except the drug hydroxyurea) within 2 weeks before the first cell infusion.
Having taken any experimental (not yet approved) medication within 14 days before the preparatory treatment, or for a time equal to five half‑lives of that drug (the period it takes for the drug level to drop by half), whichever is shorter.
Having side effects from previous cancer treatments that have not improved to mild (≤ Grade 1) levels, except for hair loss, hormone problems that are already being treated, or stable skin color changes (vitiligo).
Having received a CAR‑T cell therapy (a type of personalized immune treatment) within 30 days before the first study infusion.
Having previously experienced a severe reaction called Grade 4 cytokine release syndrome (CRS) or immune effector cell‑associated neurotoxicity syndrome (ICANS) after CAR‑T cell therapy.
Having previously had a very severe (≥ Grade 4) side effect from treatments that target CD33 or CD123 proteins.
Having had a bone‑marrow or stem‑cell transplant (allogeneic from a donor or autologous from yourself) within 3 months, or donor lymphocyte infusions (DLI) within 1 month before the study infusion.
Having active graft‑versus‑host disease (GVHD) of moderate to severe grade (II‑IV) or chronic GVHD that needs strong immune‑suppressing medicines (for those who had a transplant).
Receiving a live, weakened (attenuated) vaccine within 4 weeks before the study infusion.
Having an active infection with Hepatitis B virus.
Having an active infection with Hepatitis C virus, unless it has been successfully treated and you have a sustained viral response at 12 weeks (SVR12) or 24 weeks (SVR24) with at least a 4‑week gap before giving consent.
Testing positive for HIV (human immunodeficiency virus) by blood test.
Having a rapidly worsening disease that doctors think could make it hard to follow the study plan or affect the results.
Having uncontrolled leukemia in the brain or nervous‑system symptoms that are severe (CNS grade 3), which requires a special brain evaluation.
Having significant donor‑specific antibodies against the donor’s HLA (human leukocyte antigen) type, especially if the donor’s HLA does not match yours.
Having another active invasive cancer. Only a completely removed very early cancer (carcinoma in situ) that does not need any treatment beyond surgery may be allowed.
Having a serious heart or circulation problem classified as Class III/IV on the New York Heart Association (NYHA) scale, a heart attack (myocardial infarction) or stroke (cerebrovascular accident) within 30 days, or having unstable heart rhythm problems (arrhythmias) or unstable chest pain (angina).

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Verified sites

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

Verified
Azienda Ospedaliera Papa Giovanni XXIII
Bergamo, Italy
Verified
Fondazione IRCCS San Gerardo Dei Tintori
Monza, Italy

Trial locations

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Investigated drugs

CARCIK CD123/33 is a specially prepared cell therapy made from donor immune cells that have been engineered to recognize and attack cancer cells that have the CD123 and CD33 markers. In this trial, patients with relapsed or refractory acute myeloid leukemia (AML) or blastic plasmacytoid dendritic cell neoplasm (BPDCN) receive an infusion of these modified cells through a vein. The main purpose is to see if giving these cells is safe and can be done feasibly, and to observe any early signs that they might help control the cancer.

What is already known about the treatment

CARCIK CD123/33 - This medication is given as a sterile solution that is infused into a vein (intravenous infusion). It is an experimental cell‑based therapy currently being studied in a Phase I/II clinical trial and is not yet approved for general medical use. The therapy is designed for adults and children whose acute myeloid leukemia or blastic plasmacytoid dendritic cell neoplasm has returned or does not respond to standard treatments. It works by using lab‑grown immune cells that have been engineered to recognize the CD123 and CD33 proteins on cancer cells, allowing the cells to attach to and destroy the malignant cells; it is classified as a chimeric antigen receptor (CAR) cytokine‑induced killer (CIK) cell immunotherapy.

Investigated diseases

Acute myeloid leukemia
a fast‑growing cancer of the blood and bone marrow where abnormal white blood cells build up and crowd out normal cells. It begins in the marrow and can spread to the bloodstream, causing fatigue, bruising, and infections as the disease advances. Over time the number of immature cells increases, reducing the production of healthy red cells and platelets. The condition can progress quickly if the abnormal cells keep multiplying.
Blastic plasmacytoid dendritic cell neoplasm
a rare disorder that starts in immune‑system cells and forms tumors in the skin, bone marrow, or lymph nodes. The disease often appears as skin lesions or lumps and may spread to the blood and other organs. As it advances, more abnormal cells appear, leading to larger skin patches and involvement of internal organs. The tumor cells can increase in number, causing the disease to expand.