RecruitingRare disease

Naxitamab Added to Standard Induction Therapy for Children and Teenagers with Newly Diagnosed High-Risk Neuroblastoma

Verified siteInvestigationalNo 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 is studying neuroblastoma, including ganglioneuroblastoma, in children and teenagers who have just been diagnosed with a high-risk form of the disease. High-risk means the cancer is more likely to grow or spread again. The treatment being tested is naxitamab, given into a vein, together with GM-CSF and standard induction therapy, which is the first group of cancer treatments given soon after diagnosis. The purpose of the study is to find out whether adding naxitamab to standard treatment can help more patients have a complete response, meaning no signs of cancer can be found after the first part of treatment.

In the study, treatment is given in a planned course over several cycles. Naxitamab is added to the usual early treatment, and patients are followed through the treatment period and into the next phase of care. The study also looks at side effects, including pain during the infusion, and other safety concerns. It will compare the results with older records from similar patients who received standard treatment without naxitamab.

Neuroblastoma is a cancer that starts in nerve cells, most often in young children. The study is focused on whether this added treatment can improve the early treatment outcome for patients with newly diagnosed high-risk 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
Start of study treatment
Your study treatment begins with naxitamab, which is given as an intravenous infusion (medicine given through a vein). The dose is 150 mg each time it is given.
You also receive granulocyte-macrophage colony-stimulating factor, called GM-CSF. This medicine is used together with naxitamab and standard induction treatment.
The trial starts from your first study treatment.
Step 2
Induction therapy period
You continue with standard induction therapy together with naxitamab and GM-CSF.
The study checks how your disease responds during this induction period.
Your response is assessed using the International Neuroblastoma Response Criteria, which is a standard way to measure how well the cancer is responding to treatment.
Step 3
After cycle 2
After cycle 2, your disease response is checked.
The study looks for complete response in the bone, soft tissue, and/or bone marrow.
The study also records your overall response category, which may be complete response, partial response, minor response, stable disease, or progressive disease.
Bone marrow is the soft tissue inside bones where blood cells are made.
Step 4
After cycle 4
After cycle 4, your disease response is checked again.
The study again looks for complete response in the bone, soft tissue, and/or bone marrow.
The study also records your overall response category at this stage.
Step 5
End of induction
At the end of induction, your response to treatment is assessed again.
The study measures the rate of complete response at this point.
The study also measures complete response plus very good partial response, and overall response rate, which includes complete response, partial response, and very good partial response.
The study checks changes in the size of soft-tissue lesions (areas of abnormal tissue), primary tumor volume (the size of the main tumor), bone-marrow infiltration (cancer cells found in the bone marrow), and Curie score (a score that shows how much disease is seen on scans).
The study also checks whether disease in the bone marrow becomes mrD-negative, which means that very small amounts of disease can no longer be found by a sensitive laboratory test called RT-qPCR.
The study records any adverse events, which are unwanted medical problems during treatment, including serious adverse events.
The study records whether naxitamab causes pain during infusion, whether treatment needs to be delayed, reduced, or stopped, and whether treatment delays or prevents you from moving on to consolidation.
Step 6
End of consolidation
If you reach consolidation, the study checks whether complete response seen at the end of induction is still confirmed at the end of consolidation.
Consolidation is the treatment phase that follows induction.
Step 7
Follow-up and outcome tracking
From the start of study treatment until the end of follow-up, the study tracks event-free survival, progression-free survival, and overall survival.
Event-free survival means the time from first study treatment until relapse, progression, a second cancer, death, or the last time you are known to be in contact with the study.
Progression-free survival means the time from first study treatment until the disease gets worse or death, or until the last time you are known to be in contact with the study.
Overall survival means the time from first study treatment until death, or until the last time you are known to be in contact with the study.
The study also records the amount of naxitamab in your body, the body's handling of the medicine, and whether your body makes anti-drug antibodies, which are proteins that may react against the medicine.

Who can join the trial?

23 criteria

The patient must have neuroblastoma or ganglioneuroblastoma of the nodular or intermixed type, confirmed by a tissue exam (histology) or by finding groups of tumor cells in the bone marrow together with high levels of certain urine markers called catecholamine metabolites.
The patient must have one of the allowed disease patterns at diagnosis, including:
- Stage M disease with MYCN amplification (more than 4 times the usual number of MYCN gene signals), no matter what other tumor features are present.
- Stage M disease at age 365 to 547 days without MYCN amplification, but with at least one unfavorable feature, such as unfavorable histology (tumor appearance under the microscope), diploid tumor (DNA index = 1, meaning the tumor has the usual amount of DNA), or a segmental chromosome abnormality such as loss of 1p, 3p, 4p, or 11q, or gain of 1q, 2p, or 17q.
- Stage M disease at age older than 547 days, regardless of tumor features.
- Stage MS disease with MYCN amplification.
- Stage MS disease at age 365 to 547 days without MYCN amplification, but with an unfavorable feature such as unfavorable histology, diploid tumor, or a segmental chromosome abnormality.
- Stage L2 disease with MYCN amplification.
- Stage L2 disease at age 18 months to under 5 years without MYCN amplification, but with unfavorable histology.
- Stage L2 disease at age 5 years or older without MYCN amplification, but with undifferentiated or poorly differentiated tumor cells, meaning the cells look very abnormal and immature under the microscope.
- Stage L1 disease that was not completely removed by surgery and has MYCN amplification.
- If the patient was first diagnosed with Stage L1, L2, or MS disease and later progressed to Stage M without having chemotherapy, enrollment is allowed within 4 weeks of that progression, if the patient was older than 547 days at the original diagnosis.
- If the patient was first diagnosed with MYCN-amplified Stage L1 disease and later progressed to Stage M without receiving systemic treatment (treatment that affects the whole body), enrollment is allowed within 4 weeks of that progression, if the patient was at least 365 days old at the original diagnosis.
- The patient must be 21 years old or younger at the time of the first diagnosis.
- The patient must be older than 12 months at the time of enrollment.
- The patient must have adequate heart function, shown by either a shortening fraction of at least 27% on an echocardiogram (heart ultrasound) or an ejection fraction of at least 50% on a radionuclide test or echocardiogram. Ejection fraction means the percentage of blood the heart pumps out with each beat.
- The patient must have adequate liver function, shown by:
  - Total bilirubin no more than 1.5 times the upper normal limit for age. Bilirubin is a substance made when the body breaks down red blood cells.
  - ALT (also called SGPT, a liver enzyme) less than 5 times the upper normal limit for age.
  - The patient must have adequate kidney function, shown by one of the following:
    
    If younger than 17 years, an eGFR of at least 70 mL/min/1.73 m². eGFR means estimated glomerular filtration rate, which is a measure of how well the kidneys filter blood.
    If 17 years or older, an eGFR of at least 70 mL/min/1.73 m².
    Or a 24-hour urine creatinine clearance of at least 70 mL/min/1.73 m². Creatinine clearance is another way to measure kidney filtering.
    Female patients who can become pregnant, including those 13 years or older or those who have started menstruation, must have a negative pregnancy test in the blood.
    Male and female patients after puberty must be willing to use a highly effective birth control method from the time they agree to join the study until 6 months after treatment ends. Highly effective methods are those with a very low chance of failure when used correctly, such as certain hormonal methods, an IUD (a device placed in the uterus), an IUS (a hormone-releasing device placed in the uterus), tubal occlusion (blocking the fallopian tubes), a vasectomized partner, or sexual abstinence (not having sex).
    The patient, or the legal guardian when needed, must sign written informed consent, which means agreeing to take part after the study has been explained. When required, the patient must also give assent, meaning age-appropriate agreement to join.

Who cannot join the trial?

10 criteria

Children who are younger than 1 year old cannot take part.
Children who are 12 to 18 months old with INRGSS Stage M disease cannot take part.
Children with stage L2 disease and favorable biologic features cannot take part. This means the cancer does not have MYCN amplification (extra copies of the MYCN gene), has favorable pathology (the cancer cells look less aggressive under a microscope), and has a DNA index greater than 1 (the amount of DNA in the cancer cells is higher than normal).
Children who have already had systemic therapy cannot take part, except for localized emergency radiation to a life-threatening or function-threatening area, and/or no more than 1 cycle of chemotherapy. Systemic therapy means treatment that travels through the whole body, such as medicine given into a vein or by mouth.
Children who received immunosuppressive treatment within 4 weeks before joining the study cannot take part, except for topical treatment, inhaled treatment, and short-term emergency steroids. Immunosuppressive treatment means medicine that lowers the activity of the immune system.
Children with poor lung function cannot take part. This includes shortness of breath at rest, trouble with exercise, or a long-term need for oxygen. It also includes oxygen levels below 94% while breathing room air, or abnormal lung function tests when those tests are needed.
Pregnant or breastfeeding patients cannot take part. Breast milk cannot be saved for later use while the mother is receiving study treatment.
Children who are receiving another investigational drug at the same time cannot take part. An investigational drug is a medicine still being studied.
Children with any other medical problem that the study doctor thinks could affect the study results or make it hard to sign consent, or make it hard for the child or guardian to follow the study rules, cannot take part. Examples include malabsorption syndromes (problems absorbing nutrients from food), mental illness, or substance abuse.
Children with a significant ongoing illness that is not related to the cancer or its treatment cannot take part if it is expected to interfere with the study medicine or make treatment side effects more severe.

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Hospital Sant Joan De Deu Barcelona
Esplugues De Llobregat, Spain

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Investigated drugs

Naxitamab
is a targeted antibody treatment given through a vein. It is designed to find and attach to GD2, a marker found on neuroblastoma cells, so the immune system can help attack the cancer. In this trial, it is added to standard induction treatment to see if it can improve the chance of a complete response in children with newly diagnosed high-risk neuroblastoma.
Granulocyte-Macrophage Colony-Stimulating Factor (GM-CSF)
is a medicine that helps the body make and activate certain white blood cells. In this study, it is used together with naxitamab to help strengthen the immune response against the cancer.
Standard induction therapy
is the usual first set of cancer treatments given after diagnosis. It is used to shrink the tumor and control the disease before later treatment steps. In this trial, it serves as the base treatment that is combined with naxitamab and GM-CSF.

Naxitamab

What is already known about the treatment

Naxitamab - Naxitamab is given as a solution for infusion through a vein, so it is administered by intravenous use in a hospital or clinic setting. It is an approved and well-studied medicine in the medical literature for high-risk neuroblastoma, and it has orphan drug status because it is used for a rare disease. It is a humanized monoclonal antibody, which means it is a lab-made protein designed to find and attach to GD2, a marker found on neuroblastoma cells; once attached, it helps the immune system destroy those cancer cells. Its main use is in treating neuroblastoma and related nerve tissue tumors, especially when combined with other cancer treatments such as GM-CSF and standard induction therapy.

Investigated diseases

Neuroblastoma
A cancer that starts in nerve tissue, most often in children. It usually begins in the adrenal glands, neck, chest, abdomen, or spine area. The disease can grow quickly and may spread to nearby lymph nodes, bone, bone marrow, liver, or skin. Its course can vary, with some tumors growing rapidly and others changing more slowly over time.
Ganglioneuroblastoma, nodular
A rare nerve tissue tumor that contains both more mature and less mature cancer cells. It usually develops in children and can arise in the adrenal glands or along the nerves in the chest, abdomen, or spine. The nodular form tends to have areas that behave more aggressively alongside more mature tissue. It may enlarge locally and can spread to other parts of the body as it progresses.