Ongoing Clinical Trials for Recurrent Neuroblastoma
Currently, there is 1 ongoing clinical trial for recurrent neuroblastoma, a type of cancer that primarily affects children and develops from immature nerve cells. This trial is testing a combination treatment approach for patients whose disease has not responded to standard therapy, and is being conducted across multiple European countries including Spain, Italy, France, Germany, and Denmark.
Clinical trial locations
- Denmark
- France
- Germany
- Italy
- Spain
Study of Naxitamab and Sargramostim for High-Risk Neuroblastoma in Patients with Refractory Bone or Bone Marrow Disease
This trial is specifically designed for children with high-risk neuroblastoma whose disease has either not responded to initial treatment or has not fully responded to additional treatments. The study focuses on patients whose cancer is present in the bone or bone marrow, making it particularly relevant for those with difficult-to-treat disease.
Main inclusion criteria:
- Confirmed diagnosis of neuroblastoma through tumor biopsy, bone marrow test, or a special scan called MIBG
- High-risk disease that is refractory or has not fully responded to treatment, with detectable disease in bone or bone marrow
- At least 12 months of age
- Life expectancy of at least 6 months
- Adequate blood cell counts, including hemoglobin of at least 8 g/dL, white blood cell count of at least 1000 per microliter, and platelet count of at least 25,000 per microliter
- Acceptable liver function, with liver enzyme levels not exceeding 5 times the normal limit
- Acceptable kidney function with an estimated filtration rate above 60 mL/min/1.73 m²
- Written consent from parents or guardians, and where required, agreement from the child
Main exclusion criteria:
- Presence of a different type of cancer
- Incomplete previous cancer treatments
- Severe or uncontrolled medical conditions that could interfere with the study
- Pregnancy or breastfeeding
- Another cancer diagnosis within the past 5 years, except for certain skin cancers
- Inability to follow study procedures or attend required visits
- Allergies to the study medications or similar drugs
- Current participation in another clinical trial
Trial focus and goals:
The main goal of this study is to evaluate how well the combination of naxitamab and sargramostim works in treating high-risk neuroblastoma. Researchers will closely monitor participants’ responses to the treatment by measuring the objective response rate, which indicates how well the cancer responds. The trial will also assess the safety of this treatment combination by tracking any side effects that occur during the study period.
The treatment is organized into cycles, with naxitamab given as an intravenous infusion directly into a vein, while sargramostim is administered as an injection under the skin. Regular monitoring throughout the trial allows doctors to evaluate both effectiveness and safety. The study is expected to continue until April 2028, providing valuable long-term information about this treatment approach.
Investigational drugs:
The trial uses two main medications working together. Naxitamab is a monoclonal antibody that targets and attaches to specific proteins found on neuroblastoma cancer cells. By binding to these cells, it helps the immune system recognize and destroy them more effectively. Sargramostim, also known as GM-CSF (Granulocyte-Macrophage Colony Stimulating Factor), is a protein that stimulates the growth of white blood cells. These cells are crucial for fighting infections and may help the body respond better to cancer treatment. The combination of these two medications aims to enhance the overall treatment effectiveness against high-risk neuroblastoma.
Summary
Currently, patients with recurrent neuroblastoma have access to one clinical trial exploring a combination therapy approach. This trial is notable for its wide availability across five European countries, making it accessible to patients in Spain, Italy, France, Germany, and Denmark. The study specifically targets children whose disease has proven resistant to standard treatments, addressing an important unmet medical need in pediatric oncology.
The trial focuses on combining naxitamab, a targeted antibody therapy, with sargramostim, an immune-boosting medication. This combination approach represents an effort to enhance the body’s natural ability to fight cancer while directly targeting cancer cells. The study is particularly relevant for patients with disease in the bone or bone marrow, which can be especially challenging to treat.
While only one trial is currently available for recurrent neuroblastoma, its multi-country design and focus on refractory disease make it an important option for families seeking treatment alternatives for children who have not responded adequately to standard therapies. The trial’s expected completion in 2028 will provide valuable long-term data on this treatment approach.