This clinical trial focuses on studying treatments for high-risk neuroblastoma, a type of cancer that most commonly affects children. The study aims to evaluate and compare different treatment strategies to improve outcomes for patients with this condition. The treatments being tested include various chemotherapy drugs and a combination of chemotherapy and immunotherapy. Some of the medications involved in the study are vincristine sulfate, ifosfamide, temozolomide, cisplatin, melphalan, irinotecan hydrochloride trihydrate, thiotepa, cyclophosphamide, etoposide, dinutuximab beta, dacarbazine, doxorubicin hydrochloride, busulfan, and carboplatin. The study also includes a placebo group for comparison.
The purpose of the study is to compare the effectiveness of different treatment regimens over a three-year period. The study is divided into three phases: induction, high-dose chemotherapy, and radiotherapy. In the induction phase, two different chemotherapy regimens are compared. In the high-dose chemotherapy phase, a single high-dose treatment with busulfan and melphalan is compared to a tandem high-dose treatment with thiotepa followed by busulfan and melphalan. The radiotherapy phase compares two different radiation doses to the tumor area. Additionally, for patients who do not respond well to initial chemotherapy, a combination of chemotherapy and immunotherapy is introduced.
Participants in the study will receive one of the treatment strategies and will be monitored over time to assess the effectiveness of the treatment. The study will help determine which treatment strategy offers the best chance of improving survival and reducing the risk of cancer returning. The trial is expected to continue until 2031, with ongoing assessments of patient outcomes and responses to the treatments being tested.
1induction phase
The induction phase involves the administration of chemotherapy to reduce the size of the tumor. Two different regimens may be used: GPOH or RAPID COJEC. The choice of regimen is determined by randomization.
Medications used in this phase include vincristine sulfate, ifosfamide, temozolomide, cisplatin, melphalan, irinotecan hydrochloride trihydrate, thiotepa, cyclophosphamide, etoposide, dinutuximab beta, dacarbazine, doxorubicin hydrochloride, busulfan, and carboplatin. These are administered intravenously.
The goal is to achieve a sufficient response to proceed to the next phase.
2high-dose chemotherapy (HDC) phase
Patients with a sufficient response to induction chemotherapy enter the high-dose chemotherapy phase. This phase involves either a single high-dose chemotherapy with busulfan and melphalan (Bu-Mel) or a tandem high-dose chemotherapy with thiotepa followed by Bu-Mel.
The choice between these two strategies is determined by randomization.
The purpose of this phase is to further reduce the tumor burden and prepare for potential surgery.
3radiotherapy phase
Following high-dose chemotherapy and surgery, radiotherapy is administered to the pre-operative tumor bed.
In cases where there is no macroscopic residual disease, a dose of 21.6 Gy is given. If there is residual disease, a sequential boost up to 36 Gy may be applied.
The aim is to eliminate any remaining cancer cells.
4chemoimmunotherapy phase
Patients with insufficient metastatic response after induction chemotherapy may receive chemoimmunotherapy.
This involves four courses of a combination of irinotecan and temozolomide (TEMIRI) with dinutuximab beta (DB).
The objective is to improve the response to treatment and target any remaining cancer cells.
Who Can Join the Study?
Patients must have a confirmed diagnosis of high-risk neuroblastoma. Neuroblastoma is a type of cancer that usually affects young children.
Patients can be of any age, but specific conditions apply based on age and certain genetic factors.
Patients should not have received previous chemotherapy, except in specific cases where limited treatment was given.
Females who can have children must have a negative pregnancy test before starting the study and agree to use effective birth control during the study and for one year after.
Patients or their guardians must provide written consent to participate in the study.
Patients must be part of a social security system or have similar coverage as required locally.
Patients should be willing and able to attend study visits and follow the study procedures.
Who Cannot Join the Study?
Patients who do not have high-risk neuroblastoma cannot participate. Neuroblastoma is a type of cancer that usually affects young children.
Patients who are not within the specified age range for the study cannot participate. The study is designed for certain age groups.
Patients who are not able to undergo the specific treatments or procedures required by the study cannot participate. This includes certain types of chemotherapy and radiotherapy.
Patients who have other medical conditions that might interfere with the study treatments cannot participate. This is to ensure the safety and effectiveness of the study.
Patients who are unable to follow the study procedures or attend required visits cannot participate. This is important for accurate study results.
Patients who are pregnant or breastfeeding cannot participate. This is to protect the health of the mother and child.
Patients who have participated in another clinical trial recently may not be eligible. This is to avoid interference with study results.
Patients who have allergies or reactions to the study medications cannot participate. This is to prevent any harmful effects.
GPOH is one of the induction regimens used in the study for patients with high-risk neuroblastoma. It is designed to help reduce the size of the tumor and control the disease before moving on to more intensive treatments.
RAPID COJEC is another induction regimen being compared in the study. Like GPOH, it aims to shrink the tumor and manage the disease in patients with high-risk neuroblastoma, but it uses a different combination of chemotherapy drugs.
Busulfan and Melphalan (Bu-Mel) are used in high-dose chemotherapy (HDC) as a single treatment option. This combination is intended to destroy cancer cells more effectively after the initial induction phase.
Thiotepa followed by Busulfan and Melphalan (Bu-Mel) is a tandem high-dose chemotherapy approach. This strategy involves using Thiotepa first, followed by Bu-Mel, to enhance the treatment’s effectiveness in patients who have responded well to induction chemotherapy.
Radiotherapy is used to target the preoperative tumor bed with a specific dose of radiation (21.6 Gy). This treatment aims to eliminate any remaining cancer cells after high-dose chemotherapy and surgery.
Radiotherapy with a Sequential Boost involves an initial dose of 21.6 Gy to the tumor bed, followed by an additional boost up to 36 Gy to any residual tumor. This approach is used for patients with visible disease remaining after high-dose chemotherapy and surgery.
Irinotecan-Temozolomide (TEMIRI) combined with Dinutuximab Beta (DB) is a chemoimmunotherapy regimen. It is used for patients who have not responded sufficiently to induction chemotherapy, aiming to improve the metastatic response rate by combining chemotherapy with an immunotherapy agent.
Neuroblastoma – Neuroblastoma is a type of cancer that most commonly affects children and develops from immature nerve cells found in several areas of the body. It typically begins in the adrenal glands, but it can also develop in the neck, chest, abdomen, or spine. The disease is characterized by the formation of a solid tumor, which can spread to other parts of the body, including the bones, liver, and skin. High-risk neuroblastoma is a more aggressive form of the disease, often requiring intensive treatment. The progression of neuroblastoma can vary, with some tumors growing slowly and others rapidly. Symptoms may include a lump in the abdomen, pain, and changes in bowel habits, depending on the tumor’s location.
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