Chemo-immunotherapy for children with bone and soft tissue sarcomas

A Randomized, Multicenter, Open-label Phase III Study of Dinutuximab Beta With Investigator's Choice of Chemotherapy in Patients Under 18 Years of Age With Bone and Soft Tissue Sarcomas With High Levels of GD2 Expression and Disease Progression During 1st Line Chemotherapy

This study is testing a new combination of a drug and chemotherapy to see if it helps children with certain types of advanced bone and soft tissue cancers after their first treatment didn't work.

Quick facts

• Phase: Phase 3
• Study type: Interventional
• Enrollment: 40 (estimated)
• Ages: N/A to 18 Years
• Sex: All
• Sponsor: N.N. Petrov National Medical Research Center of Oncology (Academic / other)
• Drugs / interventions: chemotherapy, dinutuximab, immunotherapy
• Locations: 1 site (Saint Petersburg)
• Trial ID: NCT06669013 on ClinicalTrials.gov

What this trial studies

This clinical trial evaluates the efficacy and safety of dinutuximab beta combined with chemotherapy in children under 18 years of age who have advanced GD2-positive rhabdomyosarcoma, Ewing sarcoma, or osteosarcoma after failing first-line chemotherapy. The study plans to enroll 40 patients, with approximately 10 being randomized to receive the treatment. Participants will undergo 6 cycles of dinutuximab beta therapy alongside 6-8 cycles of chemotherapy, with treatment response assessed at various intervals. Follow-up will continue for 6 months post-treatment to monitor survival outcomes.

Who should consider this trial

Good fit: Ideal candidates are children under 18 years with GD2-positive osteogenic sarcoma, Ewing's sarcoma, or soft tissue sarcomas who have relapsed after first-line chemotherapy.

Not a fit: Patients who have not been previously treated with chemotherapy or those with non-GD2-positive tumors may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could provide a new effective option for children with advanced sarcomas who have not responded to previous therapies.

How similar studies have performed: Other studies have shown promising results with similar immunotherapy approaches in treating sarcomas, indicating potential for success in this trial.

Eligibility criteria

Inclusion Criteria:

1. Signed written informed consent form;
2. Age under 18 years of age;
3. Histologically confirmed GD2-positive osteogenic sarcoma, Ewing's sarcoma, soft tissue and undifferentiated sarcomas;
4. Prior to study enrollment, patients must have been treated with one or more lines of adequate chemotherapy and must have relapsed on this therapy or have refractoriness to it;
5. General satisfactory condition of the patient (Lansky scale activity 80-100% (children under 16 years of age), Karnofsky scale 80-100% (children over 16 years of age); ECOG - 0-1);
6. Sufficient cardiopulmonary reserves of the patient's organism (ECG (ELECTROCARDIOGRAPHY) data within normal limits, ventricular ejection fraction \> 75% of the upper limit of normal;
7. Adequate liver function (ALT (ALANINE AMINOTRANSFERASE) ≤ 2.5 \* VGN, AST (ASPARTATE AMINOTRANSFERASE) ≤ 2.5 \* VGN), kidney (creatinine \<1.5 \* VGN), red bone marrow (granulocytes\> 2.0 \* 109/L, platelets\> 150 \* 109/L).

   Six months later, two additional criteria were formulated:
8. Life expectancy at the time of initiation of therapy within the framework of the study is not less than 12 months
9. Oligometastasis disease (presence of 1 to 5 distant metastatic foci) at restaging at the time of the decision to include the patient in the study;

Exclusion Criteria:

1. Withdrawal of consent by the patient or his/her parent/guardian.
2. Exclusion of the patient by the investigator for safety or ethical reasons.

Where this trial is running

Saint Petersburg

• Kulyova Svetlana — Saint Petersburg, Russia (Recruiting)

Study contacts

• Principal investigator: Svetlana Kulyova, MD, Phd — Head of Children's Oncology Department
• Study coordinator: Svetlana Kulyova, MD, Phd

Conditions Bone Sarcoma; Embryonal Rhabdomyosarcoma; Alveolar Rhabdomyosarcoma