Evaluating Naxitamab for Refractory Ewing's Sarcoma

To Evaluate the Efficacy and Safety of Naxitamab in Patients With Refractory Ewing's Sarcoma

This study is testing if adding Naxitamab to chemotherapy can help people with hard-to-treat Ewing's Sarcoma feel better compared to standard treatment alone.

Quick facts

• Phase: Phase 2
• Study type: Interventional
• Enrollment: 24 (estimated)
• Ages: 2 Years to 21 Years
• Sex: All
• Sponsor: Institute of Mother and Child, Warsaw, Poland (Academic / other)
• Drugs / interventions: naxitamab, radiation
• Locations: 2 sites (Warsaw, Mazowian and 1 other locations)
• Trial ID: NCT05968768 on ClinicalTrials.gov

What this trial studies

This clinical trial aims to evaluate the efficacy and safety of Naxitamab in patients with refractory Ewing's Sarcoma. It involves a prospective, interventional, open, randomized design across multiple centers in Poland. Patients will undergo biology screening to assess GD2 expression in their tumor samples, followed by randomization into experimental and control groups based on their GD2 status. The experimental group will receive a specific regimen of chemotherapy combined with Naxitamab, while the control group will receive standard treatment.

Who should consider this trial

Good fit: Ideal candidates are children and young adults aged 2 to 21 years with histologically confirmed Ewing Sarcoma and GD2 positive tumors.

Not a fit: Patients without GD2 expression in their tumors or those who have not completed prior treatments may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could provide a new therapeutic option for patients with refractory Ewing's Sarcoma.

How similar studies have performed: Other studies have shown promise with GD2-targeted therapies in similar contexts, suggesting potential for success in this approach.

Eligibility criteria

Inclusion Criteria:

1. Histologically proven Ewing sarcoma of the bone or soft tissues.
2. Subject's archival tumour sample (formalin-fixed, paraffin-embedded; FFPE) available for evaluation of GD2 expression.
3. Documented disease progression (during or after completion of at least one line treatment) or any subsequent recurrence.
4. GD2 positive tumor assessed by IHC.
5. Age ≥ 2 years and ≤ 21 years.
6. Life expectancy of at least 12 weeks from the time informed consent was signed.
7. Previous systemic anticancer treatment completed ≥ 3 weeks, major surgery ≥ 2 weeks, and radiation therapy ≥ 4 weeks prior to study enrollment.
8. Recovered from adverse effects of prior surgery, radiotherapy, or Clinical trial protocol BUTTERFLY version 1.0 of 30.09.2022 r.anti-neoplastic therapy at the discretion of the investigator.
9. Signing of informed consent for trial participation (including for naxitamab treatment) according with current legal regulations.
10. Consent to the use of effective contraception throughout the period of the study and a minimum of 1 year after discontinuation of study treatment in patients at puberty and sexual maturity

Exclusion Criteria:

1. Failure to meet any of the inclusion criteria.
2. Not eligible to IT.
3. Previous treatment with an anti-GD2 antibody.
4. Hypersensitivity to the study drugs or any of their ingredients (covers IT and naxitamab).
5. Simultaneous treatment with other drugs which might interact with naxitamab or IT regimen.
6. Persistent toxicity related to prior therapy, making it impossible to treat with naxitamab.
7. Significant cardiac conduction abnormalities, including known familial prolonged QT syndrome, or screening corrected QT interval (QTc) \>480 msec.
8. Symptoms of congestive heart failure or left ventricular ejection fraction \<50%.
9. Inadequate pulmonary function defined as evidence of dyspnea at rest, exercise intolerance, and/or chronic oxygen requirement. In addition, room air pulse oximetry \< 94% and/or abnormal pulmonary function tests if these assessments are clinically indicated.
10. Requirement, or likely requirement, for corticosteroids at doses \>10 mg prednisolone (or equivalent) per day or other immunosuppressive agents.
11. Diagnosis of other malignancies before study inclusion.
12. Planning to become pregnant (while being treated with IT or naxitamab), pregnancy or breastfeeding.
13. Other acute or persistent disorders, behaviors or abnormal laboratory test results, which might increase the risk related to the participation in this clinical trial or to taking the study drug, or which might influence the interpretation of the study results, or which, in the investigator's opinion, disqualify a patient from participating in the tri

Where this trial is running

Warsaw, Mazowian and 1 other locations

• Mother and Child Institute — Warsaw, Mazowian, Poland (Recruiting)
• Wroclaw Medical University — Wroclaw, Poland (Not_yet_recruiting)

Study contacts

• Principal investigator: Anna Raciborska, Prof. — the Institue of Mother and Child
• Study coordinator: Anna Raciborska, Prof.

Conditions Ewing Sarcoma