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

NCT05968768 | Recruiting Phase 2

• 1 other identifier: Butterfly
• Study Type: interventional
• Target: 24
• Locations: 1 country
• Sites: 2

Brief Summary

Prospective, interventional, open, randomized, national, multicenter, non-commercial trial

Conditions

Ewing Sarcoma

Keywords

Ewing Sarcoma

Outcome Measures

Primary Outcomes (1)

• Safety assessment of the addition of naxitamab to standard 3-week chemotherapy (CHT) in patients with refractory Ewing's sarcoma (ES)

  Safety will be assessed by number of serious adverse events (SAE), by the number of adverse events (AE), by medical examination with the analysis of recorded vital signs, laboratory abnormalities according to NCI CTCAE v5.0

  up to 240 days

Secondary Outcomes (4)

• Event-Free Survival (EFS )

  3 years

• Progression-Free Survival (PFS)

  1 year

• Overall Response Rate (ORR)

  126 days

• Overall Survival (OS)

  3 years

Study Arms (2)

Experimental - Naxitamab Arm

EXPERIMENTAL

Treatment with naxitamab will be continued no longer than 6 cycles a year or until disease progression, patient consent, unacceptable toxicities, or study closure

Drug: Naxitamab

Control Group - standard treatment

NO INTERVENTION

The control group - will receive only standard treatment.

Interventions

Naxitamab DRUG

Naxitamab will be used only in a hospital setting and must be administered under the supervision of a doctor with experience in the use of oncological therapies. The medicinal product must be administered by a healthcare professional prepared to deal appropriately with severe allergic reactions, including anaphylaxis, in an environment that provides immediate, full access to resuscitation. The patient should have 2 well-functioning IV accesses before any naxitamab treatment is initiated. The solution should be administered through a peripheral or central intravenous catheter. Other concomitant intravenous medicinal products should be administered through separate intravenous access. Before the start of each infusion, premedication will be carried out.

Also known as: danyelza

Experimental - Naxitamab Arm

Eligibility Criteria

• Age: 2 Years - 21 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Histologically proven Ewing sarcoma of the bone or soft tissues.
• Subject's archival tumour sample (formalin-fixed, paraffin-embedded; FFPE) available for evaluation of GD2 expression.
• Documented disease progression (during or after completion of at least one line treatment) or any subsequent recurrence.
• GD2 positive tumor assessed by IHC.
• Age ≥ 2 years and ≤ 21 years.
• Life expectancy of at least 12 weeks from the time informed consent was signed.
• Previous systemic anticancer treatment completed ≥ 3 weeks, major surgery ≥ 2 weeks, and radiation therapy ≥ 4 weeks prior to study enrollment.
• 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.
• Signing of informed consent for trial participation (including for naxitamab treatment) according with current legal regulations.
• 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

You may not qualify if:

• Not eligible to IT.
• Previous treatment with an anti-GD2 antibody.
• Hypersensitivity to the study drugs or any of their ingredients (covers IT and naxitamab).
• Simultaneous treatment with other drugs which might interact with naxitamab or IT regimen.
• Persistent toxicity related to prior therapy, making it impossible to treat with naxitamab.
• Significant cardiac conduction abnormalities, including known familial prolonged QT syndrome, or screening corrected QT interval (QTc) \>480 msec.
• Symptoms of congestive heart failure or left ventricular ejection fraction \<50%.
• 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.
• Requirement, or likely requirement, for corticosteroids at doses \>10 mg prednisolone (or equivalent) per day or other immunosuppressive agents.
• Planning to become pregnant (while being treated with IT or naxitamab), pregnancy or breastfeeding.
• 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

Sponsors & Collaborators

• Anna Raciborska: lead
• Wroclaw Medical University: collaborator

Study Sites (2)

Mother and Child Institute

Warsaw, Mazowian, 01-211, Poland

RECRUITING

Wroclaw Medical University

Wroclaw, 50-556, Poland

NOT YET RECRUITING

Related Publications (7)

• Nakajima M, Guo HF, Hoseini SS, Suzuki M, Xu H, Cheung NV. Potent antitumor effect of T cells armed with anti-GD2 bispecific antibody. Pediatr Blood Cancer. 2021 Jul;68(7):e28971. doi: 10.1002/pbc.28971. Epub 2021 Apr 12.

  PMID: 33844437 RESULT

• Chan GC, Chan CM. Anti-GD2 Directed Immunotherapy for High-Risk and Metastatic Neuroblastoma. Biomolecules. 2022 Feb 24;12(3):358. doi: 10.3390/biom12030358.

  PMID: 35327550 RESULT

• Musumeci F, Cianciusi A, D'Agostino I, Grossi G, Carbone A, Schenone S. Synthetic Heterocyclic Derivatives as Kinase Inhibitors Tested for the Treatment of Neuroblastoma. Molecules. 2021 Nov 23;26(23):7069. doi: 10.3390/molecules26237069.

  PMID: 34885651 RESULT

• Wingerter A, El Malki K, Sandhoff R, Seidmann L, Wagner DC, Lehmann N, Vewinger N, Frauenknecht KBM, Sommer CJ, Traub F, Kindler T, Russo A, Otto H, Lollert A, Staatz G, Roth L, Paret C, Faber J. Exploiting Gangliosides for the Therapy of Ewing's Sarcoma and H3K27M-Mutant Diffuse Midline Glioma. Cancers (Basel). 2021 Jan 29;13(3):520. doi: 10.3390/cancers13030520.

  PMID: 33572900 RESULT

• Nazha B, Inal C, Owonikoko TK. Disialoganglioside GD2 Expression in Solid Tumors and Role as a Target for Cancer Therapy. Front Oncol. 2020 Jul 7;10:1000. doi: 10.3389/fonc.2020.01000. eCollection 2020.

  PMID: 32733795 RESULT

• Hensel J, Metts J, Gupta A, Ladle BH, Pilon-Thomas S, Mullinax J. Adoptive Cellular Therapy for Pediatric Solid Tumors: Beyond Chimeric Antigen Receptor-T Cell Therapy. Cancer J. 2022 Jul-Aug 01;28(4):322-327. doi: 10.1097/PPO.0000000000000603.

  PMID: 35880942 RESULT

• Mora J. Autologous Stem-Cell Transplantation for High-Risk Neuroblastoma: Historical and Critical Review. Cancers (Basel). 2022 May 24;14(11):2572. doi: 10.3390/cancers14112572.

  PMID: 35681553 RESULT

MeSH Terms

Conditions

Sarcoma, Ewing

Interventions

naxitamab

Condition Hierarchy (Ancestors)

Osteosarcoma; Neoplasms, Bone Tissue; Neoplasms, Connective Tissue; Neoplasms, Connective and Soft Tissue; Neoplasms by Histologic Type; Neoplasms; Sarcoma

Study Officials

• Anna Raciborska, Prof.

  the Institue of Mother and Child

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Anna Raciborska, Prof.

CONTACT

+48 22 32 77 205; klinika.onkologii@imid.med.pl

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Prof Ass

Study Record Dates

• First Submitted: June 13, 2023
• First Posted: August 1, 2023
• Study Start: October 24, 2023
• Primary Completion (Estimated): May 1, 2027
• Study Completion (Estimated): July 31, 2028
• Last Updated: April 10, 2025

Record last verified: 2025-04

Locations

PL (2)