Mifamurtide Combined With Post-operative Chemotherapy for Newly Diagnosed High Risk Osteosarcoma Patients
SARCOME13
Multicentre, Randomised, Phase 2 Trial of Mifamurtide Combined With Post-operative Chemotherapy for Newly Diagnosed High Risk Osteosarcoma Patients (Metastatic Osteosarcoma at Diagnosis or Localised Disease With Poor Histological Response)
2 other identifiers
interventional
60
1 country
31
Brief Summary
Trial evaluating the impact on efficacy of mifamurtide as add-on treatment to post-operative chemotherapy compared to post-operative chemotherapy alone in first-line treatment of patients with high-risk osteosarcoma (defined as metastatic osteosarcoma at diagnosis or localised osteosarcoma with poor histological response).
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P50-P75 for phase_2
Started Oct 2018
Longer than P75 for phase_2
31 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
July 11, 2018
CompletedFirst Posted
Study publicly available on registry
August 22, 2018
CompletedStudy Start
First participant enrolled
October 23, 2018
CompletedPrimary Completion
Last participant's last visit for primary outcome
January 1, 2026
CompletedStudy Completion
Last participant's last visit for all outcomes
October 1, 2033
ExpectedDecember 26, 2025
December 1, 2025
7.2 years
July 11, 2018
December 18, 2025
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Compare event-free survival in the treatment arms
Event-free survival defined as the time duration from randomisation to time of first event (loco-regional or distant relapse or progression, second malignancy, death from any cause)
Expected average duration of 3 years from randomization
Secondary Outcomes (3)
Compare overall survival in the treatment arms
Up to 10 years from randomization
Compare actual and planned cumulative dose and dose intensity of mifamurtide
Up to 36 weeks from randomization (until end of treatment)
Compare the incidence of adverse events in the treatment arms
Up to 40 weeks from randomization (4 weeks after end of treatment)
Study Arms (2)
Control arm
ACTIVE COMPARATORPost-operative chemotherapy alone (EI or M-API regimen depending on patient age) : M-API regimen (≤25 years) : Doxorubicin 60 mg/m², Day 1 Ifosfamide 3 g/m² Day 1 and 2 Cisplatin 100 mg/m², Day 2 EI regimen (26-50 years) : Etoposide 75 mg/m²/d, Day 1-4 Ifosfamide 3 g/m²/d, Day 1-4
Experimental arm
EXPERIMENTALPost-operative chemotherapy (EI or M-API regimen) combined with Mifamurtide 2 mg/m² twice weekly post-randomisation for 12 weeks then weekly for 24 weeks
Interventions
M-API regimen: One course of high-dose Methotrexate (optional) followed by 5 courses of API, every 21 days EI regimen : 5 course of EI, every 21 days
Eligibility Criteria
You may qualify if:
- Patient with a histologically proven, confirmed by experts pathologists panel (before surgery at the latest), high-grade osteosarcoma
- Registered at diagnosis into the study
- Primary tumour resected after pre-operative chemotherapy
- Osteosarcoma classified as high risk because of at least one risk factor:
- presence of distant metastases or skip metastases at diagnosis
- and/or poor histological response to pre-operative chemotherapy (\>10% residual viable cells on the analysis of the primary tumour surgical specimen)
- Pre-operative chemotherapy combining
- Methotrexate-Etoposide-Ifosfamide (M-EI regimen) for patients ≤25 years
- Doxorubicin-Cisplatin-Ifosfamide (API-AI regimen) for patients 26-50 years
- Screening laboratory values must meet the following criteria (using CTCAE v4) and should be obtained within 7 days prior to randomisation:
- Absolute neutrophil count ≥1.0 x 10⁹/L
- Platelets ≥100 x 10⁹/L
- Haemoglobin ≥8.0 g/mL
- Alanine aminotransferase (ALT)/aspartate aminotransferase (AST) ≤2.5 x upper limit of normal (ULN) in the absence of liver metastases or ≤5 x ULN in the presence of liver metastases
- Total Bilirubin ≤2 x ULN (except Gilbert Syndrome: \<3.0 mg/dL) or Total Bilirubin ≤5.0 x ULN in the presence of liver metastases
- +5 more criteria
You may not qualify if:
- Low grade osteosarcoma, parosteal or periosteal osteosarcoma
- Prior history of other malignancies other than study disease (except for basal cell or squamous cell carcinoma of the skin or carcinoma in situ of the cervix) unless the patient has been free of the disease for at least 3 years.
- Osteosarcoma with multiple metastases for whom complete removal is not expected to be feasible even after shrinkage with chemotherapy
- Progressive disease at any site under initial chemotherapy, confirmed before randomisation time, and not totally resected during surgery
- Any medical condition precluding treatment with protocol chemotherapy
- Fractional Shortening \<28% or left ventricular ejection fraction (LVEF) 50% before treatment (only for API post-operative chemotherapy) by echocardiogram or multigated acquisition (MUGA) scan
- Pregnancy or breast-feeding
- Hypersensitivity to the active substance or to any of the excipients
- Concurrent use of immunodepressive treatment such as cyclosporine, tacrolimus or other calcineurin inhibitors
- Concurrent use with high-dose non-steroidal anti-inflammatory drugs (NSAIDs, cyclooxygenase inhibitors)
- Inflammatory or auto-immune disease, allergy or asthma requiring a chronic use of steroid treatment that cannot be stopped.
- Patients with positive test for human immunodeficiency virus (HIV) or known acquired immunodeficiency syndrome (AIDS).
- Patients with positive tests for hepatitis B virus surface antigen (HBV sAg) or hepatitis C virus ribonucleic acid (HCV RNA) indicating active or chronic infection.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- UNICANCERlead
Study Sites (31)
CHU Amiens-Picardie - Service d'oncologie hématologie pédiatrique
Amiens, France
CHU d'Angers - Service d'oncologie pédiatrique
Angers, France
Institut Bergonié - Service d'oncologie médicale
Bordeaux, France
CHU de Caen - Service d'oncologie hématologie pédiatrique
Caen, France
CHU de Grenoble - Service d'oncologie hématologie pédiatrique
La Tronche, France
Centre Oscar Lambret - Unité d'onco-pédiatrie
Lille, France
Centre Léon Bérard - IHOPE
Lyon, France
Centre Léon Bérard - Service d'oncologie médicale
Lyon, France
Hôpital de la Timone - service d'oncologie médicale
Marseille, France
Hôpital de la Timone - Service d'oncologie pédiatrique
Marseille, France
CHU Arnaud de Villeneuve - Onco-hématologie pédiatrique
Montpellier, France
Institut régional du Cancer de Montpellier - Service d'oncologie médicale
Montpellier, France
CHU de Nantes - Service d'oncologie hématologie pédiatrique
Nantes, France
CHU de Nice - Service d'oncologie hématologie pédiatrique
Nice, France
Institut Curie - Service d'oncologie médicale
Paris, 75000, France
Hôpital Armand Trousseau - Service d'hématologie et d'oncologie pédiatrique
Paris, France
Hôpital Cochin
Paris, France
Institut Curie - Service d'oncologie pédiatrique
Paris, France
Centre Eugène Marquis - Service d'oncologie médicale
Rennes, France
Hôpital Charles Nicolle - Hémato-Immuno-Oncologie Pédiatrique
Rouen, France
Institut de Cancérologie de l'Ouest (Site René Gauducheau) - Service d'oncologie médicale
Saint-Herblain, France
Hôpital de Hautepierre - Onco-hématologie adulte
Strasbourg, France
Hôpital Hautepierre - Onco-hématologie pédiatrique
Strasbourg, France
CHU Toulouse - Hôpital des Enfants - Service d'Hémato-Immuno-Oncologie
Toulouse, France
Institut Claudius Regaud - service d'oncologie médicale
Toulouse, France
CHU Bretonneau - Service d'oncologie médicale
Tours, France
Hôpital Clocheville - Hématologie et oncologie pédiatrique
Tours, France
CHRU de Nancy - Onco-hématologie pédiatrique
Vandœuvre-lès-Nancy, France
Institut de Cancérologie de Lorraine - Service d'oncologie médicale
Vandœuvre-lès-Nancy, France
Institut Gustave Roussy - Service de cancérologie de l'enfant et de l'adolescent
Villejuif, 94800, France
Institut Gustave Roussy - Service d'oncologie médicale
Villejuif, France
Related Publications (2)
Hattinger CM, Patrizio MP, Magagnoli F, Luppi S, Serra M. An update on emerging drugs in osteosarcoma: towards tailored therapies? Expert Opin Emerg Drugs. 2019 Sep;24(3):153-171. doi: 10.1080/14728214.2019.1654455. Epub 2019 Aug 14.
PMID: 31401903DERIVEDBrard C, Piperno-Neumann S, Delaye J, Brugieres L, Hampson LV, Le Teuff G, Le Deley MC, Gaspar N. Sarcome-13/OS2016 trial protocol: a multicentre, randomised, open-label, phase II trial of mifamurtide combined with postoperative chemotherapy for patients with newly diagnosed high-risk osteosarcoma. BMJ Open. 2019 May 19;9(5):e025877. doi: 10.1136/bmjopen-2018-025877.
PMID: 31110092DERIVED
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Study Officials
- PRINCIPAL INVESTIGATOR
Nathalie MD GASPAR, PhD
Gustave Roussy Cancer Campus
- PRINCIPAL INVESTIGATOR
Sophie MD PIPERNO-NEUMANN, PhD
Institut Curie
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Allocation
- RANDOMIZED
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- OTHER
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
July 11, 2018
First Posted
August 22, 2018
Study Start
October 23, 2018
Primary Completion
January 1, 2026
Study Completion (Estimated)
October 1, 2033
Last Updated
December 26, 2025
Record last verified: 2025-12
Data Sharing
- IPD Sharing
- Will not share
Individual participant data will not be shared at an individual level, they will be part of the study database including all enrolled patients.