Mifamurtide Combined With Post-operative Chemotherapy for Newly Diagnosed High Risk Osteosarcoma Patients

NCT ID: NCT03643133

Last Updated: 2025-12-26

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

ACTIVE_NOT_RECRUITING

Clinical Phase

PHASE2

Total Enrollment

60 participants

Study Classification

INTERVENTIONAL

Study Start Date

2018-10-23

Study Completion Date

2033-10-31

Brief Summary

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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).

Detailed Description

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Multicentre, randomised, open-label, phase II trial, with 2 parallel groups. After pre-operative chemotherapy and surgery of the primary tumour and lung metastases (if applicable), patients presenting high-risk osteosarcoma (defined as metastatic osteosarcoma at diagnosis or localised osteosarcoma with poor histological response) will be randomised between 2 arms:

* Control arm: post-operative chemotherapy alone (with regimens adapted to the age of patient)
* Experimental arm : post-operative chemotherapy combined with mifamurtide

This randomised trial is part of a study recruiting all patients ≤50 years old with a newly diagnosed high-grade osteosarcoma.

Conditions

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Osteosarcoma

Keywords

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Bone Neoplasm, bone tissue Acetylmuramyl-Alanyl-Isoglutamine

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Control arm

Post-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

Group Type ACTIVE_COMPARATOR

EI or M-API regimen depending on patient age

Intervention Type COMBINATION_PRODUCT

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

Experimental arm

Post-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

Group Type EXPERIMENTAL

Mifamurtide

Intervention Type DRUG

48 doses overall over 36 weeks

EI or M-API regimen depending on patient age

Intervention Type COMBINATION_PRODUCT

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

Interventions

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Mifamurtide

48 doses overall over 36 weeks

Intervention Type DRUG

EI or M-API regimen depending on patient age

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

Intervention Type COMBINATION_PRODUCT

Other Intervention Names

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MEPACT

Eligibility Criteria

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Inclusion Criteria

1. Patient with a histologically proven, confirmed by experts pathologists panel (before surgery at the latest), high-grade osteosarcoma
2. Registered at diagnosis into the study
3. Primary tumour resected after pre-operative chemotherapy
4. Osteosarcoma classified as high risk because of at least one risk factor:

1. presence of distant metastases or skip metastases at diagnosis
2. and/or poor histological response to pre-operative chemotherapy (\>10% residual viable cells on the analysis of the primary tumour surgical specimen)
5. Pre-operative chemotherapy combining

1. Methotrexate-Etoposide-Ifosfamide (M-EI regimen) for patients ≤25 years
2. Doxorubicin-Cisplatin-Ifosfamide (API-AI regimen) for patients 26-50 years
6. Screening laboratory values must meet the following criteria (using CTCAE v4) and should be obtained within 7 days prior to randomisation:

1. Absolute neutrophil count ≥1.0 x 10⁹/L
2. Platelets ≥100 x 10⁹/L
3. Haemoglobin ≥8.0 g/mL
4. 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
5. 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
6. Creatinine clearance ≥60 mL/min/1.73 m² according to the Schwartz or Cockcroft formula according to patient's age
7. Women of childbearing potential must have a negative serum or urine pregnancy test (minimum sensitivity 25 IU/L or equivalent units of HCG) done within 7 days prior to randomisation
8. Provision of dated and signed written informed consent for the randomised trial prior to any study specific procedures, sampling and analyses.
9. Patient fit to undergo protocol treatment and follow-up
10. Affiliation to a social insurance regimen

Exclusion Criteria

1. Low grade osteosarcoma, parosteal or periosteal osteosarcoma
2. 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.
3. Osteosarcoma with multiple metastases for whom complete removal is not expected to be feasible even after shrinkage with chemotherapy
4. Progressive disease at any site under initial chemotherapy, confirmed before randomisation time, and not totally resected during surgery
5. Any medical condition precluding treatment with protocol chemotherapy
6. 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
7. Pregnancy or breast-feeding
8. Hypersensitivity to the active substance or to any of the excipients
9. Concurrent use of immunodepressive treatment such as cyclosporine, tacrolimus or other calcineurin inhibitors
10. Concurrent use with high-dose non-steroidal anti-inflammatory drugs (NSAIDs, cyclooxygenase inhibitors)
11. Inflammatory or auto-immune disease, allergy or asthma requiring a chronic use of steroid treatment that cannot be stopped.
12. Patients with positive test for human immunodeficiency virus (HIV) or known acquired immunodeficiency syndrome (AIDS).
13. 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.
Maximum Eligible Age

50 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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UNICANCER

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Nathalie MD GASPAR, PhD

Role: PRINCIPAL_INVESTIGATOR

Gustave Roussy Cancer Campus

Sophie MD PIPERNO-NEUMANN, PhD

Role: PRINCIPAL_INVESTIGATOR

Institut Curie

Locations

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CHU Amiens-Picardie - Service d'oncologie hématologie pédiatrique

Amiens, , France

Site Status

CHU d'Angers - Service d'oncologie pédiatrique

Angers, , France

Site Status

Institut Bergonié - Service d'oncologie médicale

Bordeaux, , France

Site Status

CHU de Caen - Service d'oncologie hématologie pédiatrique

Caen, , France

Site Status

CHU de Grenoble - Service d'oncologie hématologie pédiatrique

La Tronche, , France

Site Status

Centre Oscar Lambret - Unité d'onco-pédiatrie

Lille, , France

Site Status

Centre Léon Bérard - IHOPE

Lyon, , France

Site Status

Centre Léon Bérard - Service d'oncologie médicale

Lyon, , France

Site Status

Hôpital de la Timone - service d'oncologie médicale

Marseille, , France

Site Status

Hôpital de la Timone - Service d'oncologie pédiatrique

Marseille, , France

Site Status

CHU Arnaud de Villeneuve - Onco-hématologie pédiatrique

Montpellier, , France

Site Status

Institut régional du Cancer de Montpellier - Service d'oncologie médicale

Montpellier, , France

Site Status

CHU de Nantes - Service d'oncologie hématologie pédiatrique

Nantes, , France

Site Status

CHU de Nice - Service d'oncologie hématologie pédiatrique

Nice, , France

Site Status

Institut Curie - Service d'oncologie médicale

Paris, , France

Site Status

Hôpital Armand Trousseau - Service d'hématologie et d'oncologie pédiatrique

Paris, , France

Site Status

Hôpital Cochin

Paris, , France

Site Status

Institut Curie - Service d'oncologie pédiatrique

Paris, , France

Site Status

Centre Eugène Marquis - Service d'oncologie médicale

Rennes, , France

Site Status

Hôpital Charles Nicolle - Hémato-Immuno-Oncologie Pédiatrique

Rouen, , France

Site Status

Institut de Cancérologie de l'Ouest (Site René Gauducheau) - Service d'oncologie médicale

Saint-Herblain, , France

Site Status

Hôpital de Hautepierre - Onco-hématologie adulte

Strasbourg, , France

Site Status

Hôpital Hautepierre - Onco-hématologie pédiatrique

Strasbourg, , France

Site Status

CHU Toulouse - Hôpital des Enfants - Service d'Hémato-Immuno-Oncologie

Toulouse, , France

Site Status

Institut Claudius Regaud - service d'oncologie médicale

Toulouse, , France

Site Status

CHU Bretonneau - Service d'oncologie médicale

Tours, , France

Site Status

Hôpital Clocheville - Hématologie et oncologie pédiatrique

Tours, , France

Site Status

CHRU de Nancy - Onco-hématologie pédiatrique

Vandœuvre-lès-Nancy, , France

Site Status

Institut de Cancérologie de Lorraine - Service d'oncologie médicale

Vandœuvre-lès-Nancy, , France

Site Status

Institut Gustave Roussy - Service de cancérologie de l'enfant et de l'adolescent

Villejuif, , France

Site Status

Institut Gustave Roussy - Service d'oncologie médicale

Villejuif, , France

Site Status

Countries

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France

References

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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.

Reference Type DERIVED
PMID: 31401903 (View on PubMed)

Brard 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.

Reference Type DERIVED
PMID: 31110092 (View on PubMed)

Other Identifiers

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2017-001165-24

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

UC-0150/1704

Identifier Type: -

Identifier Source: org_study_id