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European Proof-of-Concept Therapeutic Stratification Trial of Molecular Anomalies in Relapsed or Refractory Tumors | Clinical Trials | Clareo Health

RECRUITINGPHASE1/PHASE2

European Proof-of-Concept Therapeutic Stratification Trial of Molecular Anomalies in Relapsed or Refractory Tumors

NCT02813135•Gustave Roussy, Cancer Campus, Grand Paris

View on ClinicalTrials.gov

Summary

This proof-of-concept platform trial is designed to cover the targeting of several survival pathways in oncogenesis that are currently not adequately employed for pediatric patients in Europe (Geoerger 2017; Geoerger 2019). The aims of the trial are: 1. To determine the recommended phase II dose (RP2D) of a specific anticancer agent and/or a relevant combination in a pediatric population, to document its tolerability and 2. To explore first signals of activity in a molecularly enriched study population.

Detailed Description

The first molecular profiling protocols have been launched in Europe (MOSCATO-01 (Geoerger 2014), MAPPYACTS, INFORM, iTHER, SM-PAEDS, etc.) determining multiple actionable alterations in pediatric recurrent cancers. Increasingly, stratified approaches are being implemented to enrich clinical trials of molecularly targeted agents and possibly improve outcomes in specific populations i.e. a molecularly enriched/predictive biomarker-driven approach. The diversity and heterogeneity of the detected molecular alterations and the low number of pediatric patients mandate an adapted, innovative trial design for the attributed treatment options in order to satisfy the current unmet medical need. This basket trial is designed to cover the targeting of several survival pathways in oncogenesis that are currently not adequately employed for pediatric patients in Europe.

Eligibility

Age

0 - 18 years

Sex

ALL

Healthy Volunteers

Inclusion Criteria

•1. Patients must be diagnosed with a haematologic or solid tumor malignancy that has progressed despite standard therapy, or for which no effective standard therapy exists.
•2. Age \< 18 years at inclusion; patients 18 years and older may be included after discussion with the sponsor if they have a pediatric recurrent/refractory malignancy.
•3. Patient must have had advanced molecular profiling (i.e. WES/WGS +/- RNAseq) of their recurrent or refractory tumor i.e. at the time of disease progression/relapse; exceptionally patients with advanced molecular profiling at diagnosis may be allowed.
•4. Evaluable or measurable disease as defined by standard imaging criteria for the patient's tumor type (RECIST v1.1, RANO criteria for patients with HGG, INRC criteria for patients with NB, Leukemia criteria, etc.).
•5. Patients with relapsed or refractory leukemia are eligible for this study.
•6. Performance status: Karnofsky performance status (for patients \>12 years of age) or Lansky Play score (for patients ≤12 years of age) ≥ 70%. Patients who are unable to walk because of paralysis or stable neurological disability, but who are up in a wheelchair, will be considered ambulatory for the purpose of assessing the performance score.
•7. Life expectancy ≥ 3 months
•8. Adequate organ function:
•Hematologic criteria (Leukemia patients are excluded from hematological criteria):
•* Peripheral absolute neutrophil count (ANC) ≥ 1000/μL(unsupported)
+14 more criteria

Exclusion Criteria

•1. Patients with symptomatic central nervous system (CNS) metastases who are neurologically unstable or require increasing doses of corticosteroids or local CNS-directed therapy to control their CNS disease. Patients on stable doses of corticosteroids for at least 7 days prior to receiving study drug may be included.
•2. Impairment of gastrointestinal (GI) function or GI disease that may significantly alter drug absorption of oral drugs (e.g., ulcerative diseases, uncontrolled nausea, vomiting, diarrhea, or malabsorption syndrome).
•3. Clinically significant, uncontrolled heart disease (including history of any cardiac arrhythmias, e.g., ventricular, supraventricular, nodal arrhythmias, or conduction abnormality, unstable ischemia,congestive heart failure within 12 months of screening)
•4. Active viral hepatitis or known human immunodeficiency virus (HIV) infection or any other uncontrolled infection.
•5. Presence of any ≥ CTCAE grade 2 treatment-related toxicity with the exception of alopecia, ototoxicity or peripheral neuropathy.
•6. Systemic anticancer therapy within 21 days of the first study dose or 5 times its half-life, whichever is less.
•7. Previous myeloablative therapy with autologous hematopoietic stem cell rescue within 8 weeks of the first study drug dose
•9. Radiotherapy (non-palliative) within 21 days prior to the first dose of drug (or within 6 weeks for therapeutic doses of MIBG or craniospinal irradiation).
•10. Major surgery within 21 days of the first dose. Gastrostomy, ventriculo-peritoneal shunt, endoscopic ventriculostomy, tumor biopsy and insertion of central venous access devices are not considered major surgery, but for these procedures, a 48 hour interval must be maintained before the first dose of the investigational drug is administered.
•11. Currently taking medications with a known risk of prolonging the QT interval or inducing Torsades de Pointes (Refer to Appendix 8).
+4 more criteria

Locations (21)

Rigshospitalet

Copenhagen, Denmark

Gustave Roussy

Villejuif, Val De Marne, France

CHU Angers

Angers, France

CHU Pellegrin

Bordeaux, France

Centre Oscar Lambret

Lille, France

Centre Léon Bérard

Lyon, France

Hôpital de La Timone

Marseille, France

CHU Nantes

Nantes, France

Institut Curie

Paris, France

Hôpital Armand Trousseau

Paris, France

Key Dates

Start Date

August 3, 2016

Primary Completion Date

February 1, 2031

Completion Date

February 1, 2031

Last Updated

January 16, 2026

Enrollment

472

ESTIMATED participants

Conditions

Pediatric Cancer

Interventions

Ribociclib

DRUG

Topotecan

DRUG

Temozolomide

DRUG

Everolimus

DRUG

Adavosertib

DRUG

Carboplatin

DRUG

Olaparib

DRUG

Irinotecan

DRUG

Vistusertib

DRUG

Nivolumab

DRUG

Cyclophosphamide

DRUG

Selumetinib

DRUG

Enasidenib

DRUG

Lirilumab

DRUG

Fadraciclib

DRUG

Cytarabine

DRUG

Dexamethasone

DRUG

Ceralasertib

DRUG

Futibatinib

DRUG

Capmatinib

DRUG

Avelumab

DRUG

Peposertib

DRUG

Capivasertib

DRUG

Vinorelbine

DRUG

Contacts

Birgit Geoerger, MD

CONTACT

+33 (0)1 42 11 46 61 birgit.geoerger@gustaveroussy.fr

Estelle Jullemier, MS

CONTACT

+33 (0)1 42 11 55 51 Estelle.JULLEMIER@gustaveroussy.fr

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Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: January 16, 2026Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

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European Proof-of-Concept Therapeutic Stratification Trial of Molecular Anomalies in Relapsed or Refractory Tumors

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