Intrathecal chemotherapy: IT: Methotrexate +/- prednisone/hydrocortisone/cytarabine according to the degree of central nervous involvement
Study summary
HEM-iSMART is a master protocol which investigates multiple investigational medicinal products in children, adolescents and young adults (AYA) with relapsed/refractory (R/R) ALL and LBL. Sub-protocol D is a phase I/II trial evaluating the safety and efficacy of trametinib in combination with dexamethasone, cyclophosphamide and cytarabine in children and AYA with R/R ped ALL/LBL whose tumor present with alterations in the RAS-RAF-MAPK pathway.
Eligibility
Sex
ALL
Min age
1 Year
Max age
21 Years
Healthy volunteers
No
Inclusion criteria
1. Children between 1 year (≥ 12 months) and 18 years of age at the time of first diagnosis and less than 21 years at the time of inclusion. Patients under 6 years old must weigh at least 7 kg at the time of enrollment. Patients over 6 years old must weigh at least 10 kg at the time of enrollment.
2. Performance status: Karnofsky performance status (for patients \>12 years of age) or Lansky Play score (for patients
* 12 years of age) ≥ 50% (Appendix I).
3. Written informed consent from parents/legal representative, patient, and age-appropriate assent before any study specific screening procedures are conducted, according to local, regional or national guidelines.
4. Patients must have had molecular profiling and flow-cytometric analysis of their recurrent or refractory disease at a time-point before the first inclusion into this trial (see section 9.1 of this protocol for detailed description of the molecular diagnostics required). Drug response profiling and methylation is highly recommended but not mandatory.
Patients with molecular profiling at first diagnosis lacking molecular diagnostics at relapse or refractory disease may be allowed to be included after discussion with the sponsor.
5. Patients whose tumor present RAS pathway activating mutations including but not limited to KRAS, NRAS, HRAS, FLT3, PTPN11, MAP2K1, MP2K1 hotspot mutations, cCBL; NF1 del, as detected by molecular profiling.
6. Adequate organ function:
* RENAL AND HEPATIC FUNCTION (Assessed within 48 hours prior to C1D1) :
* Serum creatinine ≤ 1.5 x upper limit of normal (ULN) for age or calculated creatinine clearance as per the Schwartz formula or radioisotope glomerular filtration rate ≥ 60 mL/min/1.73 m2.
* Direct bilirubin ≤ 2 x ULN (≤ 3.0 × ULN for patients with Gilbert's syndrome).
* Alanine aminotransferase (ALT)/serum glutamic pyruvic transaminase (SGPT) ≤ 5 x ULN; aspartate aminotransferase (AST)/serum glutamic oxaloacetic transaminase/SGOT ≤ 5 x ULN. Note: Patients with hepatic disfunction related to the underling disease can be eligible even if they do not fulfill the aforementioned values for hepatic transaminases. In these cases, patients need to be discussed with the sponsor to confirm the eligibility.
* CARDIAC FUNCTION:
* Shortening fraction (SF) \>29% (\>35% for children \< 3 years) and/or left ventricular ejection fraction (LVEF) ≥50% at baseline, as determined by echocardiography or MUGA.
* Absence of QTcF prolongation (QTc prolongation is defined as \>450 msec on baseline ECG, using the Fridericia correction), or other clinically significant ventricular or atrial arrhythmia.
Exclusion Criteria
7. Pregnancy or positive pregnancy test (urine or serum) in females of childbearing potential. Pregnancy test must be performed within 7 days prior to C1D1.
8. Sexually active participants not willing to use highly effective contraceptive method (pearl index \<1) as defined in CTFG HMA 2020 (Appendix II) during trial participation and until 6 months after end of antileukemic therapy.
9. Breast feeding.
10. 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) in case of oral IMPs.
11. Have a known immediate or delayed hypersensitivity reaction or idiosyncrasy to the study drugs, or drugs chemically related to study treatment or excipients that contraindicate their participation, including conventional chemotherapeutics (i.e. cytarabine and cyclophosphamide, intrathecal agents) and corticoids.
12. Known active viral hepatitis or known human immunodeficiency virus (HIV) infection or any other uncontrolled infection.
13. Severe concomitant disease that does not allow treatment according to the protocol at the investigator's discretion.
14. Subjects unwilling or unable to comply with the study procedures.
15. Previous treatment with trametinib.
16. Current use of a prohibited medication or herbal preparation or requires any of these medications during the study.
See Section 7 and Appendix III for details. Drugs inducing QTc changes (prolongation of the QT interval or inducing Torsade de Points) are not permitted.
17. Unresolved toxicity greater than NCI CTCAE v 5.0 ≥ grade 2 from previous anti-cancer therapy, including major surgery, except those that in the opinion of the investigator are not clinically relevant given the known safety/toxicity profile of the study treatment (e.g., alopecia and/or peripheral neuropathy related to platinum or vinca alkaloid based chemotherapy) (Common Terminology Criteria for Adverse Events (CTCAE) (cancer.gov).
18. Active acute graft versus host disease (GvHD) of any grade or chronic GvHD of grade 2 or higher. Patients receiving any agent to treat or prevent GvHD post bone marrow transplant are not eligible for this trial.
19. Received immunosuppression post allogenic HSCT within one moth of study entry.
20. History or current evidence of retina vein occlusion (RVO) or central serous retinopathy are excluded.
21. Wash-out periods of prior medication:
1. CHEMOTHERAPY: At least 7 days must have elapsed since the completion of cytotoxic therapy, with the exception of hydroxyurea, 6-mercaptopurine, oral methotrexate and steroids which are permitted up until 48 hours prior to initiating protocol therapy. Patients may have received intrathecal therapy (IT) at any time prior to study entry.
2. RADIOTHERAPY: Radiotherapy (non-palliative) within 21 days prior to the first dose of drug. Palliative radiation in past 21 days is allowed.
3. HEMATOPOIETIC STEM CELL TRANSPLANTATION (HSCT): Autologous HSCT within 2 months prior to the first study drug dose; Allogeneic HSCT within 3 months prior to the first study drug dose.
4. IMMUNOTHERAPY: At least 42 days must have elapsed after the completion of any type of immunotherapy other than monoclonal antibodies (e.g. CAR-T therapy)
5. MONOCLONAL ANTIBODIES AND INVESTIGATIONAL DRUGS: At least 21 days or 5 times the half-life (whichever is shorter) from prior treatment with monoclonal antibodies or any investigational drug under investigation must have elapsed before the first study drug.
6. SURGERY: 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.
Primary outcome measure(s)
Phase I: Maximum tolerated dose (MTD) / Recommended phase 2 dose (RP2D) — 3 years Defined as the highest dose level tested at which 0/6 or 1/6 patients experiences dose limiting toxicities (DLT) during course 1 with at least 2 patients experiencing DLT at the next higher dose
Phase II: Best Overall Response Rate (ORR) — 6 years For patients with leukemia: CR and MRD response after 1 cycle of treatment. This includes determination of CR, CRp, CRi and minimal residual disease (MRD) negativity rate in patients suffering from overt morphological relapse of T-ALL at time of enrolment (morphological disease (M2/M3)), and the MRD negativity rate in those that entered with high-MRD levels but in morphological CR. These results will together be presented as a composite endpoint Overall Response rate (ORR). MRD negativity will be defined as ≤1x10-4 as generated by multi-parameter flow cytometry.
For patients with lymphoma: Response in LBL patients is defined as CR, PR, minor response (MR) as defined in International pediatric NHL response criteria. In case of bone-marrow involvement MRD will be taken into account.
For patients with lymphoma: Response in LBL patients is defined as CR, PR, minor response (MR) as defined in International pediatric NHL response criteria
Trial sites (36)
Facility
City
Region
Status
St. Anna Kinderspital
Vienna
Austria
Not Yet Recruiting
Universitair Ziekenhuis Gent
Ghent
Belgium
Not Yet Recruiting
Rigshospitalet Copenhagen
Copenhagen
Denmark
Recruiting
Helsinki University Hospital, New Children's Hospital
Helsinki
Finland
Not Yet Recruiting
Hôpital des Enfants GH Pellegrin - CHU de Bordeaux
Bordeaux
France
Not Yet Recruiting
CHRU Lille - Hôpital Jeanne de Flandre
Lille
France
Not Yet Recruiting
Centre Léon Bérard
Lyon
France
Not Yet Recruiting
Hopital La Timone - Enfants
Marseille
France
Not Yet Recruiting
CHU Nantes Hôpital Mère-Enfant
Nantes
France
Not Yet Recruiting
Hôpital Robert Debré
Paris
France
Not Yet Recruiting
Universitätsklinikum Augsburg
Augsburg
Germany
Not Yet Recruiting
Charité Universitätsmedizin Berlin
Berlin
Germany
Recruiting
Universitätsklinikum Essen
Essen
Germany
Not Yet Recruiting
Universitätsklinikum Frankfurt
Frankfurt
Germany
Not Yet Recruiting
Universitätsklinikum Münster
Münster
Germany
Not Yet Recruiting
Our Lady's Hospital for Sick Children
Dublin
Ireland
Not Yet Recruiting
Schneider's Children's Medical Center
Petah Tikva
Israel
Not Yet Recruiting
Sheba Medical Center Hospital
Ramat Gan
Israel
Not Yet Recruiting
IRCCS Istituto Giannina Gaslini
Genova
Italy
Not Yet Recruiting
Fondazione MBBM c/o Centro ML Verga
Monza
Italy
Not Yet Recruiting
Padova Azienda Ospedaliera
Padova
Italy
Not Yet Recruiting
Ospedale Pediatrico Bambino Gesù, IRCCS
Roma
Italy
Not Yet Recruiting
Ospedale Infantile Regina Margherita
Turin
Italy
Not Yet Recruiting
Princess Máxima Center for Pediatric Oncology
Utrecht
Utrecht
Recruiting
Oslo University Hospital
Oslo
Norway
Recruiting
Hospital Vall d'Hebron
Barcelona
Spain
Not Yet Recruiting
Hospital Sant Joan de Déu de Barcelona
Barcelona
Spain
Not Yet Recruiting
Hospital Infantil Universitario Niño Jesús
Madrid
Spain
Not Yet Recruiting
La Fe
Valencia
Spain
Not Yet Recruiting
Karolinska university hospital
Stockholm
Sweden
Recruiting
University Children's Hospital Zürich
Zurich
Switzerland
Not Yet Recruiting
Bristol Royal Hospital for Children
Bristol
United Kingdom
Not Yet Recruiting
Great Ormond Street Hospital for Children NHS Trust
London
United Kingdom
Not Yet Recruiting
Royal Manchester Children's Hospital
Manchester
United Kingdom
Not Yet Recruiting
Great North Children's Hospital
Newcastle
United Kingdom
Not Yet Recruiting
Royal Marsden NHS Trust
Sutton
United Kingdom
Not Yet Recruiting
More Princess Maxima Center for Pediatric Oncology trials in Germany
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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