The number of children and adolescents with cancer is relatively small so participation in large (mostly academic) clinical trials is essential to gather enough data to draw meaningful conclusions regarding better treatment options. Many children and young adults with cancer are treated in large clinical trials since several decades. This has contributed to the better cure rates for children with cancer in general.
Clinical Trials
What is a clinical trial?
A clinical trial or study is a medical research study conducted in human subjects to find better ways to prevent, detect, diagnose or treat a disease.
In our clinical trials for childhood cancer, the existing standard of care treatment is usually compared to a new drug or a slightly adapted treatment to improve the cure rates and/or to reduce relapses and long-term side effects (therapy optimization trials).
Belgium is part of a large collaboration between European and/or international study groups and experts joining their knowledge and expertise for the set-up and performance of mostly academic clinical trials. If no clinical trial is available for a certain type of cancer, children and young adults will be treated according to the current standard of care treatment or national/international approved guidelines that are available. Every child will always receive the best available treatment.
What are the trial types?
An academic or non-commercial trial is a clinical trial that is not initiated and funded by a pharmaceutical or biotechnology company for commercial ends. These trials are organized by a public non-profit institution, usually a university or university hospital. The main goal of academic clinical trials is the improvement and optimization of already existing therapies (phase III trials). Most of the trials in children with cancer are large academic international therapy optimization trials in order to improve the existing treatment.
Commercial trials are run by pharmaceutical companies, which take responsibility to initiate and fund the trial. A large number of new anti-cancer drugs have become available in recent years. Currently, only a few of these new drugs are approved for paediatric use. However, paediatric clinical trials assessing the use and usefulness of these drugs in children, are gradually becoming more frequent.
Phase I
Phase I trials are testing new drugs, often for the first time and are helping us to find the correct dose. These trials may look at whether a drug or treatment is safe and/or which side effects it causes. They are performed only in a small group of patients, usually for those patients who received already all possible treatment options in the past (advanced cancer patients).
Phase II
Once the correct paediatric dose is found, a phase II trial will test if this treatment is also effective in a larger group of patients, and specifically focuses on the most effective dose and on side effects. A phase II study can also examine if a drug can be given in a different but possibly more effective way. Some phase II trials are randomized, this means that the patients are randomly assigned to one of the different treatment groups.
Phase III
In phase III trials, a new treatment will be compared to the existing standard treatment, involving a larger number of patients. These trials are the most common type of trials performed in children with cancer (therapy optimization trials) and they are mostly randomized. These trials can run over a relatively long time period and are typically organised internationally because large patient numbers are needed.
Do I need to participate?
No, participation in a clinical trial is voluntary. Even after you agreed to participate, you are free to withdraw your child from a clinical trial at any moment without having to justify. This will not affect the medical follow-up of your child or the quality of the subsequent care or relationship with your child’s doctor. Your child will always receive the best care and a child’s best interest remains always primordial.
Your child’s doctor will explain the whole trial and its advantages and disadvantages in detail with you and your child. If you decide that your child will participate, you will be asked to sign an informed consent form. This form explains what the trial is about and the doctor will go through the form together with you. You will receive a copy of this signed consent form.
For patients below 18 years, both parents always need to sign the informed consent form. Adolescents from 12 to 17 years also receive a separate information form and need to sign an assent form. Patients from 8 to 11 years old only receive an information form.
Conducting a clinical trial requires compliance with a large series of rules and legislations in order to protect the study subject (the patient or the child) and to ensure scientific meaningful outcomes. All the clinical trials that are potentially presented to you, are approved by several ethics committees and/or competent authority and are compliant with all applicable Belgian laws regarding clinical trials and data privacy protection.
Which trials are open?
All doctors in the Belgian centers for paediatric haemato-oncology are familiar with the clinical trials that are open and whether your child is eligible to participate or not. If an appropriate clinical trial is available for the treatment of your child in (one of) the Belgian centers, this will usually be discussed with you by the treating paediatric haemato-oncologist.
If there is no clinical trial open in Belgium that fits your child’s disease, your child will be treated with the current standard of care treatment or according to national/international approved guidelines. Your child will always receive the best available treatment. For more information you can contact your child’s doctor anytime.
| Short Name Trial | Phase | Sponsor type | Status trial | Url | cancer_type_hfilter | pathology_hfilter | sponsor_type_hfilter | phase_hfilter |
|---|---|---|---|---|---|---|---|---|
| 1321.7 -idarucizumab | III | Commercial | Open | Link | benign-haematology supportive-care | thromboembolism | commercial | iii |
| Abemaciclib - I3Y-MC-JPCS | I | Commercial | Open | Link | bone-tumour brain-tumour extracranial-germ-cell-tumours hepatic-tumour lymphoma melanoma neuroblastoma renal-tumour retinoblastoma soft-tissue-sarcomas thyroid-tumour | choroid-plexus-tumor craniopharyngeoma desmoplastic-small-round-cell-tumors dipg ependymoma ewing-sarcoma extra-cranial-germ-cell-tumors hepatic-tumor hgg high-risk-neuroblastoma hodgkin intracranial-gct lgg lymphoblastic-lymphoma medulloblastoma melanoma non-high-risk-neuroblastoma non-rms oms-des osteosarcoma renal-tumour retinoblastoma rms synovial-sarcoma thyroid-tumour | commercial | i |
| AC220-A-U202 - Quizartinib | I/II | Commercial | Open | Link | leukemia | aml | commercial | i-ii |
| II/III | Commercial | Open | Link | benign-haematology | sickle-cell-disease | commercial | ii-iii | |
| ALL SCT ped 2012 FORUM | III | Academic | Open | Link | bone-marrow-transplant leukemia | all bone-marrow-transplant | academic | iii |
| ALL Together | III | Academic | Open | Link | leukemia | all | academic | iii |
| AMoRe 2017 | II | Academic | Open | Link | leukemia | aml | academic | ii |
| ASPA | N/A | Academic | Open | leukemia lymphoma | all b-nhl-b-all | academic | n-a | |
| Baxalta 071102 -rVWF | III | Commercial | Open | Link | benign-haematology | vwb-disease | commercial | iii |
| CCTL019A2205B -CAR T-Cell Therapy | N/A | Commercial | Open | Link | leukemia lymphoma | b-nhl-b-all | commercial | n-a |
| I-CML-Ped Registry | Observational | Academic | Open | leukemia | cml | academic | observational | |
| National COVID-19 registry PHO | N/A | Academic | Open | Link | benign-haematology bone-marrow-failure-and-myelodysplasia bone-marrow-transplant bone-tumour brain-tumour extracranial-germ-cell-tumours hepatic-tumour histiocytosis leukemia lymphoma melanoma neuroblastoma renal-tumour retinoblastoma soft-tissue-sarcomas supportive-care | alcl all aml anemia apl aspergillosis-mucomycosis atrt b-nhl-b-all bone-marrow-transplant choroid-plexus-tumor cml craniopharyngeoma dipg ependymoma ewing-sarcoma extra-cranial-germ-cell-tumors gvhd hemophilia hepatic-tumor hgg high-risk-neuroblastoma hlh hodgkin intracranial-gct itp jmml lch lgg lymphoblastic-lymphoma mds medulloblastoma melanoma neutropenia non-high-risk-neuroblastoma non-rms oms-des osteosarcoma renal-tumour retinoblastoma rms saa sickle-cell-disease thalassemia thromboembolism thrombopenia thrombosis vod vwb-disease | academic | n-a |
| STAND | III | Commercial | Open | Link | benign-haematology | sickle-cell-disease | commercial | iii |
| CSEG101B2201 -Crizanlizumab | II | Commercial | Open | Link | benign-haematology supportive-care | sickle-cell-disease vod | commercial | ii |
| CTX001-121 | I, II, III | Commercial | Open | Link | benign-haematology | sickle-cell-disease | commercial | i ii iii |
| DIPG Registry | Observational | Academic | Open | brain-tumour | dipg | academic | observational | |
| Epcorcitimab | I | Commercial | Open | Link | lymphoma | burkitt-lymphoma dlbcl b-nhl-b-all | commercial | i |
| Ependymoma II | II, III | Academic | Open | Link | brain-tumour | ependymoma | academic | ii iii |
| ERN-EuroBloodNet Registry COVID-19 | N/A | Academic | Open | Link | benign-haematology | academic | n-a | |
| EsPhALL2017 | III | Academic | Open | Link | leukemia | all | academic | iii |
| EWOG MDS 2006 | Observational | Academic | Open | Link | bone-marrow-failure-and-myelodysplasia | jmml mds | academic | observational |
| EWOG SAA | Observational | Academic | Open | bone-marrow-failure-and-myelodysplasia | saa | academic | observational | |
| Frontier 2 | III | Commercial | Open | Link | benign-haematology | hemophilia | commercial | iii |
| GENA-25 | N/A | Commercial | Open | Link | benign-haematology | hemophilia | commercial | n-a |
| HEM-POWR -Damoctocog alfa pegol | Observational | Commercial | Open | Link | benign-haematology | hemophilia | commercial | observational |
| Hovon 113 | III | Academic | Open | Link | bone-marrow-transplant supportive-care | bone-marrow-transplant gvhd | academic | iii |
| HR NBL 1 | III | Academic | Open | Link | neuroblastoma | high-risk-neuroblastoma | academic | iii |
| INFORM Registry | Observational | Academic | Open | bone-marrow-failure-and-myelodysplasia bone-tumour brain-tumour extracranial-germ-cell-tumours hepatic-tumour histiocytosis leukemia lymphoma neuroblastoma renal-tumour retinoblastoma soft-tissue-sarcomas | alcl all aml atrt b-nhl-b-all choroid-plexus-tumor cml craniopharyngeoma dipg ependymoma ewing-sarcoma extra-cranial-germ-cell-tumors hepatic-tumor hgg high-risk-neuroblastoma hlh hodgkin intracranial-gct jmml lch lgg lymphoblastic-lymphoma mds medulloblastoma non-high-risk-neuroblastoma non-rms oms-des osteosarcoma renal-tumour retinoblastoma rms saa | academic | observational | |
| IntReALL HR | II | Academic | Open | Link | leukemia | all | academic | ii |
| IntReALL SR 2010 | III | Academic | Open | Link | leukemia | all | academic | iii |
| J1S-MC-JV01JV01 - Campfire -ramucirumab | II | Commercial | Open | Link | soft-tissue-sarcomas | desmoplastic-small-round-cell-tumors | commercial | ii |
| Kraniopharyngeom 2007 | N/A | Academic | Open, Stop randomization | brain-tumour | craniopharyngeoma | academic | n-a | |
| LBL 2018 | III | Academic | Open | Link | lymphoma | lymphoblastic-lymphoma | academic | iii |
| LCH IV | N/A | Academic | Open | Link | histiocytosis | lch | academic | n-a |
| LINES | III | Academic | Open | Link | neuroblastoma | non-high-risk-neuroblastoma | academic | iii |
| LOGGIC Core | N/A | Academic | Open | Link | brain-tumour | lgg | academic | n-a |
| LOXO 292 - LIBRETTO-531 | III | Commercial | Open | Link | thyroid-tumour | thyroid-tumour | commercial | iii |
| Metro-PD1 | I, II | Academic | Open | Link | bone-tumour brain-tumour hepatic-tumour lymphoma neuroblastoma renal-tumour retinoblastoma soft-tissue-sarcomas | alcl atrt b-nhl-b-all choroid-plexus-tumor craniopharyngeoma dipg ependymoma ewing-sarcoma extra-cranial-germ-cell-tumors hepatic-tumor hgg high-risk-neuroblastoma hodgkin intracranial-gct lgg lymphoblastic-lymphoma medulloblastoma non-high-risk-neuroblastoma non-rms oms-des osteosarcoma renal-tumour retinoblastoma rms | academic | i ii |
| NB-SCI | Observational | Academic | Open | neuroblastoma | high-risk-neuroblastoma non-high-risk-neuroblastoma | academic | observational | |
| NETTER-P | II | Commercial | Open | Link | soft-tissue-sarcomas | gep-net paragangliomas pheochromocytoma | commercial | ii |
| NOPHO DBH AML 2012 | III | Academic | Open | Link | leukemia | aml | academic | iii |
| PHITT | III | Academic | Open | Link | hepatic-tumour | hepatic-tumor | academic | iii |
| Ponatinib - INCB 84344-102 | I/II | Commercial | Open | Link | bone-tumour brain-tumour extracranial-germ-cell-tumours hepatic-tumour leukemia neuroblastoma renal-tumour soft-tissue-sarcomas | all aml cml craniopharyngeoma dipg ependymoma ewing-sarcoma extra-cranial-germ-cell-tumors hepatic-tumor hgg high-risk-neuroblastoma hlh intracranial-gct lgg medulloblastoma oms-des osteosarcoma renal-tumour | commercial | i-ii |
| Posaconazole - MK5592-104 | II | Commercial | Open | Link | supportive-care | aspergillosis-mucomycosis | commercial | ii |
| MK5592-127 - Posaconazole | II | Commercial | Open | Link | supportive-care | aspergillosis-mucomycosis | commercial | ii |
| SIOP HRMB | III | Academic | Open | Link | brain-tumour | medulloblastoma | academic | iii |
| SIOP Randomet 2017 | III | Academic | Open | Link | renal-tumour | renal-tumour | academic | iii |
| TARC Biomarker study | Observational | Academic | Open | Link | lymphoma | hodgkin | academic | observational |
| TGM 2013 | N/A | Academic | Guidelines, Open | Link | extracranial-germ-cell-tumours | extra-cranial-germ-cell-tumors | academic | n-a |
| Umbrella SIOP-RTSG 2016 | Observational | Academic | Open | Link | renal-tumour | renal-tumour | academic | observational |