Alle maliginiteiten met een ALK mutatie. malignancies with ALK-aberrations pediatric cancer
Conditions
Interventions
Children will be treated with oral LDK378 long as they will benefit from it.
Response assessment every 21 days.
Sponsors
Novartis
Eligibility
Age
2 Years to 17 Years
Inclusion criteria
Inclusion criteria: - Diagnosed with a locally advanced or metastatic malignancy that has progressed despite standard therapy, or for which no effective standard therapy exists ;- Age * 12 months and
Exclusion criteria
Exclusion criteria: - Symptomatic central nervous system (CNS) metastases who are neurologically unstable or require increasing doses of steroids or local CNS-directed therapy (such as radiotherapy, surgery or intrathecal chemotherapy) to control their CNS disease ;- Clinically significant, uncontrolled heart disease ;- Inadequate end organ function as defined by specified laboratory values;- History of known of interstitial lung disease or interstitial pneumonitis, including clinically significant radiation pneumonitis (i.e., affecting activities of daily living or requiring therapeutic intervention).;-Radiotherapy to lungs * 4 weeks prior to starting the study treatment or patients who have not recovered from radiotherapy related toxicities. For all other anatomic sites, radiotherapy * 2 weeks prior to starting the study treatment.;- Use of medications that are known to be strong inhibitors or inducers of CYP3A4/5 that cannot be discontinued at least 1 week prior to start of treatment with LDK378 and for the duration of the study ;- Use of medications that are mainly metabolized by CYP3A4/5 or CYP2C9 that cannot be discontinued at least 1 week prior to start of treatment with LDK378 and for the duration of the study.;- Patient has a history of pancreatitis or history of increased amylase or lipase that was due to pancreatic disease.;Other protocol-defined exclusion criteria may apply
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Primary endpoint: Incidence rate of Dose Limiting Toxicities (DLT) during the first cycle of LDK378 treatment. | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary endpoints: Endpoint 1: Adverse events and serious adverse events, changes in laboratory values, assessments of physical examinations, vital signs and electrocardiograms. Endpoint 2: Plasma concentration time profiles, PK parameters, including but not limited to AUClast, AUCtau, Cmin, Cmax, Tmax, Racc, and T1/2,acc Endpoint 3: Overall response rate (ORR) and duration of response (DOR), progression-free survival (PFS) as per RECIST 1.1 in patients with neuroblastoma and other solid tumors, and by International Working Group (IWG) criteria in patients with lymphoma. MIBG response in patients with neuroblastoma. Resolution of bone marrow disease in patients with neuroblastoma. | — |
Countries
Netherlands
Outcome results
None listed