Myeloid Leukemia in Children with Down Syndrome
Conditions
Interventions
Trade Name: Vyxeos liposomal 44mg/100mg Pulver für ein Konzentrat zur Herstellung einer infusionslösung
Product Name: Vyxeos
Product Code: CPX-351
Pharmaceutical Form: Powder for concentrate for solut
Sponsors
Gesellschaft für Pädiatrische Onkologie & Hämatologie (GPOH gGmbH)
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: • Myeloid Leukemia (ML) or Myelodysplastic Syndrome (MDS), according to WHO • Trisomy 21: Down syndrome or mosaic • Age: > 6 months and = 4 years of age with/without GATA1 mutation OR > 4 years of age =65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: • Children with Transient Abnormal Myelopoiesis (TAM), according to WHO • Cytogenetics: AML with recurrent genetic abnormalities (WHO 2016) • Previous allogeneic bone marrow, stem cell or organ transplantation • Evidence of invasive fungal infection or other severe systemic infection requiring treatment doses of systemic/parenteral therapy including known active viral infection with human immunodeficiency virus (HIV) or Hepatitis Type B and C • Symptomatic cardiac disorders (CTCAE 4.0 Grade 3 or 4) • Diagnosed Wilson’s Disease • Major surgery within 21 days of the first dose. • Any anti-cancer therapy (e.g., intensive chemotherapy, biologics or radiotherapy) for more than 14 days or within 4 weeks before start of therapy, except low-dose cytarabine for the treatment of TAM. • Concomitant treatment with any other anticancer therapy except those specified in protocol during the study therapy • Treated by any investigational agent in a clinical study within previous 4 weeks • History of hypersensitivity to the investigational medicinal product or to any drug with similar chemical structure or to any excipient present in the pharmaceutical form of the investigational medicinal product • Former Enrolment to this study • The patient concerned has been committed to an institution by virtue of an order issued either by the judicial or the administrative authorities
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: • Achieving an EFS, which is not inferior to the ML-DS 2006 trial: 5yr-EFS; 87±3%;Primary end point(s): Event-free survival (EFS), defined as time from diagnosis to the first event or last follow-up. Events are death from any cause, failure to achieve remission, relapse, and secondary malignancy. Failure to achieve remission is considered as an event on day 0. ;Timepoint(s) of evaluation of this end point: After 150 are included and the 5-yrs-follow up is over.;Secondary Objective: • Reduction of toxicity: severe adverse events (CTCAE v4.0 grade III or higher) • Identification of prognostic factors concerning the risk of relapse, toxicity and poor outcome • Evaluate the role of different methods in the determination of minimal residual disease measurement • Evaluation of somatic SNVs as a predictive biomarker: relation of patients’ outcome to the specific somatic SNVs • Exploration of the role of trisomy 8 as a predictive biomarker • Exploration of molecular resistance/relapse mechanisms | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): • Overall survival (OS), as defined as the time of diagnosis to death from any cause or last follow-up. • Disease-free survival (DFS) • Early Response Rate (CR, CRp, CRi)) after induction • Treatment-related mortality (TRM) • Minimal residual disease (FACS and NGS) • Adverse events (according to NCI CTCAE v4.0) • Duration of myelosuppression ;Timepoint(s) of evaluation of this end point: After 150 are included and the 5-yrs-follow up is over. | — |
Countries
Austria, Czechia, Denmark, European Union, France, Germany, Greece, Hungary, Italy, Netherlands, Norway, Poland, Portugal, Slovakia, Slovenia, Spain, Sweden, Switzerland
Contacts
Public ContactProf. Jan-Henning Klusmann (LKP)
University Clinic Frankfurt
Outcome results
None listed