ML-DS 2018
Phase III clinical trial with CPX-351 in children with Down syndrome 2018
Myeloid leukaemia is a rare form of blood cancer in children and adolescents and affects the entire body. Children with Down syndrome have a greatly increased risk of developing myeloid leukaemia. Although they have a better prognosis than children without Down syndrome, these patients are more susceptible to the side effects of intensive chemotherapy. For these reasons, a specialised treatment for children with ML-DS is necessary. The aim of this study is to assess whether introducing the new medication Vyxeos can reduce side effects without compromising the chances of cure. The study investigates how children respond to this treatment and what side effects they experience. The dose of the medication will also be reduced towards the end of treatment for children who have responded well to chemotherapy in order to minimise side effects. Based on positive data from previous international studies, this treatment is expected to lead to a similar survival rate while greatly reducing both acute and long-term side effects.
Is this study suitable for my child’s condition?
Leukaemia, lymphoma and bone marrow disease
- Acute lymphoblastic leukemia (ALL)
- Acute myeloid leukemia (AML)
- Chronic myeloid leukemia (CML)
- Myelodysplastic syndrome (MDS)
- Severe aplastic anemia (SAA)
- Non-Hodgkin lymphoma
- Hodgkin lymphoma
- Other leukaemia, lymphoma and bone marrow disease
Brain and spinal cord tumors
- Low-grade glioma
- High-grade glioma
- Diffuse-intrinsic pontine glioma (DIPG) / Diffuse midline glioma (DMG)
- Medulloblastoma
- Atypical teratoid rhabdoid tumours (ATRT)
- Ependymoma
- Germ cell tumours / germinoma / nongerminomatous germ cell tumours / teratoma (brain / spinal cord)
- Craniopharyngioma
- Other brain tumours or spinal cord tumours
Solid tumors
- Wilms tumours / nephroblastoma
- Neuroblastoma
- Rhabdomyosarcoma
- Ewing sarcoma
- Osteosarcoma
- Rhabdoid tumours (other than brain or spinal cord)
- Other sarcoma
- Germ cell tumours (other than brain or spinal cord)
- Retinoblastoma
- Hepatoblastoma
- Other solid tumours
Study Information
What age range of children can participate?
Up to 6 years
Official Study Information
Questions? Please contact your medical team.
If you would like more information about a study, please speak to your child’s treating physician. They are your primary point of contact and can provide personalized advice and support. If no treatment team is currently in place, you can find general contact information for the study sites on the map below.
Treatment Centers with Open Study – Map & Overview
The map shows all treatment sites in Switzerland. Locations where the study is currently open are marked in blue. You can find contact details by clicking on the towns. The information on study sites is updated regularly. However, a study may occasionally open at a new site before this is reflected on the platform. If in doubt, please contact your treatment team or one of the listed clinics directly.