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Phase III Clinical Trial for CPX-351 in Myeloid Leukemia in Children with Down Syndrome 2018
EU CTIS ID: 2022-501457-37-00
What this study is testing
Achieving an EFS, which is not inferior to the ML-DS 2006 trial: 5yr-EFS; 87±3%
- Therapeutic confirmatory (Phase III)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- Trisomy 21: Down syndrome or mosaic
- Age: > 6 months and ≤ 4 years of age with/without GATA1 mutation OR > 4 years of age < 6 years of age with GATA1 mutation
- Morphology/Immunophenotyping: FAB M0, M6 or M7
- Lansky performance score at least equal to 50; or Karnofsky performance status at least equal to 50, whichever is applicable
- Understand and voluntarily provide written permission of parental/legal representative(s) to the ICF prior to conducting any study related assessments/procedures, also concerning data and tumor material transfer according to ICH/GCP and national/local regulations
- Able to adhere to the study visit schedule and other protocol requirements
You likely can't join if
- Children with Transient Abnormal Myelopoiesis (TAM), according to WHO
- 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
- Cytogenetics: AML with recurrent genetic abnormalities (WHO 2016)
See the full eligibility criteria
- Trisomy 21: Down syndrome or mosaic
- Age: > 6 months and ≤ 4 years of age with/without GATA1 mutation OR > 4 years of age < 6 years of age with GATA1 mutation
- Morphology/Immunophenotyping: FAB M0, M6 or M7
- Lansky performance score at least equal to 50; or Karnofsky performance status at least equal to 50, whichever is applicable
- Understand and voluntarily provide written permission of parental/legal representative(s) to the ICF prior to conducting any study related assessments/procedures, also concerning data and tumor material transfer according to ICH/GCP and national/local regulations
- Able to adhere to the study visit schedule and other protocol requirements
- Acceptance that vaccination with live vaccines is not possible while participating in the trial
- Myeloid Leukemia (ML) or Myelodysplastic Syndrome (MDS), according to WHO
- Children with Transient Abnormal Myelopoiesis (TAM), according to WHO
- 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
- 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
The study team makes the final eligibility decision.
Where it's taking place
- Switzerland
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 0-17 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Switzerland. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.