Phase III Clinical Trial for CPX-351 in Myeloid Leukemia in Children with Down Syndrome 2018

Recruitment & Eligibility

Status: Recruiting

Sex: Not specified

Target Recruitment: 10

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 < 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

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 Wilsons 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.

Study & Design

Study Type: Interventional

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
<p>Event-free survival (EFS), defined as time from diagnosis to the first event or<br /><br>last follow-up. Events are death from any cause, failure to achieve remission,<br /><br>relapse, and secondary malignancy. Failure to achieve remission is considered<br /><br>as an event on day 0.</p><br>

Secondary Outcome Measures

Name	Time	Method
<p>- Overall survival (OS), as defined as the time of diagnosis to death from any<br /><br>cause or last follow-up.<br /><br>- Disease-free survival (DFS)<br /><br>- Early Response Rate (CR, CRp, CRi) after induction<br /><br>- Treatment-related mortality (TRM)<br /><br>- Minimal residual disease (FACS and NGS)<br /><br>- Adverse events (according to NCI CTCAE v4.0)<br /><br>- Duration of myelosuppression (neutrophils < 0.5 Gpt/L).</p><br>