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

Phase 1

• Conditions: Myeloid Leukemia in Children with Down Syndrome; Therapeutic area: Diseases [C] - Hemic and Lymphatic Diseases [C15]

• Registration Number: CTIS2022-501457-37-00
• Lead Sponsor: GPOH gGmbH

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2023-04-14
• Last Update Posted: 22 July 2024

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 185

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, 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

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: Achieving an EFS, which is not inferior to the ML-DS 2006 trial: 5yr-EFS; 87±3%;Secondary Objective: Reduction of toxicity: severe adverse events (CTCAE v4.0 grade III or higher), Identification of prognostic factors (e.g. trisomy 8) concerning the risk of relapse, toxicity and poor outcome, To evaluate the role of different methods in the determination of minimal residual disease measurement, To evaluate the response rate;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.

Secondary Outcome Measures

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