Clinical Trial for the Treatment of Myeloid Leukemia in Children with Down Syndrome

Phase 1

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

• Registration Number: EUCTR2018-002988-25-NL
• Lead Sponsor: Gesellschaft für Pädiatrische Onkologie & Hämatologie (GPOH gGmbH)

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2021-04-01
• Last Update Posted: 26 July 2021

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• Sex: All
• Target Recruitment: 150

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

Are the trial subjects under 18? yes
Number of subjects for this age range: 150
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

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)

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 clinical trial of medicinal product
• Study Design: Not specified