Elimination of the preleukemic clone in children with Down syndrome and transient myeloproloferative disorder (TMD) to prevent AML - Model of leukemia prevention - TMD Prevention of leukemia

Phase 1

Conditions: Preleukemic clones have been frequently detected in newborns. The transient myeloproliferative disorder (TMD) represents a preleukemic clone. More than 20% of the patients developed acute megakaryoblastic leukemia (AMKL) within 3 years after TMD. The aim is to eliminate the preleukemic clone either spontaneously or by a low-dose cytostatic treatment to prevent AMKL.

Registration Number: EUCTR2006-002962-20-NL

Lead Sponsor: Hannover Medical School

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: ot Recruiting

Sex: All

Target Recruitment: 100

Inclusion Criteria

Key inclusion criteria: TMD with GATA1s mutation and myeloproliferation (> 5% blasts in peripheral blood or bone marrow)

Are the trial subjects under 18? yes
Number of subjects for this age range:
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

Key exclusion criteria: no consent

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: Monitoring of the GATA1s positive preleukemic clone in TMD <br>Elimination of the GATA1s positive clone to prevent DS-Myeloid leukemia<br>;Secondary Objective: ;Primary end point(s): Primary efficacy endpoint: Reduction of DS-ML risk in children with TMD from 22% to 7%<br><br>Key secondary endpoint: GATA1s negativity (sensitivity 10-3/-4) at week 12<br><br>Assessment of safety: SAE/SUSAR reporting system; longterm follow-up of late adverse effects, Data monitoring committee<br>

Secondary Outcome Measures