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

Status

Active, not recruiting

Phases

Unknown

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2006-002962-20-DE

Enrollment

100

Registered

2006-11-22

Start date

2007-03-15

Completion date

Unknown

Last updated

2015-08-04

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

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.

Interventions

Pharmaceutical Form: Injection INN or Proposed INN: CYTARABINE CAS Number: 147944 Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 20-

Eligibility

Sex/Gender

All

Inclusion criteria

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

Exclusion criteria: Key exclusion criteria: no consent

Design outcomes

Primary

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

Measure

Time frame

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

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Countries

Germany, Netherlands

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026