Prevention of myeloid leukaemias in children with Down's syndrome and Transient Myeloproliferative Disorder

Status

Active, not recruiting

Phases

Unknown

Study type

Interventional

Source

ISRCTN

Registry ID

ISRCTN54575263

Enrollment

100

Registered

2007-07-02

Start date

2007-05-01

Completion date

Unknown

Last updated

2015-01-13

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

Conditions

Transient myeloproliferative disorder in children with Down's syndrome Cancer Neoplasms

Interventions

Experimental intervention: Monitoring of GATA1s positive preleukemic clones, low-dose cytarabine treatment in children with persisting GATA1s clone. Control intervention: None, historical controls

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: TMD with GATA1s mutation and myeloproliferation (greater than 5% blasts in peripheral blood or bone marrow).

Exclusion criteria

Exclusion criteria: 1. No consent 2. No trisomy 21

Design outcomes

Primary

Measure	Time frame
Reduction of Down's Syndrome Myeloid Leukaemia (DS-ML) risk in children with TMD from 22% to 7%.	—

Secondary

Measure	Time frame
1. Key secondary endpoint: GATA1s negativity (sensitivity 10-3/-4) at week 12 2. Assessment of safety: Serious Adverse Events (SAE)/Suspected Unexpected Serious Adverse Reaction (SUSAR) reporting system, long-term follow-up of late adverse effects, data monitoring committee	—

Countries

Czech Republic, Germany, Netherlands, Slovakia

Outcome results

None listed

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