Prevention of myeloid leukaemias in children with Down's syndrome and Transient Myeloproliferative Disorder
Completed
- Conditions
- Transient myeloproliferative disorder in children with Down's syndromeCancerNeoplasms
- Registration Number
- ISRCTN54575263
- Lead Sponsor
- Hannover Medical School (Germany)
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Completed
- Sex
- All
- Target Recruitment
- 100
Inclusion Criteria
TMD with GATA1s mutation and myeloproliferation (greater than 5% blasts in peripheral blood or bone marrow).
Exclusion Criteria
1. No consent
2. No trisomy 21
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Reduction of Down's Syndrome Myeloid Leukaemia (DS-ML) risk in children with TMD from 22% to 7%.<br><br>
- Secondary Outcome Measures
Name Time Method 1. Key secondary endpoint: GATA1s negativity (sensitivity 10-3/-4) at week 12<br>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