|- candidate number||5296|
|- NTR Number||NTR1667|
|- ISRCTN||ISRCTN wordt niet meer aangevraagd|
|- Date ISRCTN created|
|- date ISRCTN requested|
|- Date Registered NTR||11-feb-2009|
|- Secondary IDs||2007-168 METC ErasmusMC|
|- Public Title||Screening for transient leukemia in children with Down syndrome.|
|- Scientific Title||Screening for transient myeloproliferative disorder in children with Down syndrome.|
|- hypothesis||Prevention of the progression of TMD to ML-DS by chemotherapeutical profylaxis can be possible. |
|- Healt Condition(s) or Problem(s) studied||Down syndrome, Transient myeloproliferative disorder|
|- Inclusion criteria||1. All children with Down syndrome;|
2. Age < 4 weeks;
3. If blasts are present in pleural or pericardial effusion, or in a liverbiopsy
(in absence of blasts in peripheral blood);
4. Signed informed consent.
|- Exclusion criteria||1. Children in whom the diagnosis Down syndrome can not be confirmed;|
2. Complications that make sampling unwanted or impossible.
|- mec approval received||yes|
|- multicenter trial||yes|
|- Type||Single arm|
|- planned startdate ||1-jan-2008|
|- planned closingdate||1-mrt-2014|
|- Target number of participants||811|
|- Interventions||Treatment will be advised when there is high MRD (above threshold) at week 8.|
|- Primary outcome||1. To determine the population-based frequency of TMD;|
2. To establish and investigate the relation of TMD and ML-DS;
3. To see if treatment of TMD can prevent TMD-associated mortality and the development of ML-DS in later life.
|- Secondary outcome||1. To detect new genetic factors related to the progression of TMD to ML-DS;|
2. To investigate the presence of lymphoid pre-leukemic clones in neonatal blood samples of TMD;
3. To describe the hematological abnormalities and clinical characteristics in patients with and without TMD.
|- Timepoints||1. Time point 1: < week 4;|
2. Time point 2: at week 8;
3. Time point 2A: at week 10, only for those who were treated because of high MRD-levels at week 8;
4. Time point 3: at week 12.
|- Trial web site||http://www.erasmusmc.nl/alkg-cs/242905/243014/243045/Down?version=1|
|- status||open: patient inclusion|
|- CONTACT FOR PUBLIC QUERIES||MD, PhD-student M. Blink|
|- CONTACT for SCIENTIFIC QUERIES|| C.M. Zwaan|
|- Sponsor/Initiator ||Erasmus Medical Center, Rotterdam|
(Source(s) of Monetary or Material Support)
|Stichting Kinderen Kankervrij, Nederland, Stichting Sophia|
|- Publications||Blink M, Buitenkamp TD, van Wouwe JP, van Wering ER, van der Velden VHJ, Zwaan CM. Ontwikkelingen in de diagnostiek en behandeling van leukemie bij kinderen met Down syndroom. Tijdschrift voor Kindergeneeskunde. Accepted for publication.|
|- Brief summary||This is a prospective national screening study in which preferably all newborns with Down syndrome are screened for TMD.
If there is TMD, the children will be evaluated to see if there is a clinical indication for treatment.
Treatment is low-dose cytarabin.
Also, children who have high Minimal Residual Disease at week 8 will be treated.
Goal is to establish MRD-negativity (below threshold) at week 12.
All the children go into follow up to see if and when they develop ML-DS.
|- Main changes (audit trail)|
|- RECORD||11-feb-2009 - 14-sep-2009|