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

ISRCTN	ISRCTN54575263
DOI	https://doi.org/10.1186/ISRCTN54575263
Secondary identifying numbers	TMD Prevention 2007

Submission date: 30/05/2007
Registration date: 02/07/2007
Last edited: 17/02/2009

Recruitment status: No longer recruiting
Overall study status: Completed
Condition category: Cancer

Prospectively registered

Protocol

Statistical analysis plan

Results

Individual participant data

Record updated in last year

Plain English summary of protocol

Not provided at time of registration

Study website

Contact information

Dr Dirk Reinhardt
Scientific

Pediatric Hematology/Oncology
Hannover Medical School
Carl-Neuberg-Str. 1
Hannover
30625
Germany

Email	reinhardt.dirk@mh-hannover.de

Study information

Study design	Non-randomised, historically controlled trial
Primary study design	Interventional
Secondary study design	Non randomised controlled trial
Study setting(s)	Hospital
Study type	Prevention
Scientific title
Study acronym	TMD Prevention 2007
Study objectives	Elimination of the preleukaemic clone in children with Down's syndrome and Transient Myeloproliferative Disorder (TMD) to prevent Acute Myeloid Leukaemia (AML). As of 17/02/2009 this record was updated to include the following countries of recruitment: Netherlands, Czech Republic, Slovakia.
Ethics approval(s)	Approved by the Ethical Committee of the Hannover Medical School on the 17th November 2006 (ref: 4378M).
Health condition(s) or problem(s) studied	Transient myeloproliferative disorder in children with Down's syndrome
Intervention	Experimental intervention: Monitoring of GATA1s positive preleukemic clones, low-dose cytarabine treatment in children with persisting GATA1s clone. Control intervention: None, historical controls are used. Duration of intervention per patient: three months
Intervention type	Drug
Pharmaceutical study type(s)
Phase	Not Specified
Drug / device / biological / vaccine name(s)	Cytarabine
Primary outcome measure	Reduction of Down's Syndrome Myeloid Leukaemia (DS-ML) risk in children with TMD from 22% to 7%.
Secondary outcome measures	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
Overall study start date	01/05/2007
Completion date	30/04/2012

Eligibility

Participant type(s)	Patient
Age group	Child
Sex	Both
Target number of participants	100
Key inclusion criteria	TMD with GATA1s mutation and myeloproliferation (greater than 5% blasts in peripheral blood or bone marrow).
Key exclusion criteria	1. No consent 2. No trisomy 21
Date of first enrolment	01/05/2007
Date of final enrolment	30/04/2012

Locations

Countries of recruitment

Czech Republic
Germany
Netherlands
Slovakia

Study participating centre

Pediatric Hematology/Oncology

Hannover
30625
Germany

Sponsor information

Hannover Medical School (Germany)
Hospital/treatment centre

Carl-Neuberg-Str. 1
Hannover
30625
Germany

Email	reinhardt.dirk@mh-hannover.de
Website	http://www.mh-hannover.de/
"ROR"	https://ror.org/00f2yqf98

Funders

Funder type

Research organisation

German Research Foundation (Deutsche Forschungsgemeinschaft [DFG]) (Germany) - (ref: RE 2580/1-1)

No information available

Results and Publications

Intention to publish date
Individual participant data (IPD) Intention to share	No
IPD sharing plan summary	Not provided at time of registration
Publication and dissemination plan	Not provided at time of registration
IPD sharing plan