|- candidate number||14118|
|- NTR Number||NTR3758|
|- ISRCTN||ISRCTN wordt niet meer aangevraagd.|
|- Date ISRCTN created|
|- date ISRCTN requested|
|- Date Registered NTR||18-dec-2012|
|- Secondary IDs||2012/329 / NL-41213-042-12; METC / ABR|
|- Public Title||The effect of intranasal insulin on development and behaviour of children with Phelan-McDermid syndrome.|
Dutch: Het effect van insuline neusspray op ontwikkeling en gedrag van kinderen met het Phelan-McDermid syndroom.
|- Scientific Title||The effect of intranasal insulin on development and behaviour of children with Phelan-McDermid syndrome.|
Dutch: Het effect van intranasale insuline op ontwikkeling en gedrag van kinderen met het Phelan-McDermid syndroom.
|- hypothesis||Intranasal insulin will improve development and behaviour in children with Phelan-McDermid syndrome.|
|- Healt Condition(s) or Problem(s) studied||Phelan-McDermid syndrome, 22q13.3 deletion syndrome, Insuline, Behaviour, Development, Intranasal|
|- Inclusion criteria||1. Age between 12 months and 18 years 0 months old at 1-1-2013;|
2. Proven SHANK3 deletion by array-comparative genomic hybridization (array-CGH);
3. Parents need to speak and understand Dutch.
|- Exclusion criteria||1. A contra-indication for the use of intranasal application (e.g. anatomical obstruction);|
2. Severe perinatal brain damage (e.g. asphyxia, haemorrhage, infection);
3. A metabolic or muscle disease responsible for neurological symptoms, independent of the 22q13 deletion.
|- mec approval received||yes|
|- multicenter trial||no|
|- Type||2 or more arms, randomized|
|- planned startdate ||1-jan-2013|
|- planned closingdate||1-jan-2015|
|- Target number of participants||20|
|- Interventions||Clinical trial with stepped wedge design. Participants will start with either intranasal insulin or placebo, once or twice a day, one puff in one or both nostrils (dependent on body weight). By steps, groups of participants will convert from placebo to intranasal insulin and remain on insulin until the end of the study. |
|- Primary outcome||Developmental pace, calculated as the difference in developmental age equivalent between two assessments divided by the difference in calendar age in months at the time of these assessments (typically 6 months).|
|- Secondary outcome||Behaviour, improvement of behaviour represented by the increase in test scores between assessment (typically 6 months).|
|- Timepoints||T = 0, 6, 12 and 18 months.|
|- Trial web site||N/A|
|- CONTACT FOR PUBLIC QUERIES|| C.M.A. Ravenswaaij-Arts, van|
|- CONTACT for SCIENTIFIC QUERIES|| C.M.A. Ravenswaaij-Arts, van|
|- Sponsor/Initiator ||University Medical Center Groningen (UMCG)|
(Source(s) of Monetary or Material Support)
|ZON-MW, The Netherlands Organization for Health Research and Development|
|- Publications||On the moment of registration, the study has yet to begin. Therefore there currently are no publications concerning this study.|
|- Brief summary||Children with Phelan-McDermid syndrome have a severe general developmental delay and behavioural problems. The syndrome is caused by a deletion of 22q13.3 and the neurological problems are thought to result from haploinsufficiency of the SHANK3 gene. The SHANK3 protein is located in the postsynaptic density of neurons in conjunction with the insulin receptor. Insulin exerts effects on signal transduction and protein interactions in the postsynaptic density. Previous studies with intranasally administered insulin show a beneficial effect on cognitive function, declarative memory and behaviour. Moreover, a pilot study with six children demonstrated that intranasal insulin improves development and behaviour in children with the Phelan-McDermid syndrome.|
The aim of this project is to validate the hypothesis that intranasal insulin improves development and behaviour in children with Phelan-McDermid syndrome.
|- Main changes (audit trail)|
|- RECORD||18-dec-2012 - 4-feb-2013|