PGx validation study in GHD and Turner Syndrome pre-pubertal children

• Research type

  Research Study

• Full title

  First year growth response associated genetic markers validation Phase IV open-label study in Growth Hormone Deficient and Turner Syndrome pre-pubertal children : the PREDICT Pharmacogenetics Validation Study

• IRAS ID

  80057

• Sponsor organisation

  Merck Serono S.A.

• Eudract number

  2011-000460-10

• Research summary

  PREDICT Validation is a validation pharmacogenetic trial. ??Validation? means providing proof and confirmation. This research study involves pharmacogenetic research (the word ??pharmaco? refers to drugs, and the word ??genetic? means looking at genes). So the pharmacogenetic research looks at the genes and tries to find out why some people have better results from treatment than others.The purpose of this study is to confirm that some genes can be used to predict whether a patient diagnosed with Idiopathic Growth Hormone Deficiency (IGHD) or Turner Syndrome (TS) will respond well or poorly to a treatment with recombinant human growth hormone (rhGH).To do this, a sample of blood will be analysed.This trial is the third step of a program called the PREDICT Programme being conducted by Merck Serono. The aim of this programme is to predict response to rhGH treatment using existing normal variations in certain genes, are called polymorphisms.During the first steps of the programme children who have not yet begun puberty and who have Idiopathic Growth Hormone Deficiency or Turner Syndrome were studied. 48 genetic associations were found to be linked with the first year growth response to growth hormone treatment. In other words the presence or absence of such a polymorphism is associated either with a low or with a high response to growth hormone treatment. The aim of the trial is to confirm that when applied to an independent series of patient treated with growth hormone, some of these 48 genetic associations are indeed predictive of first year growth response to rhGH. All the genes studied were genes related to growth.

• REC name

  North West - Greater Manchester Central Research Ethics Committee

• REC reference

  11/NW/0378

• Date of REC Opinion

  3 Aug 2011

• REC opinion

  Further Information Favourable Opinion