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

• The primary objective of the trial is to confirm that at least one of the genetic markers associated to the amplitude of first year growth response to r-hGH treatment identified in PREDICT LTFU Study (28614) is replicated in an independent population. [ Time Frame: 1 year ] [ Designated as safety issue: No ]

  The list of genetic markers associated to the first year growth response to r-hGH measured by the 3 following growth parameters:

  • Change from baseline to year 1 in Height (cm)
  • Change from baseline to year 1 in, Height SDS
  • HV SDS at 1 year
  
  

• To evaluate the contribution of validated genetic markers to the amplitude of first year growth response to r-hGH therapy in IGHD children using a model derived from the GHD KIGS predictive model of the first year growth response to r hGH [ Time Frame: 1 year ] [ Designated as safety issue: No ]
  The first year growth predictive value of the models including the validated genetic markers on HV (cm/year) at 1 year in IGHD children treated with r-hGH combined to well-established clinical, auxological and biological markers derived from the KIGS GHD predictive model of the first year growth response to r-hGH therapy
  
  
• To evaluate the contribution of validated genetic markers to the amplitude of first year growth response to r-hGH therapy in TS girls using a model derived from the TS KIGS predictive mode of the first year growth response to r-hGH [ Time Frame: 1 year ] [ Designated as safety issue: No ]
  The first year growth predictive value of the models including the validated genetic markers on HV at 1 year in TS girls treated with r-hGH combined to well established clinical and auxological markers derived from the KIGS TS predictive model of the first year growth response to r-hGH therapy
  
  

This study is an open-label, interventional, retrospective, multicentre, international study, single-arm, non-randomized, and non-controlled. The subject's trial participation will include a single visit. During the visit, subjects who give consent to participate in the trial will undergo blood sampling for genetic markers testing and retrospective data will be collected relative to the first year of the subject's r-hGH treatment.

The r-hGH treatment followed by the subjects are indicated for pediatric population, therefore most of the patients included into the trial will be below 18 years old.

This study is a non-Investigational Medicinal Product (IMP) trial therefore no drug product data is provided.