PREDICT Validation is a validation pharmacogenetic trial. The purpose of this study is to confirm that some genes can be used to predict how well a subject diagnosed with idiopathic growth hormone deficiency (IGHD) or turner syndrome (TS) will respond to a treatment with recombinant human growth hormone (r-hGH).
This study is an open-label, interventional, retrospective, multicenter, international study, single-arm, non-randomized, and non-controlled study. The subject's trial participation includes 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 subject is indicated the pediatric population, therefore most of the subjects 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.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Masking
NONE
Enrollment
458
Subjects with pre-established diagnosis of IGHD and TS and were treated with r-hGH therapy for 1 year, will be observed in this retrospective cohort study wherein blood sampling will performed for genotyping of the various genetic markers along with collection of retrospective data relative to the r-hGH treatment.
Hospital de Niños Ricardo Gutiérrez
Buenos Aires, Argentina
Hospital de Pediatria Garrahan
Buenos Aires, Argentina
Hospital de Niños de la Santisima Trinidad
Córdoba, Argentina
University of Calgary - Alberta Children's Hospital
Calgary, Canada
CHU Sainte Justine Montréal
Montreal, Canada
Change From Baseline in Height at Year 1
Change from baseline in height at year 1 was one of the growth parameter to assess the first year growth response to r-hGH treatment.
Time frame: Baseline and Year 1
Change From Baseline in Height Standard Deviation Score (SDS) at Year 1
Height SDS was calculated as height minus reference mean height divided by standard deviation of the reference population. Height SDS reflects the height relative to a reference population of the same age and gender. Change from baseline in height SDS at Year 1 was one of the growth parameter to assess the first year growth response to r-hGH treatment.
Time frame: Baseline and Year 1
Height Velocity Standard Deviation Score (SDS) at Year 1
Height velocity SDS was calculated as height velocity minus reference mean height velocity divided by standard deviation of the reference population. Height velocity SDS reflects the height velocity relative to a reference population of the same age and gender. Height velocity SDS at Year 1 was one of the growth parameter to assess the first year growth response to r-hGH treatment.
Time frame: Year 1
Evaluation of the Contribution of Validated Genetic Markers to the Amplitude of First Year Growth Response to r-hGH Therapy in IGHD Children Using Growth Hormone Deficiency Kabi-Pharmacia International Growth Study (GHD KIGS) Predictive Model
GHD KIGS predictive model includes various clinical, auxological and biological markers which are as follows: maximum growth hormone (GH) response to provocation test; age at onset of therapy; birth weight SDS; average GH dose received during the first year of r-hGH therapy; height SDS at start of therapy; the difference between the pre-treatment height SDS of the subject and the mid parental height SDS; and weight SDS at start of therapy.
Time frame: Year 1
Evaluation of the Contribution of Validated Genetic Markers to the Amplitude of First Year Growth Response to r-hGH Therapy in TS Girls Using Turner Syndrome Kabi-Pharmacia International Growth Study (TS KIGS) Predictive Model
TS KIGS predictive model includes various clinical, auxological and biological markers which are as follows: maximum GH response to provocation test; age at onset of therapy; birth weight SDS; average GH dose received during the first year of r-hGH therapy; height SDS at start of therapy; the difference between the pre-treatment height SDS of the subject and the mid parental height SDS; and weight SDS at start of therapy.
Time frame: Year 1
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Centre Hospitalier Universitaire de Sherbrooke - Fleurimont
Sherbrooke, Canada
British Columbia Children's Hospital
Vancouver, Canada
Fakultní nemocnice Brno
Brno, Czechia
University Hospital Hradec Kralove
Hradec Králové, Czechia
Faculty Hospital
Olomouc, Czechia
...and 21 more locations