Validation study for genetic markers for growth hormone treatment in growth hormone deficient and turner syndrome patients

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 - PREDICT PGx Validation Study

Status

Active, not recruiting

Phases

Unknown

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2011-000460-10-GB

Enrollment

395

Registered

2011-05-17

Start date

2011-08-03

Completion date

Unknown

Last updated

2012-12-03

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

idiopathic growth hormone deficienty Turner Syndrome MedDRA version: 13.1 Level: PT Classification code 10056438 Term: Growth hormone deficiency System Organ Class: 10014698 - Endocrine disorders MedDRA version: 13.1 Level: PT Classification code 10045181 Term: Turner's syndrome System Organ Class: 10010331 - Congenital, familial and genetic disorders

Interventions

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Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: Pre-established diagnosis of IGHD or TS based on classical criteria with at least 1 year of r-hGH therapy and with Tanner stage 1 at treatment start Retrospective availability of a complete set of clinical, auxological and biological parameters necessary for building the predictive model Are the trial subjects under 18? yes Number of subjects for this age range: 350 F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 45 F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range

Exclusion criteria

Exclusion criteria: Acquired GHD Any drug or disease that could affect growth

Design outcomes

Primary

Measure	Time frame
Main Objective: To confirm that at least one of the genetic markers associated to the amplitude of first year growth response to recombinant human growth hormone (r-hGH) treatment identified in PREDICT Long- Term Follow-Up (LTFU) Study (28614) is replicated in an independent population of this study of prepubertal children with either idiopathic growth hormone deficiency (IGHD) or Turner Syndrome (TS).;Secondary Objective: To evaluate the contribution of validated genetic markers related to the amplitude of first year growth in IGHD children in response to r-hGH therapy using a model derived from the growth hormone deficiency (GHD) Kabi-Pharmacia International Growth Study (KIGS) predictive model of the first year growth response to r-hGH. To evaluate the contribution of validated genetic markers related to the amplitude of first year growth in TS girls in response to rhGH therapy in TS girls using a model derived from the TS KIGS predictive model of the first year growth response to r-hGH.;Primary end point(s): 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 standard deviation score (SDS) Height Velocity (HV) (cm/year) SDS at 1 year;Timepoint(s) of evaluation of this end point: at time of final report	—

Measure

Time frame

Main Objective: To confirm that at least one of the genetic markers associated to the amplitude of first year growth response to recombinant human growth hormone (r-hGH) treatment identified in PREDICT Long- Term Follow-Up (LTFU) Study (28614) is replicated in an independent population of this study of prepubertal children with either idiopathic growth hormone deficiency (IGHD) or Turner Syndrome (TS).;Secondary Objective: To evaluate the contribution of validated genetic markers related to the amplitude of first year growth in IGHD children in response to r-hGH therapy using a model derived from the growth hormone deficiency (GHD) Kabi-Pharmacia International Growth Study (KIGS) predictive model of the first year growth response to r-hGH. To evaluate the contribution of validated genetic markers related to the amplitude of first year growth in TS girls in response to rhGH therapy in TS girls using a model derived from the TS KIGS predictive model of the first year growth response to r-hGH.;Primary end point(s): 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 standard deviation score (SDS) Height Velocity (HV) (cm/year) SDS at 1 year;Timepoint(s) of evaluation of this end point: at time of final report

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Secondary

Measure	Time frame
Secondary end point(s): The first year growth predictive value of the models including the validated genetic markers on HV (cm/year) at 1 year in IGHD children and in TS girls treated with r-hGH combined to wellestablished clinical, auxological and biological markers derived from the KIGS GHD predictive model of the first year growth response to r-hGH therapy;Timepoint(s) of evaluation of this end point: at time of final report	—

Measure

Time frame

Secondary end point(s): The first year growth predictive value of the models including the validated genetic markers on HV (cm/year) at 1 year in IGHD children and in TS girls treated with r-hGH combined to wellestablished clinical, auxological and biological markers derived from the KIGS GHD predictive model of the first year growth response to r-hGH therapy;Timepoint(s) of evaluation of this end point: at time of final report

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Countries

Canada, Czech Republic, Italy, Russian Federation, Spain, United Kingdom

Contacts

Public ContactCommunication Center Merck KGaA

Merck KGaA

service@merck.de496151725200

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026