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NCT Number: NCT01419249

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 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).

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Key information

Conditions

Idiopathic Growth Hormone Deficiency Bone Diseases Bone Diseases, Developmental Bone Diseases, Endocrine Brain Diseases Cardiovascular Abnormalities Cardiovascular Diseases Central Nervous System Diseases Chromosome Disorders Congenital Abnormalities Congenital, Hereditary, and Neonatal Diseases and Abnormalities Disorders of Sex Development Dwarfism Dwarfism, Pituitary Endocrine System Diseases Female Urogenital Diseases Female Urogenital Diseases and Pregnancy Complications Genetic Diseases, Inborn Gonadal Disorders Gonadal Dysgenesis Heart Defects, Congenital Heart Diseases Hypopituitarism Hypothalamic Diseases Male Urogenital Diseases Musculoskeletal Diseases Nervous System Diseases Pituitary Diseases Sex Chromosome Disorders Sex Chromosome Disorders of Sex Development Turner Syndrome Urogenital Abnormalities Urogenital Diseases

Age range

Up to 18 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 4

Primary location

Hospital de Niños Ricardo Gutiérrez, Buenos Aires, Argentina

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About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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
  • Other protocol defined inclusion criteria could apply

Exclusion criteria

  • Acquired growth hormone deficiency (GHD)
  • Any drug or disease that could affect growth during the first year of r-hGH treatment
  • Other protocol defined exclusion criteria could apply

Treatment and study plan

Blood sampling

Other

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.

Primary outcomes

  1. Change From Baseline in Height at Year 1

    Time frame: Baseline and 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.

  2. Change From Baseline in Height Standard Deviation Score (SDS) at Year 1

    Time frame: Baseline and 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.

  3. Height Velocity Standard Deviation Score (SDS) at Year 1

    Time frame: 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.

Secondary outcomes

  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

    Time frame: Year 1

    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.

  2. 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

    Time frame: Year 1

    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.

Sponsors and collaborators

Lead sponsor

Merck KGaA, Darmstadt, Germany

Industry

Collaborators

  • Merck Serono S.A., Geneva

Registry information

Important dates

Study start
2011
Primary completion
2012
Study completion
2012
First posted
Aug 18, 2011
Registry last updated
Jan 16, 2014

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

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This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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