Clinical trial 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
Official 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
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE4
Conditions: Idiopathic Growth Hormone Deficiency
Turner Syndrome
Keywords: Growth Hormone Deficiency
Turner syndrome
Pharmacogenetics
Recombinant Human Growth Hormone therapy
Lead sponsor: Merck KGaA, Darmstadt, Germany
Sponsor class: INDUSTRY
Collaborators: name: Merck Serono S.A., Geneva
First posted: 2011-08-18
Enrollment: 458
Sex: ALL
Maximum age (years): 18
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SINGLE_GROUP

Brief summary: 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).

Detailed description: 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.

Eligibility 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
* 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

Primary outcomes:
measure: Change From Baseline in Height at Year 1
timeFrame: Baseline and Year 1
measure: Change From Baseline in Height Standard Deviation Score (SDS) at Year 1
timeFrame: Baseline and Year 1
measure: Height Velocity Standard Deviation Score (SDS) at Year 1
timeFrame: Year 1
Secondary outcomes:
measure: 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
timeFrame: Year 1
measure: 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
timeFrame: Year 1
Other outcomes:
Locations:
city: Buenos Aires
country: Argentina
facility: Hospital de Niños Ricardo Gutiérrez
city: Buenos Aires
country: Argentina
facility: Hospital de Pediatria Garrahan
city: Cordoba
country: Argentina
facility: Hospital de Niños de la Santisima Trinidad
city: Calgary
country: Canada
facility: University of Calgary - Alberta Children's Hospital
city: Montréal
country: Canada
facility: CHU Sainte Justine Montréal
city: Sherbrooke
country: Canada
facility: Centre Hospitalier Universitaire de Sherbrooke - Fleurimont
city: Vancouver
country: Canada
facility: British Columbia Children's Hospital
city: Brno
country: Czech Republic
facility: Fakultní nemocnice Brno
city: Hradec Kralove
country: Czech Republic
facility: University Hospital Hradec Kralove
city: Olomouc
country: Czech Republic
facility: Faculty Hospital
city: Praha
country: Czech Republic
facility: University Hospital Praha Motol
city: Bordeaux
country: France
facility: Centre d'Endocrinologie Pédiatrique
city: Bordeaux
country: France
facility: CHU Bordeaux - Hopital pédiatrique Pellegrin
city: Bron
country: France
facility: Hôpital Femme-Mère-Enfant
city: Cologne
country: Germany
facility: University of Cologne Children's Hospital
city: München
country: Germany
facility: University Children's Hospital
city: Bari
country: Italy
facility: University of Bari Aldo Moro
city: Cagliari
country: Italy
facility: Ospedale Microcitemico di Cagliari
city: Catania
country: Italy
facility: Centro di Endocrinologia e Diabetologia Pediatrica
city: Genova
country: Italy
facility: Istituto Giannina Gaslini - Clinica Pediatrica
city: Madrid
country: Spain
facility: Hospital 12 de Octubre
city: Madrid
country: Spain
facility: Hospital Infantil Universitario Niño Jesús
city: Madrid
country: Spain
facility: Hospital Universitario Gregorio Maran
city: Santiago de Compostela
country: Spain
facility: Hospital Clínico Universitario de Santiago de Compostela
city: Zaragoza
country: Spain
facility: Hospital Miguel Servet
city: Göteborg
country: Sweden
facility: Queen Silvia Children's Hospital
city: Linköping
country: Sweden
facility: Faculty of Health Sciences, Linkping University
city: Stockholm
country: Sweden
facility: Karolinska University Hospital Campus Solna
city: Birmingham
country: United Kingdom
facility: Birmingham Children's Hospital
city: Manchester
country: United Kingdom
facility: Royal Manchester Children's Hospital
city: Sheffield
country: United Kingdom
facility: Sheffield Children's Hospital
Study officials:
affiliation: Merck Serono S.A. , Geneva, Switzerland
name: Gilles Della Corte
role: STUDY_DIRECTOR
References: