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

NCT ID: NCT01419249

Last Updated: 2014-01-16

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE4
• Total Enrollment: 458 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2011-09-30
• Study Completion Date: 2012-10-31

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.

Conditions

Idiopathic Growth Hormone Deficiency; Turner Syndrome

Study Design

• Allocation Method: NON_RANDOMIZED
• Intervention Model: SINGLE_GROUP
• Blinding Strategy: NONE

Study Groups

Retrospective cohort

Group Type: OTHER

Blood sampling

Intervention Type: 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.

Interventions

Blood sampling

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.

Intervention Type: OTHER

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

Exclusion Criteria

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

• Maximum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Merck Serono S.A., Geneva

INDUSTRY

Sponsor Role: collaborator

Merck KGaA, Darmstadt, Germany

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Gilles Della Corte

Role: STUDY_DIRECTOR

Merck Serono S.A. , Geneva, Switzerland

Locations

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

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

University Hospital Praha Motol

Prague, , Czechia

Centre d'Endocrinologie Pédiatrique

Bordeaux, , France

CHU Bordeaux - Hopital pédiatrique Pellegrin

Bordeaux, , France

Hôpital Femme-Mère-Enfant

Bron, , France

University of Cologne Children's Hospital

Cologne, , Germany

University Children's Hospital

München, , Germany

University of Bari Aldo Moro

Bari, , Italy

Ospedale Microcitemico di Cagliari

Cagliari, , Italy

Centro di Endocrinologia e Diabetologia Pediatrica

Catania, , Italy

Istituto Giannina Gaslini - Clinica Pediatrica

Genova, , Italy

Hospital 12 de Octubre

Madrid, , Spain

Hospital Infantil Universitario Niño Jesús

Madrid, , Spain

Hospital Universitario Gregorio Maran

Madrid, , Spain

Hospital Clínico Universitario de Santiago de Compostela

Santiago de Compostela, , Spain

Hospital Miguel Servet

Zaragoza, , Spain

Queen Silvia Children's Hospital

Gothenburg, , Sweden

Faculty of Health Sciences, Linkping University

Linköping, , Sweden

Karolinska University Hospital Campus Solna

Stockholm, , Sweden

Birmingham Children's Hospital

Birmingham, , United Kingdom

Royal Manchester Children's Hospital

Manchester, , United Kingdom

Sheffield Children's Hospital

Sheffield, , United Kingdom

Countries

Argentina; Canada; Czechia; France; Germany; Italy; Spain; Sweden; United Kingdom

Other Identifiers

EMR 200104_010

Identifier Type: -

Identifier Source: org_study_id