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

This study has been completed.
Sponsor:
Collaborator:
Merck Serono S.A., Geneva
Information provided by (Responsible Party):
Merck KGaA
ClinicalTrials.gov Identifier:
NCT01419249
First received: August 16, 2011
Last updated: November 26, 2013
Last verified: November 2013
  Purpose

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).


Condition Intervention Phase
Idiopathic Growth Hormone Deficiency
Turner Syndrome
Other: Blood sampling
Phase 4

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: Open Label
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

Resource links provided by NLM:


Further study details as provided by Merck KGaA:

Primary Outcome Measures:
  • Change From Baseline in Height at Year 1 [ Time Frame: Baseline and Year 1 ] [ Designated as safety issue: No ]
    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.

  • Change From Baseline in Height Standard Deviation Score (SDS) at Year 1 [ Time Frame: Baseline and Year 1 ] [ Designated as safety issue: No ]
    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.

  • Height Velocity Standard Deviation Score (SDS) at Year 1 [ Time Frame: Year 1 ] [ Designated as safety issue: No ]
    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 Outcome Measures:
  • 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 ] [ Designated as safety issue: No ]
    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.

  • 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 ] [ Designated as safety issue: No ]
    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.


Enrollment: 458
Study Start Date: September 2011
Study Completion Date: October 2012
Primary Completion Date: October 2012 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Retrospective cohort Other: 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.

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

Ages Eligible for Study:   up to 18 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01419249

Locations
Argentina
Hospital de Pediatria Garrahan
Buenos Aires, Argentina
Hospital de Niños Ricardo Gutiérrez
Buenos Aires, Argentina
Hospital de Niños de la Santisima Trinidad
Cordoba, Argentina
Canada
University of Calgary - Alberta Children's Hospital
Calgary, Canada
CHU Sainte Justine Montréal
Montréal, Canada
Centre Hospitalier Universitaire de Sherbrooke - Fleurimont
Sherbrooke, Canada
British Columbia Children's Hospital
Vancouver, Canada
Czech Republic
Fakultní nemocnice Brno
Brno, Czech Republic
University Hospital Hradec Kralove
Hradec Kralove, Czech Republic
Faculty Hospital
Olomouc, Czech Republic
University Hospital Praha Motol
Praha, Czech Republic
France
CHU Bordeaux - Hopital pédiatrique Pellegrin
Bordeaux, France
Centre d'Endocrinologie Pédiatrique
Bordeaux, France
Hôpital Femme-Mère-Enfant
Bron, France
Germany
University of Cologne Children's Hospital
Cologne, Germany
University Children's Hospital
München, Germany
Italy
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
Spain
Hospital Universitario Gregorio Maran
Madrid, Spain
Hospital Infantil Universitario Niño Jesús
Madrid, Spain
Hospital 12 de Octubre
Madrid, Spain
Hospital Clínico Universitario de Santiago de Compostela
Santiago de Compostela, Spain
Hospital Miguel Servet
Zaragoza, Spain
Sweden
Queen Silvia Children's Hospital
Göteborg, Sweden
Faculty of Health Sciences, Linkping University
Linköping, Sweden
Karolinska University Hospital Campus Solna
Stockholm, Sweden
United Kingdom
Birmingham Children's Hospital
Birmingham, United Kingdom
Royal Manchester Children's Hospital
Manchester, United Kingdom
Sheffield Children's Hospital
Sheffield, United Kingdom
Sponsors and Collaborators
Merck KGaA
Merck Serono S.A., Geneva
Investigators
Study Director: Gilles Della Corte Merck Serono S.A. , Geneva, Switzerland
  More Information

No publications provided

Responsible Party: Merck KGaA
ClinicalTrials.gov Identifier: NCT01419249     History of Changes
Other Study ID Numbers: EMR 200104_010
Study First Received: August 16, 2011
Results First Received: November 26, 2013
Last Updated: November 26, 2013
Health Authority: Argentina: Administracion Nacional de Medicamentos, Alimentos y Tecnologia Medica
Canada: Ethics Review Committee
Czech Republic: State Institute for Drug Control
France: Afssaps - Agence française de sécurité sanitaire des produits de santé (Saint-Denis)
France: Institutional Ethical Committee
Germany: Ethics Commission
Italy: Ethics Committee
Spain: Comité Ético de Investigación Clínica
Spain: Ministry of Health
Sweden: Regional Ethical Review Board
Sweden: Data Inspection Agency
Sweden: Biobank Center of Västra Götaland and Sydöstra sjukvardsregionen
United Kingdom: Medicines and Healthcare Products Regulatory Agency
United Kingdom: National Health Service
United Kingdom: Research Ethics Committee

Keywords provided by Merck KGaA:
Growth Hormone Deficiency
Turner syndrome
Pharmacogenetics
Recombinant Human Growth Hormone therapy

Additional relevant MeSH terms:
Turner Syndrome
Gonadal Dysgenesis
Primary Ovarian Insufficiency
Dwarfism, Pituitary
Endocrine System Diseases
Dwarfism
Bone Diseases, Developmental
Bone Diseases
Musculoskeletal Diseases
Bone Diseases, Endocrine
Hypopituitarism
Pituitary Diseases
Hypothalamic Diseases
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Disorders of Sex Development
Urogenital Abnormalities
Sex Chromosome Disorders of Sex Development
Heart Defects, Congenital
Cardiovascular Abnormalities
Cardiovascular Diseases
Heart Diseases
Congenital Abnormalities
Sex Chromosome Disorders
Chromosome Disorders
Genetic Diseases, Inborn
Gonadal Disorders
Ovarian Diseases
Adnexal Diseases

ClinicalTrials.gov processed this record on July 31, 2014