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

NCT01419249 | Completed Phase 4 Results

• 1 other identifier: EMR 200104_010
• Study Type: interventional
• Target: 458
• Locations: 9 countries
• Sites: 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).

Conditions

Idiopathic Growth Hormone Deficiency; Turner Syndrome

Keywords

Growth Hormone Deficiency; Turner syndrome; Pharmacogenetics; Recombinant Human Growth Hormone therapy

Outcome Measures

Primary Outcomes (3)

• Change From Baseline in Height at 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.

  Baseline and Year 1

• Change From Baseline in Height Standard Deviation Score (SDS) at 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.

  Baseline and Year 1

• Height Velocity Standard Deviation Score (SDS) at 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.

  Year 1

Secondary Outcomes (2)

• 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

  Year 1

• 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

  Year 1

Study Arms (1)

Retrospective cohort

OTHER

Other: Blood sampling

Interventions

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.

Retrospective cohort

Eligibility Criteria

• Age: Up to 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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

You may not qualify if:

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

Sponsors & Collaborators

• Merck KGaA, Darmstadt, Germany: lead
• Merck Serono S.A., Geneva: collaborator

Study Sites (31)

Hospital de Niños Ricardo Gutiérrez

Buenos Aires, Argentina

Location

Hospital de Pediatria Garrahan

Buenos Aires, Argentina

Location

Hospital de Niños de la Santisima Trinidad

Córdoba, Argentina

Location

University of Calgary - Alberta Children's Hospital

Calgary, Canada

Location

CHU Sainte Justine Montréal

Montreal, Canada

Location

Centre Hospitalier Universitaire de Sherbrooke - Fleurimont

Sherbrooke, Canada

Location

British Columbia Children's Hospital

Vancouver, Canada

Location

Fakultní nemocnice Brno

Brno, Czechia

Location

University Hospital Hradec Kralove

Hradec Králové, Czechia

Location

Faculty Hospital

Olomouc, Czechia

Location

University Hospital Praha Motol

Prague, Czechia

Location

Centre d'Endocrinologie Pédiatrique

Bordeaux, France

Location

CHU Bordeaux - Hopital pédiatrique Pellegrin

Bordeaux, France

Location

Hôpital Femme-Mère-Enfant

Bron, France

Location

University of Cologne Children's Hospital

Cologne, Germany

Location

University Children's Hospital

München, Germany

Location

University of Bari Aldo Moro

Bari, Italy

Location

Ospedale Microcitemico di Cagliari

Cagliari, Italy

Location

Centro di Endocrinologia e Diabetologia Pediatrica

Catania, Italy

Location

Istituto Giannina Gaslini - Clinica Pediatrica

Genova, Italy

Location

Hospital 12 de Octubre

Madrid, Spain

Location

Hospital Infantil Universitario Niño Jesús

Madrid, Spain

Location

Hospital Universitario Gregorio Maran

Madrid, Spain

Location

Hospital Clínico Universitario de Santiago de Compostela

Santiago de Compostela, Spain

Location

Hospital Miguel Servet

Zaragoza, Spain

Location

Queen Silvia Children's Hospital

Gothenburg, Sweden

Location

Faculty of Health Sciences, Linkping University

Linköping, Sweden

Location

Karolinska University Hospital Campus Solna

Stockholm, Sweden

Location

Birmingham Children's Hospital

Birmingham, United Kingdom

Location

Royal Manchester Children's Hospital

Manchester, United Kingdom

Location

Sheffield Children's Hospital

Sheffield, United Kingdom

Location

MeSH Terms

Conditions

Turner Syndrome; Dwarfism, Pituitary

Interventions

Blood Specimen Collection

Condition Hierarchy (Ancestors)

Gonadal Dysgenesis; Disorders of Sex Development; Urogenital Abnormalities; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Sex Chromosome Disorders of Sex Development; Male Urogenital Diseases; Heart Defects, Congenital; Cardiovascular Abnormalities; Cardiovascular Diseases; Heart Diseases; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Sex Chromosome Disorders; Chromosome Disorders; Genetic Diseases, Inborn; Gonadal Disorders; 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

Intervention Hierarchy (Ancestors)

Specimen Handling; Clinical Laboratory Techniques; Diagnostic Techniques and Procedures; Diagnosis; Punctures; Surgical Procedures, Operative; Investigative Techniques

Limitations and Caveats

No genetic markers were identified, therefore data for the secondary outcome measures was not analyzed.

Results Point of Contact

• Title: Merck KGaA Communication Center
• Organization: Merck Serono, a division of Merck KGaA

service@merckgroup.com

+49-6151-72-5200

Study Officials

• Gilles Della Corte

  Merck Serono S.A. , Geneva, Switzerland

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NON RANDOMIZED
• Masking: NONE
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 16, 2011
• First Posted: August 18, 2011
• Study Start: September 1, 2011
• Primary Completion: October 1, 2012
• Study Completion: October 1, 2012
• Last Updated: January 16, 2014
• Results First Posted: January 16, 2014

Record last verified: 2013-11

Locations

DE (2); SE (3); CZ (4); IT (4); AR (3); CA (4); FR (3); ES (5); GB (3)