Genotropin Treatment In Very Young Children Born Small For Gestational Age

NCT00627523 | Completed Phase 3 Results

• 3 other identifiers: A62812872007-003949-32SGA
• Study Type: interventional
• Target: 43
• Locations: 9 countries
• Sites: 17

Brief Summary

To demonstrate the effect on height and psychomotor development of Growth Hormone treatment in very young children starting at an age of 2 years.

Conditions

Infant, Small for Gestational Age

Outcome Measures

Primary Outcomes (1)

• Change From Baseline in Height Standard Deviation Score (SDS) at Month 24.

  Height SDS was calculated at the relevant visit by means of the following formula: Height SDS = (participant height) - (normal height)/normal height standard deviation. Where participant height refers to the participant's height at the relevant visit, and normal height and the normal height standard deviation equals the population mean and standard deviation values for participants of a similar age and gender. The change from Baseline value for height SDS was calculated as the difference between the parameter values at a specific visit, and the Baseline parameter values. The scores were centred around zero. Negative score indicated a participant was smaller for their age/gender.

  Baseline and Month 24

Secondary Outcomes (9)

• Change From Baseline in Growth Velocity SDS at Month 24.

  Baseline and Month 24

• Change From Baseline in Height SDS at Month 12.

  Baseline and Month 12

• Change From Baseline in Growth Velocity SDS at Month 12.

  Baseline and Month 12

• Change From Baseline in Mental Development Using the Mental Development Index (MDI) of Bayley Scale at Month 12.

  Baseline and Month 12

• Change From Baseline in Psychomotor Development Using the Psychomotor Development Index (PDI) of Bayley Scale at Month 12.

  Baseline and Month 12

Study Arms (2)

Active

EXPERIMENTAL

The active treatment arm

Drug: Genotropin (PN-180,307) Somatropin

Control

EXPERIMENTAL

Control

Drug: Control-no treatment

Interventions

Genotropin (PN-180,307) Somatropin DRUG

Injectable Genotropin

Active

Control-no treatment DRUG

Control-no treatment

Control

Eligibility Criteria

• Age: 19 Months - 29 Months
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Caucasian male or female subjects aged between 19-29 months at Screening Visit 1.
• Born SGA (birth length and/or weight \<-2 SD for gestational age, using country-specific standards).
• Height below -2.5 SD at screening (19-29 months of age). At least one measurement of length between 12 and 18 months of age. Normal karyotype in girls to exclude Turners syndrome.

You may not qualify if:

• Severe Intra-Uterine Growth Retardation (IUGR) (birth length below - 4 SD for gestational age), if associated with dysmorphic features.
• Severe familial short stature defined as: Father's height below 155 cm or mother's height below 145 cm.
• Defined neurological defects and/or severe neurodevelopmental delay.

Sponsors & Collaborators

• Pfizer: lead

Study Sites (17)

Universitair Ziekenhuis Brussel/Pediatrie

Brussels, 1090, Belgium

Location

Universitair Ziekenhuis Antwerpen / Pediatrie

Edegem, 2650, Belgium

Location

Fakultni nemocnice Olomouc

Olomouc, 775 20, Czechia

Location

Fakultni nemocnice Ostrava, Detska klinika

Ostrava-Poruba, 708 52, Czechia

Location

Fakultni nemocnice v Motole

Prague, 150 06, Czechia

Location

CHU Toulouse, Hôpital des Enfants, Service Endocrinologie

Toulouse, 31059, France

Location

Universitaetsklinikum Erlangen, Kinder- und Jugendklinik

Erlangen, 91054, Germany

Location

Centro di Endocrinologia Pediatrica, Dipartimento di Pediatria

Catania, 95125, Italy

Location

Policlinico Universitario, Istituto di Clinica Pediatrica

Messina, 98100, Italy

Location

Dipartimento Materno Infantile, UO di Pediatria e Neonatologia

Milan, 20132, Italy

Location

Dipartimento di Medicina Pediatrica, UO di Endocrinologia e Diabetologia

Roma, 00165, Italy

Location

Sophia Children's Hospital

Rotterdam, 3015 GJ, Netherlands

Location

Consorci Hospitalari Parc Tauli

Sabadell, Barcelona, 08208, Spain

Location

Hospital Virgen Del Camino

Pamplona, Navarre, 31008, Spain

Location

Hospital Miguel Servet

Zaragoza, Zaragoza, 50009, Spain

Location

Drottning Silvias Barn och Ungdomssjukhus

Gothenburg, Sweden

Location

Inselspital

Ch-3010 Bern, Switzerland

Location

Related Publications (1)

• De Schepper J, Vanderfaeillie J, Mullis PE, Rooman R, Robertson A, Dilleen M, Gomez R, Wollmann HA. A 2-year multicentre, open-label, randomized, controlled study of growth hormone (Genotropin(R)) treatment in very young children born small for gestational age: Early Growth and Neurodevelopment (EGN) Study. Clin Endocrinol (Oxf). 2016 Mar;84(3):353-60. doi: 10.1111/cen.12968. Epub 2015 Nov 25.

  PMID: 26501737 DERIVED

Related Links

• To obtain contact information for a study center near you, click here.

MeSH Terms

Interventions

Human Growth Hormone

Intervention Hierarchy (Ancestors)

Growth Hormone; Pituitary Hormones, Anterior; Pituitary Hormones; Peptide Hormones; Hormones; Hormones, Hormone Substitutes, and Hormone Antagonists; Peptides; Amino Acids, Peptides, and Proteins

Results Point of Contact

• Title: Pfizer ClinicalTrials.gov Call Center
• Organization: Pfizer, Inc.

ClinicalTrials.gov_Inquiries@pfizer.com

1-800-718-1021

Study Officials

• Pfizer CT.gov Call Center

  Pfizer

  STUDY DIRECTOR

Publication Agreements

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

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 22, 2008
• First Posted: March 3, 2008
• Study Start: February 1, 2008
• Primary Completion: December 1, 2013
• Study Completion: December 1, 2013
• Last Updated: November 4, 2014
• Results First Posted: September 18, 2014

Record last verified: 2014-10

Locations

IT (4); DE (1); BE (2); CZ (3); ES (3); NL (1); SE (1); CH (1); FR (1)