Clinical trial NCT01082354: A Pharmaco-epidemiological Study of Short Patients Born Small for Gestational Age (SGA), Treated With Saizen® (Recombinant Somatropin)
Official title: Non Interventional Study on the Follow-up, by Specialists, of Short Children Born Small for Gestational Age and Treated With SAIZEN®- SEPAGE Study
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Infant, Small for Gestational Age
Keywords: Infant, Low birth weight
Infant, Low birth height
Infant, Small for Gestational Age
Lead sponsor: Merck KGaA, Darmstadt, Germany
Sponsor class: INDUSTRY
Collaborators: name: Merck Serono S.A.S, France
First posted: 2010-03-08
Enrollment: 150
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: 48 hospital pediatric and endocrinology centers or in mixed private and hospital practice will enroll 150 children

Brief summary: This one-arm, multi-centric, non-randomized, observational, open label, prospective study was designed to study short subjects born SGA, by pediatricians, endocrinologists or physicians in private, mixed private and hospital practice treated with Saizen.

Detailed description: The term "small for gestational age" (SGA) is a pathological medical condition known since about two decades supported by two consensus conferences of 2001. It inhibits the height and/or weight of the child at birth without prejudging the mechanisms that alter these. This study will be carried out by hospital pediatricians, endocrinologists or physicians in private, mixed private and hospital practice in metropolitan France. Subjects will be monitored by doctors in accordance with the normal scope of follow-up of pathology until their final growth and until they reach adult height and/or until the treatment is discontinued. The participating doctors will include all the short SGA subjects coming for consultation or the follow-up (for less than 12 months) of their treatment by Saizen in this study. The inclusions will take place over a period of 21 months.

OBJECTIVES

Primary objective:

* To describe the characteristics of short children born SGA (SGA subjects) treated with Saizen

Secondary objectives:

* To describe the terms and conditions for the prescription of Saizen in terms of dosage, duration of treatment, reason for discontinuation and compliance
* To describe the effects of Saizen on growth and final height
* To describe the tolerance of Saizen. For each subject and during each visit, the participating doctor will complete an electronic data collection form.

Eligibility criteria: Inclusion Criteria:

* Short children born SGA requiring treatment with Saizen, new to growth hormone treatment or being treated with growth hormones for less than 12 months of inclusion
* Subjects whose parents have given their written consent for participation in this study

Exclusion Criteria:

* Contra-indication in the treatment with Saizen

Primary outcomes:
measure: Auxological characteristics (height and weight, head circumference, growth curve used, bone age) and Clinical examination (cardiac frequency, systolic blood pressure, diastolic blood pressure, Tanner pubertal stage)
timeFrame: Inclusion visit to follow-up visits at 3, 6 and 12 month and annual until the adult height is reached and or the final discontinuation of the treatment
measure: Saizen prescription and compliance to treatment
timeFrame: Inclusion visit to follow-up visits at 3, 6 and 12 month and annual until the adult height is reached and or the final discontinuation of the treatment
measure: Temporary discontinuation of treatment with Saizen if > 1 week (number, duration, person responsible for the discontinuation, reason, dose upon resumption of treatment) and final discontinuation of treatment with Saizen (date and reason)
timeFrame: Inclusion visit to follow-up visits at 3, 6 and 12 month and annual until the adult height is reached and or the final discontinuation of the treatment
measure: Biochemical and endocrinological parameters
timeFrame: Inclusion visit to follow-up visits at 3, 6 and 12 month and annual until the adult height is reached and or the final discontinuation of the treatment
Secondary outcomes:
Other outcomes:
Locations:
city: Aix en Provence
country: France
facility: Merck Serono Investigational Site
city: Annemasse
country: France
facility: Merck Serono Investigational Site
city: Belfort
country: France
facility: Merck Serono Investigational Site
city: Besançon
country: France
facility: Merck Serono Investigational Site
city: Bordeaux
country: France
facility: Merck Serono Investigational Site
city: Brest
country: France
facility: Merck Serono Investigational Site
city: Caen
country: France
facility: Merck Serono Investigational Site
city: Chambery
country: France
facility: Merck Serono Investigational Site
city: Clermont-Ferrand
country: France
facility: Merck Serono Investigational Site
city: Dijon
country: France
facility: Merck Serono Investigational Site
city: Frejus
country: France
facility: Merck Serono Investigational Site
city: Grenoble
country: France
facility: Merck Serono Investigational Site
city: Hyères
country: France
facility: Merck Serono Investigational Site
city: La Rochelle
country: France
facility: Merck Serono Investigational Site
city: Laon
country: France
facility: Merck Serono Investigational Site
city: Le Havre
country: France
facility: Merck Serono Investigational Site
city: Le Mans
country: France
facility: Merck Serono Investigational Site
city: Lille
country: France
facility: Merck Serono Investigational Site
city: Limoges
country: France
facility: Merck Serono Investigational Site
city: Lisieux
country: France
facility: Merck Serono Investigational Site
city: Ludres
country: France
facility: Merck Serono Investigational Site
city: Lyon-Bron
country: France
facility: Merck Serono Investigational Site
city: Macon
country: France
facility: Merck Serono Investigational Site
city: Marseille
country: France
facility: Merck Serono Investigational Site
city: Montbeliard
country: France
facility: Merck Serono Investigational Site
city: Montluçon
country: France
facility: Merck Serono Investigational Site
city: Montpellier
country: France
facility: Merck Serono Investigational Site
city: Nantes
country: France
facility: Merck Serono Investigational Site
city: Nice
country: France
facility: Merck Serono Investigational Site
city: Orléans
country: France
facility: Merck Serono Investigational Site
city: Paris
country: France
facility: Merck Serono Investigational Site
city: Pringy
country: France
facility: Merck Serono Investigational Site
city: Puyricard
country: France
facility: Merck Serono Investigational Site
city: Reims
country: France
facility: Merck Serono Investigational Site
city: Saint Priest en Jarez
country: France
facility: Merck Serono Investigational Site
city: Strasbourg
country: France
facility: Merck Serono Investigational Site
city: Toulon
country: France
facility: Merck Serono Investigational Site
city: Toulouse
country: France
facility: Merck Serono Investigational Site
city: Tours
country: France
facility: Merck Serono Investigational Site
city: Vesoul
country: France
facility: Merck Serono Investigational Site
Study officials:
affiliation: Service d'Endocrinologie Pédiatrique & Diabétologie Infantile, Hôpital Femme Mère Enfant, Lyon-Bron, France
name: Pierre Chatelain, MD, Professor
role: STUDY_CHAIR
References: