A Pharmaco-epidemiological Study of Short Patients Born Small for Gestational Age (SGA), Treated With Saizen® (Recombinant Somatropin)

Merck KGaA, Darmstadt, Germany150 enrolled

Overview

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.

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.

Study Type

OBSERVATIONAL

Enrollment

150

Conditions

Infant, Small for Gestational Age

Interventions

recombinant somatropinDRUG

as per standard practice

Eligibility

Sex: ALL

Medical Language ↔ Plain English

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

Locations (40)

Outcomes

Primary Outcomes

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)

Time frame: 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

Saizen prescription and compliance to treatment

Time frame: 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

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)

Time frame: 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

Biochemical and endocrinological parameters

* Tolerance * Concomitant treatments

Time frame: 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

Data from ClinicalTrials.gov

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