Study of safety of Saizen® in children born with serious intra-uterine growth retardation
(IUGR) treated to final height. An open, phase III study involving 17 centers in France.
The study enrolled children who have completed 3 or 2 years of treatment and at least one
year of post treatment observation in the Sponsor Studies GF 4001 (Safety and Efficacy of
Saizen in the Treatment of Young Children Born with Severe IUGR) or GF 6283 (Effect of
Intermittent versus Continuous Saizen Therapy in Young Children Born with Severe IUGR),
respectively.
Detailed description: Serious IUGR is a syndrome characterized by low birth length and weight
for gestational age (less than 10 percentile). The secretion of growth hormone in response to
provocative stimuli (e.g. arginine, insulin) is normal in these children. Apart from low
birth weight, children born with IUGR may have minor or major malformations.
A catch-up period with a supraphysiological growth velocity generally occurs during the first
6 to 24 months of life in 80 to 90 percent (%) of these children. This generally allows them
to reach normal height. That means that conversely, approximately 10 to 20% of children do
maintain a statural handicap. Puberty occurs at a normal age and the retardation in bone
maturation present during the first years of life disappears very quickly. This leads to
short adult stature in subjects who have not shown spontaneous catch-up during the first
years of life. A safe and effective means of promoting growth without accelerating the timing
or tempo of puberty would therefore be desirable.