Saizen in Intra-uterine Growth Retardation

Condition(s) targeted: Children Born With Serious Intra-uterine Growth Retardation

Intervention: Saizen® A (Drug); Saizen® B (Drug); Observation only (Other)

Phase: Phase 3

Status: Completed

Sponsored by: Merck KGaA

Official(s) and/or principal investigator(s):
Medical Director, Study Director, Affiliation: Merck Serono S.A., Geneva

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.

Official title: An Open Study of the Safety and Efficacy of Saizen®, (Recombinant Human Growth Hormone, r-hGH), in Children Born With Serious Intra-uterine Growth Retardation (IUGR) Treated to Final Height

Study design: Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment

Primary outcome:

Final Height

Height Standard Deviation Score (HSDS)

Secondary outcome: Parental Adjusted Height Standard Deviation Score (PAHSDS)

Minimum age: N/A. Maximum age: N/A. Gender(s): Both.

Criteria:

Inclusion Criteria:

- Previous inclusion, good compliance and normal completion of GF4001 or GF6283 in the

treatment of growth failure in children born with serious IUGR (3-year continuous r-hGH treatment in GF4001 or 2-year continuous or intermittent r-hGH treatment in GF6283).

- Increase in height greater than 0. 5 standard deviation (SD) during the first 2 years

of r-hGH treatment in GF4001 or after 2 years of continuous or intermittent r-hGH treatment in GF6283.

- A written Informed Consent at the beginning of the pre-study visit must be obtained

from the parent(s)/legal guardian(s), with the understanding that consent may be withdrawn by the subject or parents at any time without prejudice to their future medical care. Children able to understand the trial should personally sign and date the written informed consent, too.

- Other protocol-defined inclusion criteria may apply.

Exclusion Criteria:

- Known multiple malformation syndrome with severe psychomotor retardation and/or body

hemihypertrophy.

- Severe psychomotor retardation.

- Severe congenital malformations.

- Other protocol-defined exclusion criteria may apply.

Related publications:

Sempé M, Pédron G, Roy-Pernot M-P. Auxologie, méthode et séquences. Paris: Theraplix, 1979.

Starting date: November 1998
Last updated: September 9, 2013