Saizen in Intra-uterine Growth Retardation
Condition: Children Born With Serious Intra-uterine Growth Retardation
MeSH terms: Fetal Growth Retardation
Intervention: Saizen® A; Saizen® B; Observation only
Intervention description: Continuous or intermittent treatment with recombinant human Growth Hormone (r-hGH) 0.067 milligram/kilogram/day (mg/kg/day) subcutaneously (sc).; Observed until the first signs of puberty and then continuous treatment with r-hGH 0.067 mg/kg/day sc or observed without treatment.; Subjects were only observed.
Phase: Phase 3
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: None (Open Label)
Enrollment: 91.0
Sex: All
Healthy volunteers: No
Keywords: Intra-uterine growth retardation (IUGR); Saizen; Recombinant human growth hormone (r-hGH); Final height; Bone age; SGA (Small for Gestational Age)

Summary:

      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.
    

Eligibility 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.