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Phase 3 N=91 Randomized Treatment

Saizen in Intra-uterine Growth Retardation

Children Born With Serious Intra-uterine Growth Retardation

Enrolled (actual)
91
Serious AEs
15.4%
Results posted
Sep 2012
Primary outcome: Primary: Final Height — 145.90; 150.06; 153.27; 155.59 cm

Study Design & Population

Study type
Interventional
Phase
Phase 3
Interventions
Saizen® A (Drug); Saizen® B (Drug); Observation only (Other)
Age
Pediatric, Adult, Older Adult
Sex
All
Sponsor
Merck KGaA, Darmstadt, Germany
Primary completion
Feb 2010

Outcome Measures

OutcomeResultp-value
PRIMARY
Final Height
145.90; 150.06; 153.27; 155.59
PRIMARY
Height Standard Deviation Score (HSDS)
-1.45; -1.90; -2.33; -1.99
SECONDARY
Parental Adjusted Height Standard Deviation Score (PAHSDS)
-0.82; -1.52; -1.64; -1.24

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.
View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT01400698). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.

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