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Saizen in Intra-uterine Growth Retardation

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT01400698
Recruitment Status : Completed
First Posted : July 22, 2011
Results First Posted : September 25, 2012
Last Update Posted : September 18, 2013
Merck Serono S.A., Geneva
Information provided by (Responsible Party):
Merck KGaA, Darmstadt, Germany

Brief 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.

Condition or disease Intervention/treatment Phase
Children Born With Serious Intra-uterine Growth Retardation Drug: Saizen® A Drug: Saizen® B Other: Observation only Phase 3

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 91 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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 Start Date : November 1998
Actual Primary Completion Date : February 2010
Actual Study Completion Date : February 2010

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Hormones

Arm Intervention/treatment
Experimental: Saizen® (Continuous or intermittent treatment) Drug: Saizen® A
Continuous or intermittent treatment with recombinant human Growth Hormone (r-hGH) 0.067 milligram/kilogram/day (mg/kg/day) subcutaneously (sc).
Other Names:
  • r-hGH
  • Somatropin

Experimental: Saizen® (Observed and then continuous or no treatment) Drug: Saizen® B
Observed until the first signs of puberty and then continuous treatment with r-hGH 0.067 mg/kg/day sc or observed without treatment.
Other Names:
  • r-hGH
  • Somatropin

Observation only Other: Observation only
Subjects were only observed.

Primary Outcome Measures :
  1. Final Height [ Time Frame: One year after final height was attained up to 10.6 years ]
    Final height was defined as the height reached 1 year after height velocity (HV) was less than 2 centimeter/year (cm/year). Height velocity was the change in height since the previous year's measurement. Height was measured with a wall-mounted stadiometer (or in supine position if the participant's age was less than 3 years) and the measurement was repeated thrice by the same observer. The mean of the values obtained in the repeated measurements was taken for the analysis.

  2. Height Standard Deviation Score (HSDS) [ Time Frame: One year after final height was attained up to 10.6 years ]
    HSDS was calculated as height minus reference mean height divided by SD of the reference mean height, both given by the reference growth table (Sempe) for the corresponding chronological age at the height measurement. Greater HSDS indicate greater height. (Sempe M et al., 1979)

Secondary Outcome Measures :
  1. Parental Adjusted Height Standard Deviation Score (PAHSDS) [ Time Frame: One year after final height was attained up to 10.6 years ]
    PAHSDS is the distance between the participant's current and target heights, expressed in units of SD of the height distribution of the reference population. Target height is a measure of the height which the participant could hypothetically reach based only on his parents' heights. Target height standard deviation score (THSDS) was calculated as target height minus mean adult height of the reference population divided by SD of the mean adult height of the reference population.

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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT01400698

Sponsors and Collaborators
Merck KGaA, Darmstadt, Germany
Merck Serono S.A., Geneva
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Study Director: Medical Director Merck Serono S.A., Geneva

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

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Responsible Party: Merck KGaA, Darmstadt, Germany Identifier: NCT01400698     History of Changes
Other Study ID Numbers: 20184
First Posted: July 22, 2011    Key Record Dates
Results First Posted: September 25, 2012
Last Update Posted: September 18, 2013
Last Verified: September 2013
Keywords provided by Merck KGaA, Darmstadt, Germany:
Intra-uterine growth retardation (IUGR)
Recombinant human growth hormone (r-hGH)
Final height
Bone age
SGA (Small for Gestational Age)
Additional relevant MeSH terms:
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Fetal Growth Retardation
Fetal Diseases
Pregnancy Complications
Growth Disorders
Pathologic Processes
Hormones, Hormone Substitutes, and Hormone Antagonists
Physiological Effects of Drugs