Saizen in Intra-uterine Growth Retardation

NCT01400698 | Completed Phase 3 Results

• 1 other identifier: 20184
• Study Type: interventional
• Target: 91
• Locations: 0 countries
• Sites: N/A

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.

Conditions

Children Born With Serious Intra-uterine Growth Retardation

Keywords

Intra-uterine growth retardation (IUGR); Saizen; Recombinant human growth hormone (r-hGH); Final height; Bone age; SGA (Small for Gestational Age)

Outcome Measures

Primary Outcomes (2)

• Final Height

  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.

  One year after final height was attained up to 10.6 years

• Height Standard Deviation Score (HSDS)

  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)

  One year after final height was attained up to 10.6 years

Secondary Outcomes (1)

• Parental Adjusted Height Standard Deviation Score (PAHSDS)

  One year after final height was attained up to 10.6 years

Study Arms (3)

Saizen® (Continuous or intermittent treatment)

EXPERIMENTAL

Drug: Saizen® A

Saizen® (Observed and then continuous or no treatment)

EXPERIMENTAL

Drug: Saizen® B

Observation only

OTHER

Other: Observation only

Interventions

Saizen® A DRUG

Continuous or intermittent treatment with recombinant human Growth Hormone (r-hGH) 0.067 milligram/kilogram/day (mg/kg/day) subcutaneously (sc).

Also known as: r-hGH, Somatropin

Saizen® (Continuous or intermittent treatment)

Saizen® B DRUG

Observed until the first signs of puberty and then continuous treatment with r-hGH 0.067 mg/kg/day sc or observed without treatment.

Also known as: r-hGH, Somatropin

Saizen® (Observed and then continuous or no treatment)

Observation only OTHER

Subjects were only observed.

Observation only

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• Known multiple malformation syndrome with severe psychomotor retardation and/or body hemihypertrophy.
• Severe psychomotor retardation.
• Severe congenital malformations.

Sponsors & Collaborators

• Merck KGaA, Darmstadt, Germany: lead
• Merck Serono S.A., Geneva: collaborator

Related Publications (3)

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

  BACKGROUND

• Simon D, Leger J, Fjellestad-Paulsen A, Crabbe R, Czernichow P; SGA Study Group. Intermittent recombinant growth hormone treatment in short children born small for gestational age: four-year results of a randomized trial of two different treatment regimens. Horm Res. 2006;66(3):118-23. doi: 10.1159/000093832. Epub 2006 Jun 12.

  PMID: 16772718 RESULT

• Fjellestad-Paulsen A, Czernichow P, Brauner R, Bost M, Colle M, Lebouc JY, Lecornu M, Leheup B, Limal JM, Raux MC, Toublanc JE, Rappaport R. Three-year data from a comparative study with recombinant human growth hormone in the treatment of short stature in young children with intrauterine growth retardation. Acta Paediatr. 1998 May;87(5):511-7. doi: 10.1080/08035259850158209.

  PMID: 9641731 RESULT

MeSH Terms

Interventions

Human Growth Hormone

Intervention Hierarchy (Ancestors)

Growth Hormone; Pituitary Hormones, Anterior; Pituitary Hormones; Peptide Hormones; Hormones; Hormones, Hormone Substitutes, and Hormone Antagonists; Peptides; Amino Acids, Peptides, and Proteins

Results Point of Contact

• Title: Merck KGaA Communication Center
• Organization: Merck Serono, a division of Merck KGaA

service@merck.de

+49-6151-72-5200

Study Officials

• Medical Director

  Merck Serono S.A., Geneva

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 21, 2011
• First Posted: July 22, 2011
• Study Start: November 1, 1998
• Primary Completion: February 1, 2010
• Study Completion: February 1, 2010
• Last Updated: September 18, 2013
• Results First Posted: September 25, 2012

Record last verified: 2013-09