Saizen in Intra-uterine Growth Retardation

NCT ID: NCT01400698

Last Updated: 2013-09-18

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE3
• Total Enrollment: 91 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 1998-11-30
• Study Completion Date: 2010-02-28

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

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Saizen® (Continuous or intermittent treatment)

Group Type: EXPERIMENTAL

Saizen® A

Intervention Type: DRUG

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

Saizen® (Observed and then continuous or no treatment)

Group Type: EXPERIMENTAL

Saizen® B

Intervention Type: 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.

Observation only

Group Type: OTHER

Observation only

Intervention Type: OTHER

Subjects were only observed.

Interventions

Saizen® A

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

Intervention Type: 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.

Intervention Type: DRUG

Observation only

Subjects were only observed.

Intervention Type: OTHER

Other Intervention Names

r-hGH; Somatropin; r-hGH; Somatropin

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.

Exclusion Criteria

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

• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Merck Serono S.A., Geneva

INDUSTRY

Sponsor Role: collaborator

Merck KGaA, Darmstadt, Germany

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Medical Director

Role: STUDY_DIRECTOR

Merck Serono S.A., Geneva

References

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

Reference Type: 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.

Reference Type: RESULT

PMID: 16772718 (View on PubMed)

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.

Reference Type: RESULT

PMID: 9641731 (View on PubMed)

Other Identifiers

20184

Identifier Type: -

Identifier Source: org_study_id