Children Born With Serious Intra-uterine Growth Retardation Clinical Trial
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 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.
| Status | Completed |
| Enrollment | 91 |
| Est. completion date | February 2010 |
| Est. primary completion date | February 2010 |
| Accepts healthy volunteers | No |
| Gender | Both |
| Age group | N/A and older |
| Eligibility |
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. |
Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment
| Country | Name | City | State |
|---|---|---|---|
| n/a | |||
| Lead Sponsor | Collaborator |
|---|---|
| Merck KGaA | Merck Serono S.A., Geneva |
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 i — View Citation
Sempé M, Pédron G, Roy-Pernot M-P. Auxologie, méthode et séquences. Paris: Theraplix, 1979.
Simon D, Leger J, Fjellestad-Paulsen A, Crabbé 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 regimen — View Citation
| Type | Measure | Description | Time frame | Safety issue |
|---|---|---|---|---|
| Primary | 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 | No |
| Primary | 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 | No |
| Secondary | Parental Adjusted Height Standard Deviation Score (PAHSDS) | 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. | One year after final height was attained up to 10.6 years | No |