Dettagli per singolo studio

DEC-NET Serial number FR520

Published online	02/03/2006 23.05.00
Last updated	02/03/2006 23.04.29

Other protocol ID number	A6281271
Current trial status	Open (actively recruiting new participants)
Major Disease (ICD9 class)	Lack of expected normal physiological development; Failure to gain weight; Short stature
Experimental drug
SOMATROPIN Treatment regimen (dosage and duration) Genotonorm administrated at a weekly dose of 0.46 mg/kg divided in 7 daily subcutaneous injections. GH treatment duration will be up to 3 years and will be stopped earlier if glucocorticoids have been withdrawn for 6 months or more.
Gender	Both
Age (range)	< 13 years
Eligibility criteria
Inclusion criteria
-Bone age < 13 years for a boy and <11 years for a girl -Naïve child : measured height < -2 SD. -Child currently treated by GH : Annual growth rate > SD for CA -Glucocorticosteroid treatment or 12 months at least. -Glucocorticosteroid dose > 0.2 mg/kg/day of prednisone or equivalent dose over the last 12 months -Glucocorticosteroid treatment is treatment is anticipated to be sustained for 1 more year at least. -GH treatment request by a physician who can initiate Genetonorm treatment according to the French SCP. That is to say a physician working in a hospital pediatric department or in a hospital endocrinology and metabolic disease department and, who is a pediatrican and/or a pediatric endocrinologist. -The child benefits of the French social security cover. -Informed consent parents and patient
Exclusion criteria
-Glucose intolerance on an oral glucose tolerance test dated less than 3 months or diabetes mellitus. -Syndrome known to be associated with an increased risk of cancer e.g. family history of adenomatous polyposis -Pathological condition or disease for which GH treatment is already approved in France. -Participation in any other studies involving investigational or marketed products, concomitantly or within 30 days prior to entry in the study. -Unable and/or unlikely to comprehend and/or follow GH treatment and /or the protocol. -A previous history of intolerance or hypersensitivity to the study drug, or to drugs with similar chemical structures. -Patients who are known or are suspected allergic to the preservative metacresol. -Other severe acute or chronic medical or psychiatric condition or laboratory abnormality that may increase the risk associated with trial participation or investigational product administration or may interfere with the interpretation of trial results and, in the judment of the investigator, would make the subject inappropriate for entry into this trial.
Trial design/methodology
Phase	3
Kind of study	Efficacy Safety
Design
Purpose of study
Primary: -To show an increase in height after 3 years of treatment. Height in SD for chronological age (CA) after 3 years will be compared to height in SD for CA before inclusion in the trial. Secondary: -To estimate the evolution of height and growth rate after 1, 2 and 3 years of GH treatment. -To estimate the prognostic factors of total height increased (in SD for CA) and growth rate (SD for CA) after 3 years of GH treatment. -To confirm the good clinical and biological safty of GH treatment in such children.
Primary outcomes
-Difference between height in SD for chronological age after 3 years and height in SD for chronological age at visit 2 (start GH treatment).
Secondary outcomes
Clinical and radiological parameters: Height, Weight , bone age and bone age/ Chronological age after 1, 2 and 3 years the treatment.
Summary of study design, objectives, and ongoing research findings
This is prospective, longitudinal, open , non-randomized and uncontrolled study. The efficacy of Genotonorm is evaluated once a year over 3 years. Genotonorm administrated at a weekly dose of 0.46 mg/kg divided in 7 daily subcutaneous injections. The main outcome of efficacy will be the difference between height in SD for chronological age after 3 years and height in SD for chronological age at visit 2 (starting of GH treatment).