Yükleniyor... / Loading...
Efficacy and Safety Evaluation of Recombinant Human Growth Hormone (r-hGH), Saizen®, on a Population of Children With Hypochondroplasia, Treated at Least 3 Years or Until Near Final Height, When Applicable, in Comparison With a Historic Cohort of Non-treated Children
Bu çalışma, Hipokondroplazi (hypochondroplasia, bir cücelik tipi) olan çocuklarda rekombinant insan büyüme hormonu (Saizen) tedavisinin boy üzerindeki etkinliğini ve güvenliğini, tedavi almamış tarihsel bir grupla karşılaştırıyor. FGFR3 geni mutasyon analizi yapılmış, 3 yaş üstü, boyu kısa çocuklar dahil edilmiştir. Faz 2 aşamasında yürütülmüş ve tamamlanmıştır.
Yukarıdaki özet, resmî kaydın sade dile aktarılmış halidir. Orijinal İngilizce başlık referans için gösterilir.
Kriterler ClinicalTrials.gov kaydından orijinal İngilizce haliyle alınmıştır. Uygunluk kararını yalnızca deneyi yürüten ekip verebilir.
Inclusion Criteria: * Male or female children with hypochondroplasia defined by a disproportional short limb height and a X-ray evidence of shortening of the long bones and failure of increase in the interpedicular distance between lumbar vertebrae L1 and L5 * Result of genetic analysis for mutation of gene FGFR3 already known or ongoing analysis at the beginning of the study * Chronological age greater than or equal to 3 years * Height for chronological age less than or equal to - 2 SDS * Bone age less than or equal to 11 years for girls and 13 years for boys * A written informed consent at the beginning of the pre-treatment period must be obtained from the parent(s)/legal guardian(s). Children able to understand the trial should personally sign and date the written informed consent Additional inclusion criteria for each study prolongation: * Bone age at Month 36 or Month 60 is compatible with treatment prolongation according to investigator opinion * Subject is still under r-hGH treatment with Saizen® at Month 36 or Month 60 * Height gain greater than or equal to + 1 SDS after the 2 first years of treatment for treatment prolongation at Month 36 and growth velocity greater than or equal to 5 centimeter (cm) per year, with bone age less than 14 years for females or less than 16 years for males for treatment prolongation at Month 60 * According to investigator opinion, gene mutations of the subjects are not in connection with observed side effects during the 3 or 5 first years of treatment * An updated written informed consent must be obtained from the parent(s)/legal guardian(s) before the start of each study prolongation. Children able to understand the trial should personally sign and date the written informed consent Exclusion Criteria: * Turner's Syndrome in girls * Active malignant neoplastic disease * Severe congenital malformations * Proliferative or preproliferative diabetic retinopathy * Evidence of any progression or recurrence of an underlying intra-cranial space occupying lesion * Severe psychomotor retardation * Diabetes mellitus or history of significant glucose intolerance as defined by a fasting blood glucose greater than 6.4 millimole per liter (mmol/L) * Known renal insufficiency as defined by serum creatinine level 1.0 milligram per deciliter (mg/dL) (88 micromole per liter \[mcmol/L\]) * Known hepatic disease as defined by elevated liver enzymes or total bilirubin (\* 2 Normal) * Current congestive heart failure, untreated hypertension, serious chronic edema of any cause * Chronic infectious disease * History of intracranial hypertension with papilledema * Previous or ongoing treatment with sex steroid therapy such as estrogens or testosterone * Previous or ongoing treatment with any therapy that may directly influence growth, including Growth Hormone (GH), Growth Hormone Releasing Hormone (GHRH) and long duration corticosteroids therapy * Known hypersensitivity to somatropin or any of the excipients * Epiphyseal fusion * Participation to any clinical study within the 30 days preceding study entry * Pregnant females