A phase IV open-label study of predictive markers in Growth Hormone Deficient and Turner Syndrome prepubertal children treated with SAIZEN.Estudio abierto en fase IV sobre marcadores predictivos en niños prepuberales con Déficit de Hormona de Crecimiento y síndrome de Turner tratados con Saizen R. - Predictive markers in GHD and TS children treated with SAIZEN.
- Conditions
- - growth failure in children caused by decreased or absent secretion of endogenous growth hormone.- growth failure in girls with gonadal dysgenesis (Turner Syndrome), confirmed by chromosomal analysis.MedDRA version: 7.1Level: PTClassification code 10056438
- Registration Number
- EUCTR2004-005054-31-ES
- Lead Sponsor
- Serono International S.A.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 318
- Children with one of the following diagnoses who are candidate for Saizen therapy:
A) GHD: documented pre-established diagnosis of GHD with a GH peak response of <10 micrograms/L with 2 GH stimulation tests, without priming with oestradiol
B) Turner syndrome: documented pre-established diagnosis by karyotype
- Prepubertal status according to Tanner (stage 1)
- Pre-established history of normal tyroid function or adequate substitution for at least 3 months
- Weight for stature within the population specific normal range (>5th and <95th percentiles) for gender
- Willingness and ability to comply with the protocol for the duration of the trial
- Parent's or guardian's written informed consent with the understanding that consent may be withdrawn at any time without prejudice to future medical care. If the child is old enough to read and write, a separate form will be given
Are the trial subjects under 18? yes
Number of subjects for this age range:
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range
- Acquired GHD due to central nervous system tumour, trauma, infection, infiltration (documented by imaging), and a history of irradiation or cranial surgery;
- Previous treatment with GH, GHRH, anabolic steroids or any treatment affecting growth;
- Previous treatment with corticosteroids except topical or inhaled for atopic disease; or when used for hormonal substitution if the condition and treatment regimen has been stable for at least 3 months;
- Severe associated pathology affecting growth such as malnutrition, malabsorption or bone dysplasia;
- Chronic severe kidney or liver or infectious disease;
- Acute or severe illness during the previous 6 months;
- Active malignancy;
- History or active Idiopathic intra-cranial hypertension;
- Diabetes Mellitus type I and II
- Any autoimmune disease;
- Use of investigational drug or participation in another clinical study within the last three months.
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Main Objective: To identify the most representative serum biomarkers after one month of Saizen therapy in Growth hormone deficiency (GHD) and Turner Syndrome (TS) children.;Secondary Objective: - To explore the contribution of selected genes to the phenotype of GHD and TS children;<br>- To explore the contribution of gene polymorphisms to the levels of serum biomarkers in GHD and TS children after one month of Saizen therapy;<br>- To explore the relationship between changes in gene expression profiling, the changes in serum biomarkers and the spectrum of gene polymorphisms in a subset of GH and TS children (defined as the <25th and >75th percentiles of IGF-I levels) after one months of Saizen therapy.;Primary end point(s): To identify the most responsive serum biomarkers after one month of Saizen therapy in GHD and TS children.
- Secondary Outcome Measures
Name Time Method