A phase IV open-label study of predictive markers in Growth Hormone Deficient and Turner Syndrome prepubertal children treated with Saize
- 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: 7.1 Level: PT Classification code 10056438
- Registration Number
- EUCTR2004-005054-31-GB
- Lead Sponsor
- Serono International S.A.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- Not specified
- 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- Parent's or guardian's written informed consent with the understanding that consent may be withdrawn at any time without prejudice to future medical care.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 (except non-melanomatous skin malignancies that have undergone surgical excision and / or biopsy, diagnosis and treatment to resolution);
- 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.;<br> 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.<br> ;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