E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
- 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.
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MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 7.1 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10056438 |
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E.1.3 | Condition being studied is a rare disease | No |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
To identify the most representative serum biomarkers after one month of Saizen therapy in Growth hormone deficiency (GHD) and Turner Syndrome (TS) children. |
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E.2.2 | Secondary objectives of the trial |
- To explore the contribution of selected genes to the phenotype of GHD and TS children; - To explore the contribution of gene polymorphisms to the levels of serum biomarkers in GHD and TS children after one month of Saizen therapy; - 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. |
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E.2.3 | Trial contains a sub-study | Information not present in EudraCT |
E.3 | Principal inclusion criteria |
- 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the duration of the trial - Parent's or guardian's written informed consent with the understanding that consent may be withdrawn at any time without prejudice to future medical care.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 |
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E.4 | Principal exclusion criteria |
- 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. |
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E.5 End points |
E.5.1 | Primary end point(s) |
To identify the most responsive serum biomarkers after one month of Saizen therapy in GHD and TS children. |
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E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | No |
E.6.2 | Prophylaxis | No |
E.6.3 | Therapy | No |
E.6.4 | Safety | No |
E.6.5 | Efficacy | No |
E.6.6 | Pharmacokinetic | No |
E.6.7 | Pharmacodynamic | No |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | No |
E.6.10 | Pharmacogenetic | Information not present in EudraCT |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | Yes |
E.6.13.1 | Other scope of the trial description |
To identify the most responsive serum biomarkers |
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E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | No |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | No |
E.7.1.3.1 | Other trial type description | |
E.7.2 | Therapeutic exploratory (Phase II) | No |
E.7.3 | Therapeutic confirmatory (Phase III) | No |
E.7.4 | Therapeutic use (Phase IV) | Yes |
E.8 Design of the trial |
E.8.1 | Controlled | No |
E.8.1.1 | Randomised | No |
E.8.1.2 | Open | Information not present in EudraCT |
E.8.1.3 | Single blind | Information not present in EudraCT |
E.8.1.4 | Double blind | Information not present in EudraCT |
E.8.1.5 | Parallel group | Information not present in EudraCT |
E.8.1.6 | Cross over | Information not present in EudraCT |
E.8.1.7 | Other | Information not present in EudraCT |
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | Information not present in EudraCT |
E.8.2.2 | Placebo | Information not present in EudraCT |
E.8.2.3 | Other | Information not present in EudraCT |
E.8.3 |
The trial involves single site in the Member State concerned
| No |
E.8.4 | The trial involves multiple sites in the Member State concerned | Yes |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | Yes |
E.8.6.2 | Trial being conducted completely outside of the EEA | Information not present in EudraCT |
E.8.6.3 | If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned |
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E.8.7 | Trial has a data monitoring committee | Information not present in EudraCT |
E.8.8 |
Definition of the end of the trial and justification where it is not the last
visit of the last subject undergoing the trial
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For administrative and safety reporting purposes, the end of the study will be defined as the date of the final clinical database lock. This provides for a single and conservative definition across all study sites. |
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E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | |
E.8.9.1 | In the Member State concerned months | 12 |
E.8.9.1 | In the Member State concerned days | |
E.8.9.2 | In all countries concerned by the trial months | 12 |