E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
growth failure associated with: - inadequate growth hormone secretion - Turner syndrome, confirmed by karyotype - chronic renal insufficiency up to renal transplantation
|
|
MedDRA Classification |
E.1.3 | Condition being studied is a rare disease | Yes |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
To establish an optimal monitoring regimen for IGF-I in NutropinAq treated children, using a newly developed capillary blood spot IGF-I measurements technology. |
|
E.2.2 | Secondary objectives of the trial |
1.To assess under clinical practice conditions, the biological variability of IGF-I (if any) in children treated with NutropinAq (physiological intra-individual variations, timing, dose of NutropinAq) and in different diseases conditions 2. Assuming a significant biological variability is observed, to identify parameters contributing to or related to this variability 3. To further validate the filter paper versus serum method IGF-I assay in the study setting 4. To assess auxological parameters during treatment with NutropinAq 5. To assess the acceptability of the NutropinAq Pen
|
|
E.2.3 | Trial contains a sub-study | Information not present in EudraCT |
E.3 | Principal inclusion criteria |
Children (boys and girls under 18) with growth failure associated with: - inadequate growth hormone secretion - Turner syndrome, confirmed by karyotype - chronic renal insufficiency up to renal transplantation Written informed consent signed by both parents or by the liable parent or by the legal guardian when applicable AND the child when applicable
|
|
E.4 | Principal exclusion criteria |
Children with hypersensitivity to somatropin or any of the excipients Children with closed epiphyses Children with active neoplasm Children with acute critical illness due to surgery, multiple accidental trauma Children with acute respiratory failure Children having received any unlicensed drug within the previous 30 days
|
|
E.5 End points |
E.5.1 | Primary end point(s) |
The number of capillary blood spot IGF-I measurements and the optimal timing of samples to assess the IGF-I status of NutropinAq treated patients |
|
E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | Yes |
E.6.2 | Prophylaxis | No |
E.6.3 | Therapy | No |
E.6.4 | Safety | No |
E.6.5 | Efficacy | Yes |
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 | No |
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) | Yes |
E.7.4 | Therapeutic use (Phase IV) | No |
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) | No |
E.8.2.2 | Placebo | No |
E.8.2.3 | Other | No |
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 |
|
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
| |
E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 1 |
E.8.9.1 | In the Member State concerned months | 6 |
E.8.9.1 | In the Member State concerned days | |
E.8.9.2 | In all countries concerned by the trial years | 2 |
E.8.9.2 | In all countries concerned by the trial months | 3 |