E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Children presenting growth failure associated with Growth Hormone Deficiency (GHD), Tuner Syndrome (TS) or Chronic Renal Insufficiency (CRI).
NutropinAq™ is given as a daily subcutaneous injection dose: GDH: 0,025- 0,035 mg/kg/day, TS : up to 0,05 mg/kg/day, CRI : up to 0,05 mg/kg/day. No dose adaptation will be allowed during the study period unless tolerance or safety mandates a dose reduction.
|
|
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 7.0 |
E.1.2 | Classification code | 10056438 |
|
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 |
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.2.2 | Secondary objectives of the trial |
The factors affecting the variability of capillary IGF-I measurements; i.e. to establish whether the variability of IGF-I levels is homogeneously distributed in the patient population and/or if individual factors affect the day to day variability of IGF-I levels.
The precision profile of capillary vs plasma IGF-I measurements.
The auxological parameters during NutropinAq treatment: changes in height, weight, growth velocity and height standard deviation score at each visit (M3 & M6).
The acceptability of the NutropinAq Pen by a questionnaire administered at M5.
|
|
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
Secundary end points:
The factors affecting the variability of capillary IGF-I measurements; i.e. to establish whether the variability of IGF-I levels is homogeneously distributed in the patient population and/or if individual factors affect the day to day variability of IGF-I levels.
The precision profile of capillary vs plasma IGF-I measurements.
The auxological parameters during NutropinAq treatment: changes in height, weight, growth velocity and height standard deviation score at each visit (M3 & M6).
The acceptability of the NutropinAq Pen by a questionnaire administered at M5.
|
|
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 | Yes |
E.6.4 | Safety | Yes |
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 | Yes |
E.6.13.1 | Other scope of the trial description |
Acceptability of delivery device. |
|
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) | 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 | No |
E.8.6.2 | Trial being conducted completely outside of the EEA | Information not present in EudraCT |
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
|
The end of the study is last partient last visit, that is 6 months after the patient's first injection. |
|
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 | 1 |
E.8.9.2 | In all countries concerned by the trial months | 6 |