E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Children Born With Serious Intra-uterine Growth Retardation
|
|
E.1.1.1 | Medical condition in easily understood language |
Children Born With Serious Intra-uterine Growth Retardation
|
|
E.1.1.2 | Therapeutic area | Body processes [G] - Metabolic Phenomena [G03] |
MedDRA Classification |
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 primary objective was to follow children born with serious IUGR, and treated with 1.4 IU/kg/week (0.47 mg/kg/week) of Saizen according to various therapeutic schedules, until they reach final height while assessing both efficacy and safety (studies A, B and C).
|
|
E.2.2 | Secondary objectives of the trial |
-To compare the effect on final height of continuous versus intermittent long term r-hGH administration (study A)
- To study the efficacy and safety of r-hGH therapy during puberty in children born with serious IUGR (study B) as well as the effect of r-hGH on the timing and tempo of puberty in this population
- To assess the effect of the schedule and duration of the initial Saizen treatment (during studies 4001 and 6283) on final height (study C). |
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
•Previous inclusion, good compliance and normal completion of GF4001 or GF6283 in the treatment of growth failure in children born with serious IUGR (3-year continuous r-hGH treatment in GF4001 or 2-year continuous or intermittent r-hGH treatment in GF6283).
•Increase in height greater than 0.5 standard deviation (SD) during the first 2 years of r-hGH treatment in GF4001 or after 2 years of continuous or intermittent r-hGH treatment in GF6283.
•A written Informed Consent at the beginning of the pre-study visit must be obtained from the parent(s)/legal guardian(s), with the understanding that consent may be withdrawn by the subject or parents at any time without prejudice to their future medical care. Children able to understand the trial should personally sign and date the written informed consent, too.
•Other protocol-defined inclusion criteria may apply.
|
|
E.4 | Principal exclusion criteria |
•Known multiple malformation syndrome with severe psychomotor retardation and/or body hemihypertrophy.
•Severe psychomotor retardation.
•Severe congenital malformations.
•Other protocol-defined exclusion criteria may apply.
|
|
E.5 End points |
E.5.1 | Primary end point(s) |
Final Height
Height Standard Deviation Score (HSDS) |
|
E.5.1.1 | Timepoint(s) of evaluation of this end point |
One year after final height was attained up to 10.6 years |
|
E.5.2 | Secondary end point(s) |
Parental Adjusted Height Standard Deviation Score (PAHSDS) |
|
E.5.2.1 | Timepoint(s) of evaluation of this end point |
One year after final height was attained up to 10.6 years |
|
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 | No |
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 | Yes |
E.8.1.1 | Randomised | Yes |
E.8.1.2 | Open | Yes |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | No |
E.8.1.5 | Parallel group | Yes |
E.8.1.6 | Cross over | No |
E.8.1.7 | Other | No |
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 | Yes |
E.8.2.3.1 | Comparator description |
|
E.8.2.4 | Number of treatment arms in the trial | 6 |
E.8.3 |
Will this trial be conducted at a single site globally?
| No |
E.8.4 | Will this trial be conducted at multiple sites globally? | Yes |
E.8.6 Trial involving sites outside the EEA |
E.8.6.2 | Trial being conducted completely outside of the EEA | No |
E.8.6.3 | Specify the countries outside of the EEA in which trial sites are planned |
|
E.8.7 | Trial has a data monitoring committee | No |
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.8.9.2 | In all countries concerned by the trial years | 12 |
E.8.9.2 | In all countries concerned by the trial months | 3 |