E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Children and adolescents with growth failure associated with primary insulin-like growth factor deficiency (IGFD). |
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E.1.1.1 | Medical condition in easily understood language |
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E.1.1.2 | Therapeutic area | Diseases [C] - Hormonal diseases [C19] |
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 |
The primary objective of this extension study is to collect safety and efficacy data on the continued use of Increlex in children and adolescents treated for primary IGFD (height Standard Deviation Score (SDS) and IGF-1 SDS <–2 and normal stimulated Growth Hormone (GH)). |
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E.2.2 | Secondary objectives of the trial |
The secondary objective of the study was the construction of multivariate models to understand the relationship between IGF-1 exposure and the promotion of normal growth and pubertal development. |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
- Completion of the assessments of Visit 9 (12 months) of Protocol MS301 |
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E.4 | Principal exclusion criteria |
- Incomplete participation in MS301 defined as having poor dosing compliance (omission of ≥ 20% of scheduled Increlex doses in 2 three-month intervals) or failing to comply with visit schedule
- Known or suspected allegy to the trial product (mecasermin, recombinant human IGF-1 injection) or its formulation
- Development or presence of a chronic condition including but not limited to diabetes mellitus, inborn errors of the metabolism, osteo- or chondrodystrophies, disease of the genitourinary, cardiopulmonary, gastrointestinal or central nervous system or chronic daily medication use except as approved by Medical Monitor
- Pregnancy
- Any social or medical condition that, in the opinion of the investigator would be detrimental to either the subject or the study |
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E.5 End points |
E.5.1 | Primary end point(s) |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
From Baseline (Visit 1) to week 52 (Visit 4) |
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E.5.2 | Secondary end point(s) |
- For the twice-daily dosing portion of the study the secondary efficacy endpoint is change in height SD score.
- For the once-daily dosing portion of the study the secondary efficacy endpoint is the change in height SDS during the year of once-daily dosing. |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
From Baseline (Visit 1) to week 52 (Visit 4) |
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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 | 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 | No |
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 |
Increlex different dosage combinations |
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E.8.2.4 | Number of treatment arms in the trial | 3 |
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 | Yes |
E.8.6.3 | Specify the countries outside of the EEA in which trial sites are planned |
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E.8.7 | Trial has a data monitoring committee | Yes |
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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E.8.9 Initial estimate of the duration of the trial |
E.8.9.2 | In all countries concerned by the trial years | 1 |