E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Small for Gestational Age |
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E.1.1.1 | Medical condition in easily understood language |
Growth Hormone Deficiency |
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E.1.1.2 | Therapeutic area | Body processes [G] - Bones and nerves physological processes [G11] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 19.0 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10041093 |
E.1.2 | Term | Small for gestational age |
E.1.2 | System Organ Class | 100000004868 |
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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 evaluate safety of long-term administration of PNU-180307 (Genotropin®) until a final height was reached in short children born SGA without epiphyseal closure. |
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E.2.2 | Secondary objectives of the trial |
1. To examine height velocity, height velocity Standard Deviation Score (SDS) for chronological age, height SDS for chronological age and ∆ height SDS for chronological age 2. To comprehensively evaluate height velocity SDS for bone age, height SDS for bone age and ∆ height SDS for bone age to examine the relationship between bone age and height increase 3. To examine changes in daily lives of children with PNU-180307 (Genotropin®) treatment by means of questionnaires |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
Children with short stature due to SGA who received treatment in the study GENASG-0021-002 |
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E.4 | Principal exclusion criteria |
- Children who have any chronic disease requiring treatment with steroid hormone that may affect growth promotion including estrogen, androgen, anabolic hormone, and corticosteroids (except those for external use), and have received the treatment. - Children who have received radiotherapy or chemotherapy. - Children who have serious cardiac disease, renal disease, or hepatic disease. - Children who have diabetes mellitus with a manifestation of abnormal glucose metabolism. - Children who have serious chronic disease. - Children who have malignant tumor. - Children who are allergic to m-cresol.
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E.5 End points |
E.5.1 | Primary end point(s) |
Safety (laboratory test parameters and adverse events)
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
Month 12 (at the end of previous study) to 156 |
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E.5.2 | Secondary end point(s) |
- Height velocity SDS for chronological age. - Height velocity. - Height SDS for chronological age. - Height velocity SDS for bone age. - Height SDS for bone age.
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
Every 6 months of Treatment. |
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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) | 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 | 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 | No |
E.8.3 |
Will this trial be conducted at a single site globally?
| Yes |
E.8.4 | Will this trial be conducted at multiple sites globally? | No |
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 | 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
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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 | 13 |