E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
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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] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] |
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 | 10063020 |
E.1.2 | Term | Parsonage-Turner syndrome |
E.1.2 | System Organ Class | 100000004852 |
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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 |
To test the null hypothesis that subjects with Turner syndrome who received 2 years of early Humatrope treatment (initiated between 9 months and 4 years of age in study GDFG) achieve similar height (expressed as a standard deviation score; SDS) at or beyond near-final height to those who did not receive early Humatrope treatment |
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E.2.2 | Secondary objectives of the trial |
The secondary objectives of the study are to test the null hypotheses that at long-term follow-up, subjects who received 2 years of early Humatrope treatment versus those who did not (based on original GDFG randomization groups) have similar values for the following: Efficacy: • Height SDS at various ages • Chronological age at attainment of Tanner stage 2 breast development • Chronological age at attainment of near-final height (defined as height velocity ≤ 2.0 cm/year over the preceding year in the absence of additional growth impairing process or bone age ≥ 14.5 years) Safety: • Percentage of subjects for whom serious adverse events are reported • Percentage of subjects for whom certain non-serious, pre-specified adverse events (those that are commonly observed in Turner syndrome or are known to be related to GH treatment) are reported • Percentage of subjects with abnormal tympanometry and audiometry results at baseline, age 10 years, and age 16 years or endpoint |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
Previously randomized in study B9R-US-GDFG; karyotype-proven Turner syndrome |
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E.4 | Principal exclusion criteria |
Immediate family members of study site personnel directly affiliated with the study |
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E.5 End points |
E.5.1 | Primary end point(s) |
Adult height at the end of growth |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
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E.5.2 | Secondary end point(s) |
Height SDS at various ages; age at attainment of Tanner 2 breast development; Chronological age at fist visit subject attained bone age of 14.5 yrs; Reports of serious adverse events; Occurrence of pre-specified clinically relevant events; Tympanometry and audiometry results for assessment of middle ear function and hearing |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
over the course of the study for all secondary except for chronological age at first visit subject attained bone age of 14.5 years |
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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 | 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) | 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?
| 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 | 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 | 10 |
E.8.9.2 | In all countries concerned by the trial months | 0 |
E.8.9.2 | In all countries concerned by the trial days | 0 |