E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Growth hormone deficiency (GHD) in prepubertal children |
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E.1.1.1 | Medical condition in easily understood language |
Lack of growth hormone in the body |
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E.1.1.2 | Therapeutic area | Diseases [C] - Hormonal diseases [C19] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 20.0 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10056438 |
E.1.2 | Term | Growth hormone deficiency |
E.1.2 | System Organ Class | 10014698 - Endocrine disorders |
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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 assess long-term safety of weekly TransCon hGH in children with GHD previously treated in a phase 3 TransCon hGH trial |
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E.2.2 | Secondary objectives of the trial |
- To assess annualized height velocity (HV) with long-term dosing of weekly TransCon hGH treatment - To assess the proportion of subjects with IGF-1 standard deviation score (SDS) in the normal range of 0.0 to +2.0 with long-term dosing of weekly TransCon hGH treatment - To evaluate the change in height standard deviation scores (ΔHSDS) with long-term dosing of weekly TransCon hGH treatment - To determine the incidence of antibodies against TransCon hGH (anti-hGH and anti-PEG) with long-term dosing of weekly TransCon hGH treatment - To assess the preference for weekly TransCon hGH or daily Genotropin - To assess the treatment satisfaction of weekly TransCon hGH over time
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1) Children who have completed a prior phase 3 TransCon hGH trial 2) Children who have not permanently discontinued investigational product in the prior trial 3) Written, signed, informed consent of the parent or legal guardian of the subject and written assent of the subject as required by the institutional review board/human research ethics committee/independent ethics committee (IRB/HREC/IEC) |
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E.4 | Principal exclusion criteria |
1) Poorly-controlled diabetes mellitus (HbA1c ≥ 8.0%) or diabetic complications 2) Evidence of closed epiphyses, defined as bone age > 14.0 years for females or > 16.0 years for males 3) Major medical conditions unless approved by Medical Expert 4) Known hypersensitivity to the components of the trial medication 5) Likely to be non-compliant with respect to trial conduct (in regard to the subject and/or the parent/legal guardian/caregiver) 6) Pregnancy 7) Any other reason that in the opinion of the investigator would prevent the subject from completing participation or following the trial schedule |
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E.5 End points |
E.5.1 | Primary end point(s) |
Safety Endpoints The safety endpoints as measured throughout the long-term dosing of weekly TransCon hGH treatment include the following: Incidence of AEs Incidence of antibodies against hGH, including neutralizing antibodies Incidence of antibodies against PEG Incidence of antibodies against TransCon hGH Incidence of IGF-1 SDS > 2.0, > 3.0 Parameters of HbA1c and lipids Hormone levels, including thyroid status and morning cortisol All other hematology and chemistry parameters Vital sign measurements |
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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) |
Efficacy Endpoints The efficacy endpoints of long-term weekly TransCon hGH treatment include the following: Annualized HV ΔHSDS Proportion of subjects with IGF-1 SDS of 0 to +2.0. Additionally, cut points of -2.0 to +2.0 and -1.0 to +2.0 will be assessed. IGF-1 SDS IGFBP-3 SDS Pharmacodynamic Endpoint Serum IGF-1 SDS at 5 days ±1 day post-dose Other Endpoints Preference for weekly TransCon hGH or daily Genotropin treatment Satisfaction with weekly TransCon hGH
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
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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 | Yes |
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 | 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 |
The trial involves single site in the Member State concerned
| Yes |
E.8.4 | The trial involves multiple sites in the Member State concerned | No |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 20 |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | Yes |
E.8.6.2 | Trial being conducted completely outside of the EEA | No |
E.8.6.3 | If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned |
Armenia |
Australia |
Belarus |
Bulgaria |
Canada |
Georgia |
Greece |
Italy |
New Zealand |
Poland |
Romania |
Russian Federation |
Turkey |
Ukraine |
United States |
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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.8.9.1 | In the Member State concerned years | 3 |
E.8.9.1 | In the Member State concerned months | |
E.8.9.1 | In the Member State concerned days | |
E.8.9.2 | In all countries concerned by the trial years | 3 |