E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
|
E.1.1.1 | Medical condition in easily understood language |
Short stature due to Noonan syndrome |
|
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 | 20.0 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10029748 |
E.1.2 | Term | Noonan syndrome |
E.1.2 | System Organ Class | 10010331 - Congenital, familial and genetic disorders |
|
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 evaluate the growth promoting effect of NN-220 (somatropin [genetical recombination]) from baseline to 104 weeks of treatment in short stature due to Noonan syndrome. |
|
E.2.2 | Secondary objectives of the trial |
To evaluate the safety profile of NN-220 from baseline to 104 weeks of treatment in short stature due to Noonan syndrome. |
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
- Japanese children with Noonan syndrome clinically diagnosed in one of the following ways o Clinically diagnosed by at least two medical experts using van der Burgt score list o Clinically diagnosed by one medical expert using van der Burgt score list and diagnosed by result of genetic testing for Noonan syndrome. o Clinically diagnosed by one medical expert using van der Burgt score list and diagnosed by the same medical expert based on the results of centralised evaluation of facial change using van der Burgt score list. - Height Standard Deviation Score (SDS): −2 SDS or below (according to the Japanese reference data) - Age: boys 3 to less than 11 years, girls 3 to less than 10 years - Height records must be available within the period between 40 and 64 weeks prior to Visit 1 (screening) - Prepubertal children (definition for girls breast and pubes of Tanner stage is I, and none of menses, and for boys testicular volume less than 4 mL, and pubes and penis of Tanner stage is I) |
|
E.4 | Principal exclusion criteria |
- Children with known or suspected hypersensitivity against human growth hormone (hGH) or related products (including any components of the trial products). - Children with diabetic type diagnosed with the Japanese Diabetes Society Classification. - Children with history or presence of active malignancy. - Children who have received GH (growth hormone) treatment. - Children who have received systemic administration of the following medications within two years prior to Visit 1 (screening): Thyroid hormone (except replacement therapy), antithyroid hormone, androgen, oestrogen, progesterone, anabolic steroid, adrenocortical steroid (treatment period greater than or equal to 13 weeks), derivative of gonadotropin releasing hormone and somatomedin C (IGF-I). |
|
E.5 End points |
E.5.1 | Primary end point(s) |
|
E.5.1.1 | Timepoint(s) of evaluation of this end point |
From baseline to 104 weeks of treatment |
|
E.5.2 | Secondary end point(s) |
1. Incidence of treatment emergent adverse events (AEs) 2. Change in IGF-I 3. Change in HbA1c |
|
E.5.2.1 | Timepoint(s) of evaluation of this end point |
1. During 104 weeks of treatment 2. From baseline to 104 weeks of treatment 3. From baseline to 104 weeks of treatment |
|
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 | No |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | Yes |
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 | 2 |
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 |
|
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 | 4 |
E.8.9.2 | In all countries concerned by the trial months | 10 |
E.8.9.2 | In all countries concerned by the trial days | 0 |