E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Short stature in children born small for gestational age with no catch-up growth by 2 years of age or older |
Bambini con più di 2 anni di età con bassa statura e nati piccoli per l'età gestazionale e con un recupero di crescita non adeguato |
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E.1.1.1 | Medical condition in easily understood language |
Short stature in children born small for gestational age with no catch-up growth by 2 years of age or older |
Bambini con più di 2 anni di età con bassa statura e nati piccoli per l'età gestazionale e con un recupero di crescita non adeguato |
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E.1.1.2 | Therapeutic area | Diseases [C] - Musculoskeletal Diseases [C05] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 21.1 |
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 the effect of somapacitan versus Norditropin® on longitudinal growth in children with short stature born small for gestational age with no catch-up growth by 2 years of age or older. |
Confrontare l’efficacia di somapacitan rispetto a Norditropin® sulla crescita longitudinale nei bambini con bassa statura nati piccoli per l'età gestazionale e con un recupero della crescita non adeguato a partire dai due anni di età |
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E.2.2 | Secondary objectives of the trial |
To evaluate the effect and safety of somapacitan versus Norditropin® in children born small for gestational age with no catch-up growth by 2 years or older. |
Confrontare l’efficacia e la sicurezza di somapacitan rispetto a Norditropin® sulla crescita longitudinale nei bambini con bassa statura nati piccoli per l'età gestazionale e con un recupero della crescita non adeguato a partire dai due anni di età |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
- Pre-pubertal children: a) Boys: Age more than or equal to 2 years and 26 weeks and less than 11.0 years at screening. Testes volume less than 4 ml. b) Girls: Age more than or equal to 2 years and 26 weeks and less than 10.0 years at screening. Tanner stage 1 for breast development (no palpable glandular breast tissue) - Born small for gestational age (birth length and/or weight less than -2 standard deviation scores (SDS)) (according to national standards) - Impaired height defined as at least 2.5 standard deviations below the mean height for chronological age and gender at screening according to the standards of Centers for Disease Control and Prevention at screening - Impaired height velocity defined as annualised height velocity below the 50th percentile for chronological age and gender according to the standards of Prader calculated over a time span of minimum 6 months and maximum 18 months prior to screening - No prior exposure to growth hormone therapy or Insulin-like Growth Factor-I (IGF-I) treatment |
-Bambini in età prepuberale: a) Maschi: o Età = 2 anni e 26 settimane e < 11,0 anni allo screening. o Volume testicolo < 4 ml. b) Femmine: o Età = 2 anni e 26 settimane e < 10,0 anni allo screening. o Fase 1 di Tanner per lo sviluppo del seno (nessun tessuto mammario ghiandolare palpabile). -Nati piccoli per età gestazionale (peso e/o lunghezza alla nascita < -2 SDS) (secondo gli standard nazionali). -Altezza insufficiente definita come almeno 2,5 di deviazione standard al di sotto dell’altezza media per età cronologica e sesso allo screening secondo gli standard Centers for Disease Control and Prevention -Velocità di crescita ridotta, definita come velocità di crescita annualizzata inferiore al 50° percentile per età cronologica e sesso secondo gli standard Prader calcolati su un periodo di tempo minimo di 6 mesi e massimo di 18 mesi prima dello screening. -Nessuna precedente esposizione alla terapia con ormone della crescita o trattamento con IGF-I. |
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E.4 | Principal exclusion criteria |
- Any known or suspected clinically significant abnormality likely to affect growth or the ability to evaluate growth with standing height measurements - Children with hormonal deficiencies including suspected or confirmed growth hormone deficiency according to local practise - Current inflammatory diseases requiring systemic corticosteroid treatment for longer than 2 consecutive weeks within the last 3 months prior to screening - Children requiring inhaled glucocorticoid therapy at a dose of greater than 400 µg/day of inhaled budesonide or equivalents for longer than 4 consecutive weeks within the last 12 months prior to screening - Concomitant administration of other treatments that may have an effect on growth, e.g. but not limited to methylphenidate for treatment of attention deficit hyperactivity disorder - Diagnosis of attention deficit hyperactivity disorder - Prior history or presence of malignancy including intracranial tumours |
-Qualsiasi anomalia clinicamente significativa nota o sospetta che possa influire sulla crescita o sulla capacità di valutare la crescita con misurazioni dell’altezza in piedi: -Bambini con carenze ormonali, compreso il deficit di ormone della crescita sospetto o confermato secondo standard nazionali. -Malattie infiammatorie che hanno richiesto un trattamento sistemico con corticosteroidi per più di 2 settimane consecutive negli ultimi 3 mesi precedenti allo screening -Bambini che hanno avuto bisogno di una terapia con glucocorticoidi per via inalatoria ad una dose superiore a 400 µg/die di budesonide o equivalenti per via inalatoria per più di 4 settimane consecutive nei 12 mesi precedenti allo screening -Somministrazione concomitante di altri trattamenti che possono avere un effetto sulla crescita, quali, a titolo esemplificativo ma non esaustivo, metilfenidato per il trattamento del disturbo da deficit di attenzione e iperattività (Attention Deficit Hyperactivity Disorder, ADHD -Diagnosi di disturbo da deficit di attenzione e iperattività. -Anamnesi pregressa o presenza di tumori maligni noti inclusi i tumori intracranici. |
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E.5 End points |
E.5.1 | Primary end point(s) |
Height velocity |
Velocità di crescita |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
From baseline (week 0) to visit 5 (week 13) |
Dalla visita basale alla visita 5 (settimana 13) |
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E.5.2 | Secondary end point(s) |
1. Change in bone age 2. Change in height SDS 3. Height velocity SDS 4. Change in fasting plasma glucose (FPG) 5. Change in homeostatic model assessment (HOMA) 6. Change in glycated haemoglobin (HbA1c) 7. Change in IGF-I SDS 8. Change in Insulin-like growth factor binding protein 3 (IGFBP-3) SDS |
1. Cambiamento dell'età ossea 2. Modifica del punteggio di deviazione standard dell'altezza 3. Punteggio di deviazione standard della velocità in altezza 4. Variazione del glucosio plasmatico a digiuno 5. Modifica della valutazione del modello omeostatico 6. Modifica dell'emoglobina glicata (HbA1c) 7. Modifica del punteggio di deviazione standard del fattore di crescita I (IGF-I) insulino-simile 8. Modifica del punteggio di deviazione standard del fattore di crescita 3 (IGFBP-3) insulino-simile |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
1: From baseline (week 0) to visit 8 (week 52) 2, 3, 7 & 8: From baseline (week 0) to visit 5 (week 13) 4 - 6: From screening (visit 1) to visit 5 (week 13) |
1: Dalla visita basale ( settimana 0) alla visita 8 (settimana 52), 2, 3, 7 & 8: Dalla visita basale ( settimana 0) alla visita 5 (settimana 13) 4 - 6: dalla visita di screening (visita 1) alla visita 5 (settimana 13) |
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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) | Yes |
E.7.3 | Therapeutic confirmatory (Phase III) | No |
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 | 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) | Yes |
E.8.2.2 | Placebo | No |
E.8.2.3 | Other | No |
E.8.2.4 | Number of treatment arms in the trial | 5 |
E.8.3 |
The trial involves single site in the Member State concerned
| No |
E.8.4 | The trial involves multiple sites in the Member State concerned | Yes |
E.8.4.1 | Number of sites anticipated in Member State concerned | 4 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 16 |
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 | Information not present in EudraCT |
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 |
Algeria |
Canada |
Israel |
Japan |
Russian Federation |
Thailand |
Ukraine |
United States |
Switzerland |
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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 | 1 |
E.8.9.1 | In the Member State concerned months | 10 |
E.8.9.1 | In the Member State concerned days | 20 |
E.8.9.2 | In all countries concerned by the trial years | 1 |
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 | 20 |