E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Growth hormone deficiency in children |
Deficit di ormone della crescita nei bambini |
|
E.1.1.1 | Medical condition in easily understood language |
Growth hormone deficiency in children |
Deficit di ormone della crescita nei bambini |
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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 |
|
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 compare the effect of somapacitan vs Norditropin® on longitudinal growth in children with growth hormone deficiency. |
Confrontare l’effetto di somapacitan rispetto a Norditropin® sulla crescita longitudinale nei bambini con un deficit di ormone della crescita. |
|
E.2.2 | Secondary objectives of the trial |
To compare the safety of somapacitan vs Norditropin® in children with growth hormone deficiency. |
Confrontare la sicurezza di somapacitan rispetto a Norditropin® nei bambini con deficit di ormone della crescita. |
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
- Prepubertal children: - Boys: - Age greater than or equal to 2 years and 26 weeks and less than 11.0 years at screening - Testis volume less than 4 mL - Girls: - Age greater 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) - Confirmed diagnosis of growth hormone deficiency determined by two different growth hormone stimulation tests performed within 12 months prior to randomisation, defined as a peak growth hormone level of less than or equal to 10.0 ng/mL using the world health organisation (WHO) International Somatropin 98/574 standard - Impaired height defined as at least 2.0 standard deviations below the mean height for chronological age and gender at screening according to the standards of Center for Disease Control and Prevention - Impaired height velocity, defined as annualised height velocity below the 25th 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 - Insulin-like Growth Factor-I (IGF-I) less than -1.0 Standard Deviation Score at screening, compared to age and gender normalized range measured at central laboratory - No prior exposure to growth hormone therapy or Insulin-like Growth Factor-I treatment |
-Bambini in età prepuberale: a) Maschi: • Età = 2 anni e 26 settimane e < 11,0 anni allo screening • Volume testicolo < 4 ml. b) Femmine: • Età = 2 anni e 26 settimane e < 10,0 anni allo screening • Fase 1 di Tanner per lo sviluppo del seno (nessun tessuto mammario ghiandolare palpabile)
- Diagnosi confermata di deficit dell’ormone della crescita determinato da due diversi test di stimolazione dell’ormone della crescita effettuati nei 12 mesi prima della randomizzazione, definito come un picco di ormone della crescita di = 10,0 ng/ml utilizzando lo Standard internazionale per la somatropina 98/574 dell’OMS (Organizzazione Mondiale della Sanità)
-Altezza insufficiente definita come almeno 2.0 deviazioni standard al di sotto dell’altezza media per età cronologica e sesso allo screening secondo gli standard del Center for Disease Control and Prevention
-Velocità di crescita ridotta, definita come velocità di crescita annualizzata inferiore al 25° percentile per età e sesso cronologici 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
- IGF-I < -1.0 SDS allo screening, confrontato con l’intervallo normalizzato di età e genere misurato presso il laboratorio centrale |
|
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 - 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 - Diagnosis of attention deficit hyperactivity disorder - 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 - Prior history or presence of malignancy including intracranial tumours |
- Ipersensibilità nota o sospetta al/ai prodotto/i sperimentale/i o ai prodotti correlati
-Somministrazione di qualsiasi medicinale sperimentale nei 3 mesi precedenti lo screening oppure alla partecipazione ad un’altra sperimentazione clinica al momento della randomizzazione
- 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:
- 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 inclusi i tumori intracranici |
|
E.5 End points |
E.5.1 | Primary end point(s) |
Height velocity |
Velocità di crescita |
|
E.5.1.1 | Timepoint(s) of evaluation of this end point |
From baseline (week 0) to visit 7 (week 52) |
Dalla visita basale (settimana 0) alla visita 7 (settimana 52) |
|
E.5.2 | Secondary end point(s) |
1. Change in bone age 2. Change in Height Standard Deviation Score 3. Height Velocity Standard Deviation Score 4. Change in fasting plasma glucose 5. Change in homeostatic model assessment 6. Change in Glycated haemoglobin (HbA1c) 7. Change in Insulin-like growth factor I (IGF-I) Standard Deviation Score 8. Change in Insulin-like growth factor binding protein 3 (IGFBP-3) Standard Deviation Score |
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 insulino-simile 3 (IGFBP-3) |
|
E.5.2.1 | Timepoint(s) of evaluation of this end point |
1 & 4 – 6: From screening (week -2) to visit 7 (week 52), visit 11 (week 104), visit 15 (week 156) and visit 19 (week 208) 2, 3, 7 & 8: From baseline (week 0) to visit 7 (week 52), visit 11 (week 104), visit 15 (week 156) and visit 19 (week 208) |
1 & 4 – 6: Dallo screening (settimana -2) alla visita 7 (settimana 52), visita 11 (settimana 104), visita 15 (settimana 156) e visita 19 (settimana 208) 2, 3, 7 & 8: Dalla visita basale ((settimana 0) alla visita 7 (settimana 52), visita 11 (settimana 104), visita 15 (settimana 156) e visita 19 (settimana 208) |
|
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 | 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 | 2 |
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 | 29 |
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 |
India |
Israel |
Japan |
Korea, Republic of |
Russian Federation |
Thailand |
Ukraine |
United States |
Switzerland |
|
E.8.7 | Trial has a data monitoring committee | Yes |
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.8.9.1 | In the Member State concerned years | 5 |
E.8.9.1 | In the Member State concerned months | 1 |
E.8.9.1 | In the Member State concerned days | 15 |
E.8.9.2 | In all countries concerned by the trial years | 5 |
E.8.9.2 | In all countries concerned by the trial months | 1 |
E.8.9.2 | In all countries concerned by the trial days | 15 |