E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Secondary Hyperparathyroidism (sHPT) Receiving Maintenance Haemodialysis |
Iperparatiroidismo secondario (SHPT) nei pazienti pediatrici con malattia renale cronica (CKD) in emodialisi. |
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E.1.1.1 | Medical condition in easily understood language |
Secondary Hyperparathyroidism (sHPT) |
Iperparatiroidismo secondario (sHPT) |
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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 | 10020708 |
E.1.2 | Term | Hyperparathyroidism secondary |
E.1.2 | System Organ Class | 10014698 - Endocrine disorders |
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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 evaluate the efficacy of etelcalcetide in reducing the intact parathyroid hormone (iPTH) level in children ages > = 2 to < 18 years with secondary hyperparathyroidism (SHPT) receiving maintenance hemodialysis. |
Valutare l'efficacia di etelcalcetide nel ridurre i livelli di paratormone intatto (iPTH) in bambini di età compresa tra > = 2 e < 18 anni con iperparatiroidismo secondario (SHPT) sottoposti emodialisi di mantenimento |
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E.2.2 | Secondary objectives of the trial |
- To evaluate the efficacy of etelcalcetide in reducing the iPTH level by > 30%. - To characterize change in laboratory markers of chronic kidney disease (CKD) following etelcalcetide treatment. - To characterize the safety of etelcalcetide treatment based on laboratory values. - To characterize the pharmacokinetic (PK) of etelcalcetide treatment after single and multiple doses. |
- Valutare l'efficacia di etelcalcetide nel ridurre i livelli di iPTH di > 30% - Caratterizzare la mutazione dei marker di laboratorio della malattia renale cronica (CKD) in seguito al trattamento con etelcalcetide - Caratterizzare la sicurezza del trattamento con etelcalcetide sulla base dei valori di laboratorio - Caratterizzare la farmacocinetica (PK) del trattamento con etelcalcetide dopo dose singola e dose multipla |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
- Subject's legally acceptable representative has provided informed consent when the subject is legally too young to provide informed consent and the subject has provided written assent based on local regulations and/or guidelines prior to any study-specific activities/procedures being initiated. - Male or female subjects > = 2 to < 18 years of age at the time of enrollment. - Targeted Dry weight > = 7 kg at the time of screening week -1 - Diagnosed with CKD and SHPT undergoing hemodialysis/hemodiafiltration TIW at the time of screening > = 1 month. - Diagnosis of SHPT with the mean of the 2 consecutive central laboratory iPTH values > 300 pg/mL during screening, on separate days and within 2 weeks of enrollment obtained from the central laboratory during screening.
*Please, refers to procotol for the full list. |
- Il rappresentante legalmente accettabile del soggetto ha fornito il consenso informato quando il soggetto è legalmente troppo giovane per fornire il consenso informato e il paziente ha fornito un assenso scritto sulla base di regolamenti locali e/o linee guida prima di avviare qualsiasi attività/procedura studio specifica - Soggetti di sesso maschile o femminile di età compresa tra > = 2 e < 18 anni al momento dell'arruolamento - Peso > = 7 kg al momento dello screening, settimana 1 - Diagnosi con CKD e SHPT sottoposti ad emodialisi / emodiafiltrazione TIW al momento dello screening > = 1 mese - Diagnosi di SHPT con la media dei 2 valori consecutivi di iPTH emersi dalle analisi del laboratorio centrale > 300 pg/mL durante lo screening, in giorni diversi ed entro 2 settimane dall'arruolamento
*Fare riferimento al protocollo per la lista completa |
|
E.4 | Principal exclusion criteria |
- History of congenital long QT syndrome, second or third degree heart block, ventricular tachyarrhythmia's, history of symptomatic ventricular dysrhythmias Torsades de Pointes or other conditions associated with prolonged QT interval. - Anticipated or scheduled parathyroidectomy during the study period. - Anticipated or scheduled kidney transplant during the study period. - Subject has received a parathyroidectomy within 6 months prior to enrollment. - Current malignancy or history of other malignancy, except nonmelanoma skin cancers within the last 5 years. Prior/Concomitant Therapy - Use of concomitant medications that may prolong the QTc (eg, ondansetron, albuterol, sotalol, amiodarone, erythromycin, or clarithromycin).
*Please, refers to protocol for the full list. |
- Anamnesi di sindrome congenita del QT lungo, blocco cardiaco di secondo o terzo grado, tachiaritmia ventricolare, anamnesi di aritmie ventricolari sintomatiche torsioni di punta o altre condizioni associate all'intervallo QT prolungato. - Paratiroidectomia prevista o programmata durante lo studio - Trapianto di rene previsto o programmato durante lo studio - Il soggetto ha subito una paratiroidectomia nei 6 mesi precedenti l'arruolamento - Malignità attuale o storia di altre neoplasie maligne, tranne i tumori della pelle non melanoma negli ultimi 5 anni. Terapia preventiva / concomitante - Uso concomitante di farmaci che potrebbero prolungare il QTc (es. ondansetron, albuterolo,sotalolo, amiodarone, eritromicina o claritromicina)
*Fare riferimento al protocollo per la lista completa |
|
E.5 End points |
E.5.1 | Primary end point(s) |
Percent change in iPTH from baseline during the efficacy assessment period (EAP) at weeks 20 to 26. |
Variazione percentuale nell'iPTH rispetto al basale durante il periodo di valutazione dell'efficacia (EAP) nelle settimane da 20 a 26. |
|
E.5.1.1 | Timepoint(s) of evaluation of this end point |
Day 1, Week 20, 21, 22, 23, 24, 25 and 26. |
Giorno 1, Settimana 20, 21, 22, 23, 24, 25 e 26. |
|
E.5.2 | Secondary end point(s) |
- Achievement of a > 30% reduction from baseline in mean iPTH during the EAP. - Percent change from baseline in corrected total serum calcium (Ca) and serum phosphorus from baseline during the EAP. - Occurrence of corrected serum Ca levels < 8.0 mg/dL (2.0 mmol/L) any time during the study. - Changes in laboratory parameters, including clinical chemistry. - Occurrence of hypocalcemia (corrected serum Ca levels < 8.4 mg/dL). - Etelcalcetide plasma concentrations before and at the end of dialysis after single and multiple doses. - Etelcalcetide PK parameters including, but not limited to, maximum plasma concentration (Cmax) and plasma trough concentrations (Cmin). |
- Raggiungimento di una riduzione > 30% nell'IPTH medio rispetto al basale durante l'EAP. - Variazione percentuale rispetto al basale dei livelli sierici di calcio (Ca) corretto totale e del fosforo sierico rispetto al basale durante l'EAP - Rilevazione di livelli di Ca sierici corretti <8,0 mg/dL (2,0 mmol/L) in qualsiasi momento durante lo studio - Variazioni nei parametri di laboratorio, inclusi i parametri ematochimici - Rilevazione di ipocalcemia (livelli di Ca sierici corretti <8,4 mg/dL) - Concentrazioni plasmatiche di etelcalcetide prima e al termine della dialisi dopo dosi singole e multiple - Parametri PK dell'etelcalcetide inclusi, ma non limitati a, concentrazione plasmatica massima (Cmax) e concentrazioni plasmatiche a valle (Cmin) |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
- Day 1, Week 20, 21, 22, 23, 24, 25 and 26 - Day 1, Week 20, 21, 22, 23, 24, 25 and 26 - Weekly throughout study - Day 1 (pre-dialysis/post-dialysis), Week 5 (pre-dialysis/post-dialysis), Week 9 (pre-dialysis/post-dialysis), Week 13 (pre-dialysis/postdialysis), Week 17 (pre-dialysis/post-dialysis), Week 21 (predialysis/post-dialysis), Week 27 (pre-dialysis), End of Study (predialysis). |
- Giorno 1, Settimana 20, 21, 22, 23, 24, 25 e 26 - Giorno 1, Settimana 20, 21, 22, 23, 24, 25 e 26 - Settimanalmente durante lo studio - Giorno 1 (pre-dialisi/post-dialisi), Settimana 5 (pre-dialisi/post-dialisi), Settimana 9 (pre-dialisi/post-dialisi), Settimana 13 (pre-dialisi/post-dialisi), Settimana 17 (pre-dialisi/post-dialisi), Settimana 21 (pre-dialisi/post-dialisi), Settimana 27 (pre-dialisi/post-dialisi), conclusione dello studio (pre-dialisi). |
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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 | Yes |
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 | No |
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.8.2.4 | Number of treatment arms in the trial | 1 |
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.8.4.1 | Number of sites anticipated in Member State concerned | 1 |
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 | No |
E.8.6.2 | Trial being conducted completely outside of the EEA | Information not present in EudraCT |
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.8.9.1 | In the Member State concerned years | 2 |
E.8.9.1 | In the Member State concerned months | 10 |
E.8.9.1 | In the Member State concerned days | 9 |
E.8.9.2 | In all countries concerned by the trial years | 2 |
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 | 9 |