E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
transfusional hemosiderosis in adult and paediatric patients |
Emosiderosi trasfusionale in pazienti adulti e pediatrici |
|
E.1.1.2 | Therapeutic area | Diseases [C] - Blood and lymphatic diseases [C15] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 14.1 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10040641 |
E.1.2 | Term | Sickle cell anaemia |
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 |
Valutare il profilo di sicurezza e tollerabilita' a lungo termine di ICL670 nei pazienti con drepanocitosi ed emosiderosi trasfusionale che necessitano di terapia chelante. |
|
E.2.2 | Secondary objectives of the trial |
Consentire ai pazienti che hanno completato con successo le 52 settimane di trattamento con ICL670 e deferoxamina nello studio ICL670A0109 di proseguire/iniziare la terapia con ICL670.
Stimare le modificazioni relative e assolute delle concentrazioni epatiche di ferro (LIC) e il tasso di escrezione complessiva di ferro (TBIE: Total Body Iron Excretion) in sottogruppi definiti dai valori al basale di LIC (2-7 e > 7 mg/g di tessuto epatico- peso secco),e dai trattamenti (ICL670 e DFO) durante lo studio principale.
Valutare la correlazione tra LIC e potenziali indicatori surrogati quali: ferritina sierica,sideremia,transferrina e saturazione della transferrina per le dosi di ICL670,nonche' indicatori di tollerabilita' indicativi di possibile sovra-chelazione.
Valutare le potenzialita' della Risonanza Magnetica Nucleare (RMN) come metodo non invasivo per la misurazione di LIC in un sottocampione di pazienti.
Raccogliere dati di proteomica,al fine di identificare marker biologici. |
|
E.2.3 | Trial contains a sub-study | Yes |
E.2.3.1 | Full title, date and version of each sub-study and their related objectives |
ALTRI SOTTOSTUDI: BIOMARKERS, RMN
|
|
E.3 | Principal inclusion criteria | |
E.4 | Principal exclusion criteria | |
E.5 End points |
E.5.1 | Primary end point(s) | |
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 | 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 | Yes |
E.8.1.7.1 | Other trial design description |
STUDIO DI ESTENSIONE IN APERTO, NON COMPARATIVO |
|
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | Information not present in EudraCT |
E.8.2.2 | Placebo | Information not present in EudraCT |
E.8.2.3 | Other | Information not present in EudraCT |
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 | No |
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 | No |
E.8.7 | Trial has a data monitoring committee | Information not present in EudraCT |
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 | |
E.8.9.1 | In the Member State concerned months | |
E.8.9.1 | In the Member State concerned days | |