E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Transfusion-dependent ß thalassemia (TDT). Severe sickle cell disease (SCD). |
Beta-talassemia trasfusione dipendente (UDT) Anemia Falciforme grave (SCD) |
|
E.1.1.1 | Medical condition in easily understood language |
Transfusion-dependent ß thalassemia. Severe sickle cell disease. |
Beta-talassemia trasfusione dipendente (TDT) Anemia Falciforme grave (SCD) |
|
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 | 10043391 |
E.1.2 | Term | Thalassaemia beta |
E.1.2 | System Organ Class | 10010331 - Congenital, familial and genetic disorders |
|
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 21.0 |
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 | Yes |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
Evaluate long-term safety up to 15 years after CTX001 infusion in subjects who received CTX001 for treatment of transfusion-dependent thalassemia (TDT) or severe sickle cell disease (SCD) |
Valutare la sicurezza a lungo termine fino a 15 anni dopo l’infusione di CTX001 in soggetti che hanno ricevuto CTX001 per il trattamento della talassemia trasfusione-dipendente (UDT) o dell’anemia falciforme (SCD) grave |
|
E.2.2 | Secondary objectives of the trial |
To evaluate efficacy of CTX001 up to 15 years after CTX001 infusion, in subjects who received CTX001 for treatment of TDT or SCD |
Valutare l'efficacia di CTX001 fino a 15 anni dopo l'infusione di CTX001, in soggetti che hanno ricevuto CTX001 per il trattamento di TDT o SCD |
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. Subjects or legal representative or guardian (if applicable) must sign and date informed consent form (ICF). 2. Subjects must have received CTX001 infusion. |
1. I soggetti o il legale rappresentante o tutore (se applicabile) devono firmare e datare il modulo di consenso informato (ICF) 2. I soggetti devono aver ricevuto l'infusione di CTX001 |
|
E.4 | Principal exclusion criteria |
1. There are no exclusion criteria. |
1. Non ci sono criteri di esclusione |
|
E.5 End points |
E.5.1 | Primary end point(s) |
• New malignancies • New or worsening hematologic disorders (e.g. immune-mediated cytopenias, aplastic anemia, primary immunodeficiencies) • All-cause mortality • All serious adverse events (SAEs) occurring up to 5 years after CTX001 infusion • CTX001-related AEs and SAEs |
• Nuove neoplasie • Disturbi ematologici nuovi o in peggioramento (ad es. citopenie immunomediate, anemia aplastica, immunodeficienze primarie) • Mortalità per tutte le cause • Tutti gli eventi avversi seri (SAE) che si verificano fino a 5 anni dopo l'infusione di CTX001 • Eventi avversi correlati a CTX001 e SAE |
|
E.5.1.1 | Timepoint(s) of evaluation of this end point |
Up to 15 years after CTX001 infusion (with exception of SAEs as noted above). |
Fino a 15 anni dopo l'infusione di CTX001 (ad eccezione dei SAE, come segnalato sopra) |
|
E.5.2 | Secondary end point(s) |
• Total Hemoglobin (Hb) concentration (pre-transfusion) over time • Fetal hemoglobin (HbF) concentration (pre-transfusion) over time • Proportion of alleles with intended genetic modification present in peripheral blood leukocytes over time TDT: • TDT-related transfusion • Iron overload as measured by liver iron concentration (LIC), cardiac • iron concentration (CIC), and ferritin • Proportion of subjects receiving iron chelation therapy over time SCD: • Severe vaso-occlusive crisis (VOC) • SCD-related transfusions; • Hemoglobin (Hb) concentrations over time • HbF concentrations over time TDT: • TDT-related transfusion • Iron overload as measured by liver iron concentration (LIC), cardiac iron concentration (CIC), and ferritin SCD: • Severe vaso-occlusive crisis (VOC) |
• Concentrazioni totale di emoglobina (Hb) (pre-trasfusione ) nel tempo • Concentrazioni di HbF (pre-trasfusione ) nel tempo • Proporzione di alleli con modificazione genetica prevista presente in leucociti del sangue periferico nel tempo
TDT: • Trasfusione dovuta a TDT • Sovraccarico di ferro misurato dalla concentrazione di ferro nel fegato (LIC), concentrazione di ferro cardiaco (CIC) e ferritina • Proporzione di soggetti sottoposti a terapia di chelazione del ferro nel tempo SCD: • Crisi vaso-occlusiva grave (VOC) • SCD correlata a trasfusioni; • Concentrazioni di emoglobina (Hb) nel tempo • Concentrazioni di HbF nel tempo TDT: • Trasfusione dovuta a TDT • Sovraccarico di ferro misurato dalla concentrazione di ferro nel fegato (LIC), concentrazione di ferro cardiaco (CIC) e ferritina SCD: • Crisi vaso-occlusiva grave (VOC) |
|
E.5.2.1 | Timepoint(s) of evaluation of this end point |
Up to 15 years after CTX001 infusion (with exception of LIC and CIC which will be assessed for up to 5 years).; Up to 5 years after CTX001 infusion. |
Fino a 15 anni dopo l'infusione di CTX001 (ad eccezione di LIC e CIC Che saranno rilevati fino a 5 anni).; Fino a 5 dopo l'infusione di CTX001 |
|
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 |
In aperto, follow up a lungo termine |
Open, long term follow up |
|
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 | 6 |
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 |
Canada |
United States |
Belgium |
France |
Germany |
Greece |
Italy |
United Kingdom |
|
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 | 20 |
E.8.9.1 | In the Member State concerned months | 4 |
E.8.9.1 | In the Member State concerned days | 0 |
E.8.9.2 | In all countries concerned by the trial years | 21 |
E.8.9.2 | In all countries concerned by the trial months | 0 |
E.8.9.2 | In all countries concerned by the trial days | 0 |