E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Chronic hepatitis C |
Przewlekłe wirusowe zapalenie wątroby typu C |
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E.1.1.1 | Medical condition in easily understood language |
Chronic hepatitis C caused by hepatitis C virus (HCV) infection |
Przewlekłe wirusowe zapalenie wątroby typu C wywołane przez wirus HCV |
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E.1.1.2 | Therapeutic area | Diseases [C] - Virus Diseases [C02] |
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 | 10019744 |
E.1.2 | Term | Hepatitis C |
E.1.2 | System Organ Class | 10021881 - Infections and infestations |
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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 |
Main objective: to cure patients with chronic hepatitis C: evaluation of the efficacy of sofosbuvir / velpatasvir (SOF / VEL) therapy assessed as SVR (sustained viral response) 12 weeks after the end of treatment |
Cel główny: wyleczenie pacjentów z przewlekłym wirusowym zapaleniem wątroby typu C: ocena skuteczności terapii sofosbuwirem/welpataswirem (SOF/VEL) oceniana jako SVR (sustained viral response, trwała odpowiedź wirusologiczna) po 12 tygodniach od zakończenia leczenia; |
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E.2.2 | Secondary objectives of the trial |
Secondary goals: • assessment of the safety of therapy, • monitoring of patients during treatment, • long-term post-treatment evaluation of patients - assessment of the durability of treatment effects and possible long-term side effects, including effects on children's development |
Cele drugorzędowe: • ocena bezpieczeństwa terapii, • monitorowanie pacjentów w trakcie leczenia, • długoterminowa ocena pacjentów po leczeniu – ocena trwałości efektów terapii i ewentualnych odległych działań niepożądanych, w tym wpływu na rozwój dzieci
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
• written, voluntary informed consent to participate in the study • diagnosis of chronic hepatitis C • age 6-18 years |
• pisemna, dobrowolna i świadoma zgoda na udział w badaniu • rozpoznanie przewlekłego wirusowego zapalenia wątroby typu C • wiek 6-18 lat
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E.4 | Principal exclusion criteria |
• refusal to consent to participate in the study • hypersensitivity to the active substance or excipients of the drug • no HCV RNA in blood serum in the presence of anti-HCV antibodies • pregnancy or breastfeeding • hepatitis B infection (active or past) • simultaneous intake of drugs that affect the interaction with active substances, including rosuvastatin or strong inhibitors of P-glycoprotein • active addiction to alcohol or intoxicants
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• brak zgody na udział w badaniu • nadwrażliwość na substancję czynną lub substancje pomocnicze leku • niestwierdzenie HCV RNA w surowicy krwi przy obecności przeciwciał anty-HCV • ciąża lub karmienie piersią • zakażenie wirusem zapalenia wątroby typu B (aktywne lub przebyte) • równoczesne przyjmowanie leków wpływających na interakcję z substancjami czynnymi, w tym rozuwastatyny lub silnych inhibitorów glikoproteiny P • czynne uzależnienie od alkoholu lub środków odurzających
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E.5 End points |
E.5.1 | Primary end point(s) |
Assessment of the efficacy of sofosbuvir / velpatasvir (SOF / VEL) therapy assessed as SVR (sustained viral response) 12 weeks after the end of treatment;
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Ocena skuteczności terapii sofosbuwirem/welpataswirem (SOF/VEL) oceniana jako SVR (sustained viral response, trwała odpowiedź wirusologiczna) po 12 tygodniach od zakończenia leczenia;
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
12 weeks after the end of the treatment |
12 tygodni od zakończenia leczenia |
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E.5.2 | Secondary end point(s) |
• change in liver enzyme levels • change in total bilirubin • reduction in HCV viral load after 4, 8 and 12 weeks of treatment • change in the stage of liver fibrosis in an elastographic examination, • change of the patient's physical parameters (body weight, height, BMI z-score), • assessment of the occurrence of side effects of treatment, • assessment of the impact of clinical and laboratory factors (including the HCV genotype) on the effectiveness of treatment, • patients' quality of life assessment
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• zmiana stężenia enzymów wątrobowych • zmiana stężenia bilirubiny całkowitej • obniżenie wiremii HCV po 4, 8 i 12 tygodniach leczenia • zmiana zaawansowania włóknienia wątroby w badaniu elastograficznym, • zmiana parametrów fizycznych pacjenta (masy ciała, wzrostu, BMI z-score), • ocena występowania działań niepożądanych leczenia, • ocena wpływu czynników klinicznych oraz laboratoryjnych (w tym genotypu HCV) na skuteczność leczenia, • ocena jakości życia pacjentów
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
4, 8, 12 weeks after the beginning of the treatment; 12 weeks and a year after the end of the treatment |
Po 4, 8, 12 tygodniach leczenia, po 12 tygodniach i po roku od zakończenia leczenia |
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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 | Yes |
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) | No |
E.7.4 | Therapeutic use (Phase IV) | Yes |
E.8 Design of the trial |
E.8.1 | Controlled | No |
E.8.1.1 | Randomised | No |
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 | 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.8.5 | 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 | 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 | 2 |
E.8.9.1 | In the Member State concerned months | 3 |
E.8.9.1 | In the Member State concerned days | 0 |