emoglobinuria parossistica notturna (enp) sindrome di marchiafava-micheli dati epidemiologici la patologia puo presentarsi qualsiasi eta anche se si presenta. In medicina l’anemia emolitica microangiopatica, in inglese Microangiopathic Haemolytic altre malattie: eclampsia, rigetto dell’allotrapianto renale, emoglobinuria parossistica notturna, sclerodermia e vasculiti come poliarterite nodosa e. Trattamento dell’emoglobinuria parossistica notturna. Latvian. Ekulizumabs. Paroksismālas nakts hemoglobinūrijas ārstēšana. Lithuanian. Ekulizumabas.
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Clinical trials The European Union Clinical Trials Register allows you to search for protocol and results information on: Cancer AND drug name.
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Both Female Only Male Only. IMP with orphan designation in the indication.
Trials with results Trials without results. Clear advanced search filters. Date paroesistica which this record was first entered in the EudraCT database:. Title of the trial for lay people, in easily understood, i. The IMP has been designated in this indication as an orphan drug in the Community. Committee on Advanced therapies CAT has issued a classification for this product. Combination product that includes a device, but does not involve an Advanced Therapy.
Paroxysmal nocturnal hemoglobinuria PNH.
BC: Articoli scritti da M.C. Sanzari
Paroxysmal Nocturnal Hemoglobinuria is a rare, acquired, life-threatening disease of the blood that leads to the premature death and impaired production of red blood cells. Valutare sicurezza lungo termine RO Parts 1, 2, and 3: Parti 1, 2 e 3: Safety Parts paorssistica, 2, and 3: Incidence of dose-limiting events 2.
Ex vivo liposome lysis in serum and ex-vivo lysis of antibody-coated erythrocytes 4.
Total and target engaged C5 concentration 5. Sicurezza Parti 1, 2 e 3: Incidenza di eventi dose-limitanti 2. Lisi liposomiale ex vivo nel siero e lisi ex vivo degli eritrociti rivestiti di anticorpi 4. Concentrazione totale e target di C5 occupato 5.
Anemia emolitica microangiopatica
Safety and PD Parts 1, 2, and 3: Approximately 6 months for Part 1; approximately 8 months for Parts 2 and 3. Sicurezza e PD Parti 1, 2 e 3: Approssimativamente 6 mesi per la Parte 1; approssimativamente 8 mesi per le Parti 2 e 3. Efficacy Parts nottjrna and 3: Change in LDH 2. Change in free-haemoglobin hotturna. Proportion of patients with stabilized haemoglobin levels 4. Change in fatigue as measured by the functional assessment of chronic illness therapy fatigue 5.
Change in health-related quality of life as measured by the European Organization for Emlglobinuria and Treatment of Cancer quality of life questionnaire-core 30 6. Number of packed RBC units transfused per patient 7. Time to 1 first transfusion or 2 persistent elevation of LDH 8.
Proportion of patients with complement suppression throughout the dosing interval PK Parts 1, 2, and 3: Efficacia Parti 2 e 3: Cambiamento dei LDH 2. Cambiamento dei livelli di emoglobina libera 3. Proporzione di pazienti con livelli stabilizzati di emoglobina 4.
Cambiamento della condizione di affaticamento misurata tramite il functional assessment of chronic illness therapy FACIT fatigue 5. Efficacy and PK Parts 1, 2, and 3: Approximately 6 months for Part 1; approximately 8 months for Parts 2 and 3; up to a maximum of two years from entry into OLE. Efficacia e PK Parti 1, 2 e parossisticx The trial involves single site in the Member Emoglibinuria concerned. Definition of the end of the trial and justification where it is not the last visit of the last subject nktturna the trial.
Plans for treatment or care after the subject has ended the participation in the trial if it is different from the expected normal treatment of that condition.
Anemia emolitica microangiopatica – Wikipedia
The Sponsor will offer post-trial access to the study drug RO free of charge to eligible patients, i. Please see section 4. Si prega di fare riferimento alla sezione 4.