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Národní klinické studie

Suisse Alémanique
ZÜRICH
Prospective Assessment of MRI for Morphological and Functional Imaging in the Thorax
Universitätsspital Zürich
Institut für diagnostische und interventionelle Radiologie

Cambridgeshire
CAMBRIDGE
APRIL: Safety of Abatacept in Rheumatoid Arthritis Associated Interstitial Lung Disease (a feasibility trial)
Addenbrooke's Hospital
Addenbrookes Hospital

Greater London
LONDON
RBHIPF004 - A randomized, double blind controlled trial comparing rituximab against intravenous cyclophosphamide in connective tissue disease associated interstitial lung disease
National Heart and Lung Institute, Imperial College, Royal Brompton Campus
Interstitial Lung Disease Unit

WIEN
WIEN


HCQ4Surfdefect: Hydroxychloroquine (HCQ) in pediatric ILD (interstitial lung disease) - AT
Allgemeines Krankenhaus der Stadt Wien
Klinische Abteilung für Pädiatrische Pulmologie, Allergologie und Endokrinologie

WIEN
WIEN
GOOD-IDES: An Open-Label Phase II Study in Anti-GBM Disease (Goodpasture's Disease) With Adverse Renal Prognosis to Evaluate the Efficacy and Safety of IdeS - AT
Allgemeines Krankenhaus der Stadt Wien
Klinische Abteilung für Nephrologie und Dialyse

ARRONDISSEMENT BRUSSELS-CAPITAL
BRUSSELS
Un essai en double aveugle, randomisé et contrôlé par placebo évaluant l'efficacité et l'innocuité du BI 1015550 sur 52 semaines chez des patients atteints de maladie pulmonaire interstitielle fibrosante progressive - BE
Cliniques Universitaires de Bruxelles - Hôpital Erasme
Service de Pneumologie

ARRONDISSEMENT BRUSSELS-CAPITAL
BRUSSELS
Une étude multicentrique, randomisée, en double aveugle, contrôlée par placebo, de phase 2 portant sur l'efficacité, l'innocuité et la tolérabilité du BMS-986278 chez des participants atteints de fibrose pulmonaire - BE
Cliniques universitaires Saint-Luc - UCLouvain
Service de pneumologie

Bayern
MÜNCHEN

chILD-EU_EAA: Stop exogenous allergic alveolitis in childhood (StopEAA) - Europäische, prospektive, multizentrische, randomisierte, doppelblinde, Placebo-kontrollierte Studie zur Implementierung eines Verfahrens für die tägliche Behandlung der exogen-allergischen Alveolitis (Phase II)
Kinderklinik und Kinderpoliklinik im Dr. von Haunerschen Kinderspital
Christiane Herzog Ambulanz

Victoria
ADDRESS: NOT PROVIDED - AU
Effects of N-Acetyl-L-Leucine on Niemann-Pick Disease Type C (NPC): A Phase III, Randomized, Placebo-controlled, Double-blind, Crossover Study -AU
Institution: Information not provided - AU

STEIERMARK
GRAZ
ANCA_CGM: Influence of once vs twice daily corticosteroid administration on glycemia assessed by continuous glucose monitoring in patients with ANCA vasculitis (phase 4)
Medizinische Universität Graz
Klinische Abteilung für Nephrologie

South-West region
SOFIA

ENGAGE - A phase 3, randomized, double-blind, placebo-controlled, multi-center study confirming the efficacy and safety of Genz-112638 in patients with Gaucher disease type 1 - BG -
University Hospital Alexandrovska
Clinic of Hematology

Capital City Prague
PRAHA

ENCORE : A phase 3, randomized, multi-center, multi-national, open-label, active comparator study to evaluate the efficacy and safety of Genz-112638 in patients with Gaucher disease type 1 who have reached therapeutic goals with enzyme replacement therapy - CZ -
Charles University - First faculty of medicine
National Centre for Treatment of Gaucher Disease

HAUTS-DE-FRANCE
ADDRESS: NOT PROVIDED - FR

MASTER-ANCA: Etude prospective multicentrique randomisée comparant l'arrêt ou la poursuite du traitement immunosuppresseur d'entretien des vascularites associées aux ANCA chez les patients en Insuffisance Rénale Terminale.
Institution: Information not provided - FR

ILE-DE-FRANCE
PARIS
PEXIVAS: Plasma Exchange and Glucocorticoid Dosing in the Treatment of Anti-neutrophil Cytoplasm Antibody Associated Vasculitis: an International Randomized Controlled Trial (phase III) - FR
AP-HP.Centre - Université de Paris - Hôpital Cochin
Unité fonctionnelle de Médecine interne et centre de référence maladies rares

Nordrhein-Westfalen
MÜNSTER

Auswirkungen von N-Acetyl-L-Leucin auf die Niemann-Pick-Krankheit Typ C (NPC): Eine randomisierte, plazebokontrollierte, doppelblinde Crossover-Studie der Phase III
Universitätsklinikum Münster
Centrum für seltene Erkrankungen (ZSE) Münster

ISRAEL
PETAH TIKVA

ENGAGE - A phase 3, randomized, double-blind, placebo-controlled, multi-center study confirming the efficacy and safety of Genz-112638 in patients with Gaucher disease type 1 - IL -
Rabin Medical Center - Beilinson Hospital
Recanati Genetic Institute

CAMPANIA
NAPOLI
Valutazione dell'efficacia e del trattamento con Miglustat in pazienti affetti da malattia di Niemann-Pick di tipo c
Azienda Ospedaliera Universitaria "Federico II"
Laboratorio Dipartimento di Pediatria

FRIULI VENEZIA GIULIA
UDINE

ENCORE : Studio di fase 3, multicentrico nazionale, open-label, randomizzato per valutare l'efficacia e la sicurezza di Genz-112638 in pazienti con patologia di Gaucher tipo 1 trattati con terapia enzimatica sostitutiva.
Azienda Ospedaliero-Universitaria "Santa Maria della Misericordia" di Udine
Centro di Coordinamento Regionale per le Malattie Rare

PUGLIA
BARI

Terapia combinata della crioglobulinemia mista HCV-correlata con Interferone-alpha pegilato, ribavirina e rituximab
Azienda Ospedaliero-Universitaria Consorziale Policlinico di Bari
Unità di Epatologia - Sezione di Medicina Interna e Oncologia Clinica

Beyrouth
BEIRUT

ENGAGE - A phase 3, randomized, double-blind, placebo-controlled, multi-center study confirming the efficacy and safety of Genz-112638 in patients with Gaucher disease type 1 - LB -
Hôtel-Dieu de France, Université Saint-Joseph
Oncology & Hematology

Bratislavsky kraj
ADDRESS: NOT PROVIDED - SK
Effects of N-Acetyl-L-Leucine on Niemann-Pick Disease Type C (NPC): A Phase III, Randomized, Placebo-controlled, Double-blind, Crossover Study -SK
Institution: Information not provided - SK

Madrid
ADDRESS: NOT PROVIDED - ES


ELIKIDS: Estudio abierto, de dos cohortes (con y sin imiglucerasa) y multicéntrico para evaluar la farmacocinética (FC), seguridad y eficacia de eliglustat en pacientes pediátricos con enfermedad de Gaucher (EG) tipo 1 (EG1) y tipo 3 (EG3) -ES
Institution: Information not provided - ES

Madrid
ADDRESS: NOT PROVIDED - ES

TransportNPC: Estudio fase 3, multicéntrico, aleatorizado, doble ciego, de grupos paralelos y controlado con placebo, para evaluar la seguridad, la tolerabilidad y la eficacia de 2000 mg/kg de Trappsol Cyclo (hidroxipropil-beta-ciclodextrina) y tratamiento estándar comparado con placebo y tratamiento estándar en pacientes con la enfermedad de Niemann-Pick de tipo C1 -ES
Institution: Information not provided - ES

Suisse Alémanique
BERN
Effects of N-Acetyl-L-Leucine on Niemann-Pick Disease Type C (NPC): A Phase III, Randomized, Placebo-controlled, Double-blind, Crossover Study - CH
Universitätsspital Inselspital
University Hospital Inselspital

TUNISIA
TUNIS

ENGAGE - A phase 3, randomized, double-blind, placebo-controlled, multi-center study confirming the efficacy and safety of Genz-112638 in patients with Gaucher disease type 1 - TN -
Hôpital La Rabta
Service de Pédiatrie

TURKEY
IZMIR

ENCORE : A phase 3, randomized, multi-center, multi-national, open-label, active comparator study to evaluate the efficacy and safety of Genz-112638 in patients with Gaucher disease type 1 who have reached therapeutic goals with enzyme replacement therapy - TR -
Ege Üniversitesi
Department of Pediatrics - Cocuk asgligi ve Hastaliklari Anabilim Dali

Cambridgeshire
CAMBRIDGE

Rituximab and Belimumab Combination Therapy in PR3 COMBIVAS
Addenbrooke's Hospital
Addenbrookes Hospital

Greater London
ADDRESS: NOT PROVIDED - GB
Effects of N-Acetyl-L-Leucine on Niemann-Pick Disease Type C (NPC): A Phase III, Randomized, Placebo-controlled, Double-blind, Crossover Study -GB
Institution: Information not provided - GB

Greater London
LONDON

ENDURRANCE-1: Exploring durable remission with rituximab in ANCA associated vasculitis-GB
Imperial College London, main campus
Imperial College London Headquarters

Greater London
LONDON

ENDURRANCE-1: Exploring durable remission with rituximab in ANCA associated vasculitis-GB
Imperial College NHS Trust

Washington
ADDRESS: NOT PROVIDED - US


Role of Oxidative Stress and Inflammation in Type 1 Gaucher Disease (GD1): Potential Use of Antioxidant/Anti-inflammatory Medications
Institution: Information not provided - US

WIEN
ADDRESS: NOT PROVIDED - AT

OCEAN: A 52-week, randomized, double-blind, double-dummy, parallel-group, multi-centre, non-inferiority study to investigate the efficacy and safety of depemokimab compared with mepolizumab in adults with relapsing or refractory Eosinophilic Granulomatosis with Polyangiitis (EGPA) receiving standard of care (SoC) therapy - AT
Institution: Information not provided - AT

AUVERGNE-RHONE-ALPES
PIERRE-BENITE

MAINEPSAN: Etude prospective, multicentrique, contrôlée, randomisée, évaluant une administration prolongée de prednisone en traitement d'entretien dans les vascularites systémiques associées aux ANCA.
CHU de Lyon HCL - GH Sud
Service de médecine interne et pathologie vasculaire

HAUTS-DE-FRANCE
ADDRESS: NOT PROVIDED - FR
Programme d'accès à long terme au mépolizumab pour les sujets ayant participé à l'étude MEA115921 (étude du mépolizumab dans le traitement de la granulomatose éosinophilique avec polyangéite chez des sujets recevant un traitement de référence) - FR
Institution: Information not provided - FR

ILE-DE-FRANCE
PARIS

CHUSPAN2 : Evaluation d'une nouvelle stratégie de traitement de patients atteints de polyangéite microscopique, périartérite noueuse ou syndrome de Churg-Strauss sans facteur de pronostic défavorable (Phase III)
AP-HP.Centre - Université de Paris - Hôpital Cochin
Unité fonctionnelle de Médecine interne et centre de référence maladies rares

Baden-Württemberg
MANNHEIM

Eine randomisierte, doppelblinde, Double-Dummy, aktiv kontrollierte, multizentrische, 2-teilige Phase-II-Studie zum Ersatz von Steroiden durch IFX-1 bei aktiver Granulomatose mit Polyangiitis (GPA) und mikroskopischer Polyangiitis (MPA)
Universitätsmedizin Mannheim
Universitätsklinikum Mannheim

Berlin
ADDRESS: NOT PROVIDED - DE
Mepolizumab Langzeitzugangsprogramm für Probanden, die an der Studie MEA115921 (Placebo-kontrollierte Studie von Mepolizumab zur Behandlung von eosinophiler Granulomatose mit Polyangiitis bei Probanden mit Standardtherapie) teilgenommen haben. - DE-
Institution: Information not provided - DE

Berlin
BERLIN

Eine randomisierte, doppelblinde, Double-Dummy, aktiv kontrollierte, multizentrische, 2-teilige Phase-II-Studie zum Ersatz von Steroiden durch IFX-1 bei aktiver Granulomatose mit Polyangiitis (GPA) und mikroskopischer Polyangiitis (MPA)
Charité - Universitätsmedizin Berlin

Nordrhein-Westfalen
ESSEN

Eine randomisierte, doppelblinde, Double-Dummy, aktiv kontrollierte, multizentrische, 2-teilige Phase-II-Studie zum Ersatz von Steroiden durch IFX-1 bei aktiver Granulomatose mit Polyangiitis (GPA) und mikroskopischer Polyangiitis (MPA)
Universitätsklinikum Essen

Nordrhein-Westfalen
MÜNSTER

Eine randomisierte, doppelblinde, Double-Dummy, aktiv kontrollierte, multizentrische, 2-teilige Phase-II-Studie zum Ersatz von Steroiden durch IFX-1 bei aktiver Granulomatose mit Polyangiitis (GPA) und mikroskopischer Polyangiitis (MPA)
Universitätsklinikum Münster

Sachsen
DRESDEN

Eine randomisierte, doppelblinde, Double-Dummy, aktiv kontrollierte, multizentrische, 2-teilige Phase-II-Studie zum Ersatz von Steroiden durch IFX-1 bei aktiver Granulomatose mit Polyangiitis (GPA) und mikroskopischer Polyangiitis (MPA)
Universitätsklinikum Carl Gustav Carus an der TU Dresden
Universitätsklinikum Dresden

Thüringen
JENA

Groningen
GRONINGEN


Prevention of Relapses in Proteinase 3 (PR3)-Anti-Neutrophil Cytoplasmic Antibodies (ANCA)-Associated Vasculitis
UMCG - Universitair Medisch Centrum Groningen
Afdeling Nefrologie

Cataluña
BARCELONA

ABROGATE: Abatacept (CTLA4-Ig) para el tratamiento de la recaída no grave de granulomatosis con poliangeítis (Wegener)
Hospital Clínic de Barcelona

Madrid
ADDRESS: NOT PROVIDED - ES

OCEAN: Un estudio de 52 semanas, aleatorizado, doble ciego, con doble simulación, de grupos paralelos, multicéntrico, de no inferioridad para investigar la eficacia y seguridad de depemokimab en comparación con mepolizumab en adultos con granulomatosis eosinofílica con poliangeítis (EGPA, por sus siglas en inglés) recidivante o refractaria que reciben el tratamiento habitual -ES
Institution: Information not provided - ES

Washington
ADDRESS: NOT PROVIDED - US


Phase 1/2a Study of 2-Hydroxypropyl-Beta-Cyclodextrin Therapy for Infantile Liver Disease Associated With Niemann-Pick Disease, Type C
Institution: Information not provided - US
Nadnárodní klinické studie

Alberta
MOUNTAIN VIEW

Utrecht
ADDRESS: NOT PROVIDED - NL
A Multicenter Extension Study of Taliglucerase Alfa in Pediatric Subjects With Gaucher Disease (Phase III) (Coordination)
Institution: Information not provided - NL

Utrecht
ADDRESS: NOT PROVIDED - NL
EDGE: A Phase 3, Randomized, Multi-Center, Multi-National, Double-Blind Study to Evaluate the Efficacy, Safety, and Pharmacokinetics of Once Daily versus Twice Daily Dosing of Genz-112638 in Patients with Gaucher Disease Type 1 who have Demonstrated Clinical Stability on a Twice Daily Dose of Genz-112638
Institution: Information not provided - NL

Zuid-Holland
LEIDEN
ENDURRANCE-1: Exploring durable remission with rituximab in ANCA associated vasculitis
LUMC - Leids Universitair Medisch Centrum

Greater London
ADDRESS: NOT PROVIDED - GB
Effects of N-Acetyl-L-Leucine on Niemann-Pick Disease Type C (NPC): A Phase III, Randomized, Placebo-controlled, Double-blind, Crossover Study
Institution: Information not provided - GB

Oxfordshire
BEGBROKE

Massachusetts
CAMBRIDGE

Pennsylvania
PHILADELPHIA
PEXIVAS: Plasma Exchange and Glucocorticoid Dosing in the Treatment of Anti-neutrophil Cytoplasm Antibody Associated Vasculitis: an International Randomized Controlled Trial (phase III) (coordination)
Hospital of the University of Pennsylvania
Rheumatology Division

Suisse Romande
ADDRESS: NOT PROVIDED - CH
A phase IIa, international, multicentre, open-label, uncontrolled study to evaluate the safety and pharmacokinetics of 4× 375 mg/m2 intravenous rituximab in paediatric patients with severe granulomatosis with polyangiitis (Wegener's) or microscopic polyangiitis
Institution: Information not provided - CH

Greater London
ADDRESS: NOT PROVIDED - GB
A Double-blind, Randomised, Placebo-controlled Study to Investigate the Efficacy and Safety of Mepolizumab in the Treatment of Eosinophilic Granulomatosis with Polyangiitis in Subjects Receiving Standard of Care Therapy (Phase III)
Institution: Information not provided - GB

Greater London
ADDRESS: NOT PROVIDED - GB